A Phase II Randomized, Placebo-Controlled, Double Blinded, Multi-Centre Clinical Trial of Pimozide in Patients With Amyotrophic Lateral Sclerosis

Study Purpose:

This study will look at whether Pimozide may help to slow the progression of Amyotrophic Lateral Sclerosis.

100 people from several Canadian centres with ALS who have provided their consent will be randomly assigned into one of 2 groups. The first group will receive a dose of up to 2mg of Pimozide per day and the second group will receive placebo (lactose tablets). Subjects will be assigned randomly (like by a flip of a coin) to receive either Pimozide 2 mg per day or placebo tablets. There will be a fifty-fifty chance of receiving Pimozide or placebo.

Participants will be on study medication up to 22 weeks, and on study up to 26 weeks. There are 8 clinic visits and 1 phone visit over the course of the Treatment Phase of the study. The second phase which is Observational, is optional with follow-up for up to 5 years from the end of the Treatment Phase.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Lawrence Korngut, M.D. University of Calgary and Alberta Health Services

Clinicaltrials.gov ID (11 digit #):

NCT03272503

Neals Affiliated?

No

Coordinating Center Contact Information

University of Calgary
Janet Petrillo, M.Sc. / .(JavaScript must be enabled to view this email address) / 403-210-7006
.(JavaScript must be enabled to view this email address) Calgary, Alberta Canada

Full Study Summary:

Amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease is a neuromuscular disease that results in rapid decline in normal muscle function and tone leading to difficulties with mobility, eating, drinking, breathing, sleeping, and communicating. The disease is progressive and no cure currently exists. Most people diagnosed with ALS succumb within 3 to 5 years. Rilutek® (riluzole) has been approved as a treatment to slow progression of ALS, but is minimally effective with mean increase in survival of only a few months. Radicava® or Radicut® (edaravone) has recently been approved in Canada, USA, Japan and South Korea.

Muscular dysfunction present in people with ALS is caused by nerve breakdown and a dysfunction in the communication between the muscles and the nerves. The area where these communications occur is called the neuromuscular junction. Some recent studies have focused on using different medications to enhance communication at the neuromuscular junction with the goal of improving muscle function as a result. This approach is unproven but may help to slow the progression of the disease.

Pimozide is a medication that has been demonstrated to enhance communication at the neuromuscular junction in fish and mice. This study will look at whether Pimozide may help to slow the progression of ALS.

There are two parts to this study.

Treatment Phase: In the first part of this study, 100 people from several Canadian centres with ALS who have provided their consent will be randomly assigned into one of 2 groups. The first group will receive a dose of up to 2mg of Pimozide per day and the second group will receive placebo (lactose tablets). Subjects will be assigned randomly (like by a flip of a coin) to receive either Pimozide 2 mg per day or placebo tablets. There will be a fifty-fifty chance of receiving Pimozide or placebo.

Each Pimozide tablet contains 2 mg of Pimozide. The matching placebo tablets for this study will look exactly like the Pimozide tablets. Placebos are used in clinical trials to find out if the results observed in the study are due the drug being tested, or for other reasons.

Neither the subject nor their doctor will know which group a patient belongs to. However, if an emergency should arise, information about a treatment group will be shared with their doctor to ensure appropriate medical care. Participants will take their treatment once a day, every day for about 22 weeks. The total time in the study from the screen visit up until the last phone call communication is about 26 weeks.

Observational Phase: The second part of this study is optional. It is each subject's decision whether to participate only in the first part of this study, or in both parts of the study, or not at all. In the second part of this study, the Canadian Neuromuscular Disease Registry (CNDR) will collect data on overall ALS progression using the Revised ALS Functional Rating Scale (ALSFRS-R) and breathing using Vital Capacity data collected during breathing tests. This information will be collected from a subject medical record following each routine clinical appointment. Data will be collected at each routine clinic visit for up to 5 years from the end of the first part of the study. There will be no extra visits for this part of the study beyond routine ALS clinic visits.

The information collected during this part of the study will be used to compare the progression of ALS, after the clinical trial is completed, among the two treatment types (Placebo or 2 mg per day). By analyzing this information, the researchers conducting this study hope to determine if Pimozide may help to slow the progression of ALS. To participate in this part of the study consent must be provided to join the CNDR. A subject who hasn't already provided their CNDR consent, and wishes to participate in this part of the study will be given a CNDR consent form to review and sign in addition to this consent form. A subject already participating in the CNDR will just need to sign the main study consent form.

Study Sponsor:

University of Calgary

Participant Duration:

Estimated Enrollment:

100

Estimated Study Start Date:

10/27/2017

Estimated Study Completion Date:

12/31/2019

Posting Last Modified Date:

10/15/2019

Date Study Added to alsconsortium.org:

10/15/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Patients classified as having clinically definite, clinically probable, clinically probable (laboratory-supported) or clinically possible ALS according to the El-Escorial diagnostic criteria for ALS (see Appendix 2).
    Able to comprehend and willing to sign Informed Consent Form (ICF).
    Age 18 years of age or greater.
    ALS Symptom onset of muscle weakness or speech impairment no more than 48 months prior to screen visit. Fasiculations should not be considered.
    Slow Vital Capacity (SVC) greater than or equal to 50% predicted for sex, age and height at screen.
    Has the ability to swallow tablets/capsules whole at study entry.
    Subject with clinical laboratory findings within the normal range or, if outside the normal range, determined by the Investigator at the Screening visit to be not clinically significant.
    If the subject is taking Riluzole the dose must be stable for 30 days prior to the randomization visit. Riluzole cannot be initiated during the study.
    If the subject is receiving Edaravone therapy, the dose must be stable for at least 1 cycle of infusion treatments before the randomization visit.

    Exclusion Criteria:

    History of laryngeal spasm, dystonia, or akathisia.
    Diagnosis of ongoing symptomatic Restless Leg Syndrome or undergoing current treatment for Restless Leg Syndrome. If subject has symptoms that resemble or have the potential to be Restless Leg Syndrome, then further investigation should be undertaken to confirm or rule out diagnosis of Restless Leg Syndrome.
    Any history of moderate or severe traumatic brain injury as defined by a Glasgow Coma Scale Score of less than 13/15 at any time point following a head injury without sedation or other reason for a decreased level of consciousness.
    History of neuroleptic malignant syndrome.
    History of hypersensitivity or serious adverse reaction(s) to a neuroleptic medication.
    History of hyponatremia < 130 mmol/L
    Subject with aspartate aminotransferase (AST) or alanine aminotransferase (ALT) value >3.0 times the upper limit of normal at the Screening visit.
    Current heparin or warfarin use.
    History of hepatic and/or renal impairment that may affect pimozide metabolism
    History of current pregnancy, or breastfeeding women, or women planning to become pregnant. Female subjects of childbearing potential (sexually mature female who has not undergone a hysterectomy or who has not been post-menopausal for 12 consecutive months), must practise effective contraception during the study and be willing and able to continue contraception until the Follow-up phone visit after discontinuing study medication. Abstinence can be considered an acceptable method of contraception at the discretion of the investigator.
    Current antipsychotic use
    Presence of central nervous system depression, comatose states, liver disorders, renal insufficiency, and blood dyscrasias
    Presence of Parkinson's syndrome
    Presence of major depressive disorders as determined by site Investigator.
    History of clinically significant ECG abnormalities at screen visit, including QTc>500ms.
    History of congenital long QT syndrome or with a family history of this syndrome and in patients with a history of cardiac arrhythmias or Torsade de Pointes.
    Presence of acquired long QT interval, and/or concomitant use of drugs known to prolong the QT interval (TCAs, opioids such as methodone, quinolone antibiotics (ciprofloxacin), antimalarials (quinine), Detrol, azole antifungals, Class 1A, III and 1C antiarrhythmics, and macrolide antibiotics.
    Presence of clinically significant bradycardia (heart rate < 50 beats per minute)
    Presence of hypokalemia or hypomagnesemia.
    The concomitant use of CYP 3A4-inhibiting drugs such as azole antimycotics, antiviral protease inhibitors, macrolide antibiotics and nefazodone.
    The concomitant use of CYP 2D6-inhibiting drugs such as quinidine is also contraindicated.
    Concomitant use of serotonin reuptake inhibitors, such as, sertraline, paroxetine, citalopram and escitalopram.*
    Has taken any compound under current or known future study as a potential therapy (including Withania) for ALS less than 30 days prior to dosing OR history of exposure to stem cell therapy for treatment of ALS at any time.

    Current Neurological impairment due to a condition other than ALS:

    • Subject in whom causes of neuromuscular weakness other than ALS have not been excluded.
    • Subject with a diagnosis of other neurodegenerative diseases (e.g., Parkinson's disease, Frontotemporal dementia, Alzheimer's disease, etc.)

    Use of non-invasive ventilation (BiPAP or CPAP) prior to Baseline visit at any time.
    Cognitive impairment as determined by the Site Investigator, subject must not have an impaired ability to provide informed consent and must be able to understand study processes and comply with study procedures.
    Extrapyramidal Symptom Rating Scale (ESRS) Parkinsonism score of 2 on 2 items or a score > 3 on 1 item; OR Dystonia score of >3 on at least 1 item or a score of 2 on 2 items; OR Tardive Dyskinesia score of >3 on at least 1 item or a score of 2 on 2 items. Do not consider scores greater than 3 for Tremor in any region if due to Benign Essential, Exaggerated, or Physiological Tremor.

    The concomitant use of SSRIs and tricyclic antidepressants (e.g. amitriptyline, amoxaprine, desipramine, doxepin, imipramine, nortriptyline, protripyline, trimipramine) - and Tolterodine (Detrol) CYP2D6 inhibitor.

    • Prohibited medications such as tricyclic antidepressants, antimalarials, and serotonin reuptake inhibitors,(ie sertraline, paroxetine, citalopram, fluoxetine, vilazodone and escitalopram) may be weaned to full discontinuation at the screening visit after consent has been signed (no study procedures including adjustments to medication may occur until informed consent has been provided).

  • Site Contact Information

    South Health Campus
    Janet Petrillo, MSc / .(JavaScript must be enabled to view this email address) / 403-210-7006
    Calgary, Alberta
    Canada

    University of Alberta
    Kelsey Tymkow / .(JavaScript must be enabled to view this email address) / 780-915-3425
    Edmonton, Alberta
    Canada

    Stan Cassidy Centre for Rehabilitation
    Susan McCully, MN / .(JavaScript must be enabled to view this email address) / 506-447-4095
    Fredericton, New Brunswick
    Canada

    McMaster University/Hamilton Health Services
    Daniela Trapsa / .(JavaScript must be enabled to view this email address) / 905-521-2100 ext 76365
    Hamilton, Ontario
    Canada

    London Health Sciences Centre
    Christine Piechowicz, BA / .(JavaScript must be enabled to view this email address) / 519-685-8500 ext 34858
    London, Ontario
    Canada

    Ottawa Hospital Research Institute
    Sergio Guber, MSc / .(JavaScript must be enabled to view this email address) / 613-798-5555 ext 19627
    Ottawa, Ontario
    Canada

    Sunnybrook Research Institute
    Liane Phung, BSc / .(JavaScript must be enabled to view this email address) / 416-480-6100 ext 87561
    Toronto, Ontario
    Canada

    Reserche Sepmus Inc.
    Kim Desilets, B.Sc. / .(JavaScript must be enabled to view this email address) / 450-672-1931 ext 247
    Greenfield Park, Quebec
    Canada

Radicava®/(Edaravone) Findings in Biomarkers From ALS (REFINE ALS)

Study Purpose:

The purpose of the study is to identify putative biomarker(s) to serve as quantifiable biological non-clinical measure(s) of Edaravone’s pharmacodynamic effect in volunteers with ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Observational Study

Study Category:

Biomarkers/Imaging

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

James Berry, MD, MPH, Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):

Neals Affiliated?

Yes

Coordinating Center Contact Information

Massachusetts General Hospital
Matthew Davis / .(JavaScript must be enabled to view this email address) / 617-724-4246
.(JavaScript must be enabled to view this email address) Neurological Clinical Research Institute
165 Cambridge Street
Boston, Massachusetts 02114 United States

Full Study Summary:

This is a prospective, observational, longitudinal, multicenter study.  Participants, who are scheduled to receive Edaravone and fulfill all eligibility criteria, will be prospectively enrolled by investigators.The decision to treat participants with Edaravone will be independent from the decision to enroll the participant in the study. The intervention is limited to the collection of blood and urine samples for biomarker testing.Participants in this study will be followed from enrollment up to 24 weeks after treatment initiation (6 treatment cycles, corresponding to a treatment period of approximately 24 weeks) or premature study discontinuation.

Study Sponsor:

Mitsubishi Tanabe Pharma America, Inc.

Participant Duration:

 24 weeks

Estimated Enrollment:

300

Estimated Study Start Date:

07/29/2019

Estimated Study Completion Date:

01/31/2020

Posting Last Modified Date:

09/19/2019

Date Study Added to alsconsortium.org:

09/19/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria

    1. Male and female aged 18 years or older at enrollment
    2. Sporadic or familial ALS diagnosed as possible, probable, probable-laboratory supported or definite as defined by the World Federation of Neurology revised El Escorial criteria
    3. Decision made to prescribe Edaravone prior to screening
    4. Participant will likely be able to obtain commercial Edaravone and likely to complete 6 cycles of treatment, per site investigator estimation
    5. Participant either naïve to Edaravone or who did not receive any Edaravone dose within 1 month prior to screening
    6. Signed informed consent by the subject, or a witness if a subject cannot read or write or is physically unable to talk or write, obtained before any study-related activities are undertaken

    Exclusion Criteria

    Study participants meeting any of the following criteria during screening evaluations will be excluded from entry into the study:

    1. Participant with a contraindication to Edaravone
    2. Participant is participating in an interventional clinical trial

  • Site Contact Information

    Barrow Neurological Institute
    Gale Kittle, RN / .(JavaScript must be enabled to view this email address) / 602-406-4792
    Phoenix, Arizona 85013
    United States

    Neuromuscular Research Center
    Kristy Osgood / .(JavaScript must be enabled to view this email address) / 480-314-1007
    Phoenix, Arizona 85028
    United States

    Phoenix VA Health Care System
    Angela Kuramoto / .(JavaScript must be enabled to view this email address) / 602-277-5551
    Phoenix, Arizona 85028
    United States

    University of California, San Francisco (UCSF)
    Hannah George / .(JavaScript must be enabled to view this email address) / 415-502-9676
    San Francisco, California 94143
    United States

    UCLA ALS CLINIC
    Gilda Avila-Rinek / .(JavaScript must be enabled to view this email address) / 310-825-1806
    Los Angeles, California 90095
    United States

    UC Irvine
    Veronica Martin / .(JavaScript must be enabled to view this email address) / 714-456-7760
    Orange, California 92868
    United States

    Forbes Norris MDA/ALS Research Center
    Marguerite Engel / .(JavaScript must be enabled to view this email address) / 415-600-3758
    San Francisco, California 94115
    United States

    University of California, Davis
    Colleen Anthonisen / .(JavaScript must be enabled to view this email address) / 916-734-4307
    Sacramento, California 95817
    United States

    VA GLA ALS Multidisciplinary Clinic
    Andrea Hanssen / .(JavaScript must be enabled to view this email address) / 310-478-3711 x47091
    Los Angeles, California 90073
    United States

    University of Colorado
    Brianna Blume / .(JavaScript must be enabled to view this email address) / 303-724-6386
    Aurora, Colorado 80045
    United States

    Mayo Clinic Florida
    Janay Caradonna / .(JavaScript must be enabled to view this email address) / 904-953-3128
    Jacksonville, Florda 32224
    United States

    University of South Florida
    Brittany Harvey / .(JavaScript must be enabled to view this email address) / 813-974-9413
    Tampa, Florida 33612
    United States

    University of Florida, Jacksonville
    Cathy Bailey / .(JavaScript must be enabled to view this email address) / 904-244-9814
    Jacksonville, Florida 32209
    United States

    Northwestern University
    Ben Joslin / .(JavaScript must be enabled to view this email address) / 312-503-7504
    Chicago, Illinois 601611
    United States

    University of Kansas Medical Center
    Samantha Colgan / .(JavaScript must be enabled to view this email address) / 913-945-9938
    Kansas City, Kansas 66160
    United States

    Johns Hopkins University
    Betsy Mosmiller / .(JavaScript must be enabled to view this email address) / 410-502-0495
    Baltimore, Maryland 21287
    United States

    University of Massachusetts Medical School
    Catherine Douthwright / .(JavaScript must be enabled to view this email address) / 774-441-7696
    Worcester, Massachusetts 01655
    United States

    Massachusetts General Hospital
    Cassandra Lieberman / .(JavaScript must be enabled to view this email address) / 617-643-7428
    Boston, Massachusetts 02114
    United States

    Spectrum Health
    Nichole Roderique / .(JavaScript must be enabled to view this email address) / 616-774-8637
    Grand Rapids, Michigan 49525
    United States

    Henry Ford Health System
    Anne Vallis / .(JavaScript must be enabled to view this email address) / 313-916-1364
    Detroit, Michigan 48202
    United States

    Mayo Clinic Rochester
    Michelle Turner / .(JavaScript must be enabled to view this email address) / 507-538-5523
    Rochester, Minnesota 55905
    United States

    HealthPartners Institute
    Carlie Leyde / .(JavaScript must be enabled to view this email address) / 651-495-6371
    Minneapolis, Minnesota 55414
    United States

    University of Missouri Health Care
    Natalie Taylor / .(JavaScript must be enabled to view this email address) / 573-884-4862
    Columbia , Missouri 65212
    United States

    Washington University School of Medicine
    Elizabeth Karanja / .(JavaScript must be enabled to view this email address) / 314-273-1871
    St. Louis, Missouri 63110
    United States

    Saint Louis University
    Susan Brown / .(JavaScript must be enabled to view this email address) / 314-977-4900
    St. Louis, Missouri 63104
    United States

    Neurology Associates, P.C.
    Ashley Calhoun / .(JavaScript must be enabled to view this email address) / 402-770-7403
    Lincoln, Nebraska 68506
    United States

    Columbia University
    Jessica Singleton / .(JavaScript must be enabled to view this email address) / 212-305-2233
    New York, New York 10032
    United States

    Atrium Health
    Allison Newell-Sturdivant / .(JavaScript must be enabled to view this email address) / 704-355-5285
    Carolinas HealthCare System Neurosciences Institute
    Charlotte, North Carolina 28203
    United States

    The Cleveland Clinic
    Debbie Hastings / .(JavaScript must be enabled to view this email address) / 216-444-0173
    Cleveland, Ohio 44195
    United States

    Providence Brain and Spine ALS Center
    Arlena Cummings / .(JavaScript must be enabled to view this email address) / 503-962-1171
    Portland, Oregon 97213
    United States

    Temple University
    Kathleen Hatala, RN, BSN / .(JavaScript must be enabled to view this email address) / 215-707-4171
    Philadelphia , Pennsylvania 19140
    United States

    Medical University of South Carolina
    Katrina Madden / .(JavaScript must be enabled to view this email address) / 843-792-9186
    Charleston, South Carolina 29425
    United States

    Houston Methodist
    Sharon Halton / .(JavaScript must be enabled to view this email address) / 713-441-3420
    Houston, Texas 77030
    United States

    University of Texas Health Science Center, San Antonio
    Pamela Kittrell / .(JavaScript must be enabled to view this email address) / 210-450-0524
    San Antonio, Texas 78229
    United States

    Virginia Commonwealth University
    Tamika Walthour / .(JavaScript must be enabled to view this email address) / 804-874-5674
    Richmond, Virginia 23298
    United States

    Swedish Medical Center
    Lindsey Maassel / .(JavaScript must be enabled to view this email address) / 206-320-7121
    Seattle, Washington 98122
    United States

    University of Washington Medical Center
    Laura Sissons-Ross / .(JavaScript must be enabled to view this email address) / 206-543-0081
    Seattle, Washington 98195
    United States

    Medical College of Wisconsin
    Lynn Wheeler / .(JavaScript must be enabled to view this email address) / 414-805-9307
    Milwaukee, Wisconsin 53226
    United States

Open Label, Off Label Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of Ciprofloxacin/Celecoxib Combination in Patients With ALS

Study Purpose:

This is an open label, off label study, to provide interested ALS patients with Ciprofloxacin/Celecoxib fixed dose combination, while assessing safety and tolerability, routine disease progression measures (ALSFRS-R and Vital Capacity) and change in serum pNFH.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not yet enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Jeremy Shefner, MD, PhD Barrow Neurological Institute
Jinsy Andrews, MD, MSc Columbia University

Clinicaltrials.gov ID (11 digit #):

NCT04090684

Neals Affiliated?

No

Coordinating Center Contact Information


Avital Pushett, MSc, CRA / .(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

Patients will be prescribed a fixed dose combination of Ciprofloxacin and Celecoxib to be taken twice daily, and will be monitored for safety and tolerability. Additionally, routine progression measures will be assessed, as well as change in serum pNFH.

Study Sponsor:

NeuroSense Therapeutics Ltd.

Participant Duration:

Estimated Enrollment:

30

Estimated Study Start Date:

09/30/2019

Estimated Study Completion Date:

03/31/2021

Posting Last Modified Date:

09/17/2019

Date Study Added to alsconsortium.org:

09/17/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    75

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Able to comprehend and willing to sign an Informed Consent Form (ICF)
    Males or females between the ages of 18 and 75 years of age, inclusive
    Diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria) less than 5 years prior to baseline
    Patients may be on Riluzole and/or Edaravone; 30 days of stable use is required to make safety assessments more reliable
    Upright Forced Vital Capacity (FVC) ≥ 50% of predicted for age, height and sex at screening
    Patient is able to swallow tablets/ capsules
    A caregiver (if one is needed)
    Female patients must be post-menopausal (≥ 1 year) OR sterilized, OR if of childbearing potential (i.e., females who have had their first period unless they are anatomically and physiologically incapable to become pregnant), must have a negative pregnancy test, and agree to use contraceptive drugs or devices (e.g., diaphragm plus spermicide, or oral contraceptives) for the duration of the study and 10 weeks after the last treatment dose AND require male partners to use a condom during sexual intercourse

    Exclusion Criteria:

    A past history of adverse reaction/hypersensitivity to either NSAIDs, celecoxib or fluoroquinolones, ciprofloxacin
    Any known clinically significant abnormal gastric mucosal initial gastroscopic of an erosion, ulcer or tumor or/and GI disorder
    Known history of impaired renal function.
    Known or suspected congestive heart and/or coronary heart disease, previous history of myocardial infarction, uncontrolled arterial hypertension, or rhythm abnormalities requiring permanent treatment
    Known history of QT/QTc prolongation, Torsade de pointes (TdP) (e.g. heart failure, hypokalemia, family history of Long QT syndrome) and the use of concomitant medications that prolong the QT/QTc interval.
    Known or suspected diagnosis or family history of epilepsy

    Presence at screening of any medically significant cardiac, pulmonary, musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety data, including, but not limited to:

    • Mean systolic blood pressure >180 mm Hg; mean diastolic blood pressure >100 mm Hg (measurements taken after few min rest) that persist on 3 successive measurements taken at least 2 minutes apart
    • NYHA Class II or greater congestive heart failure
    • Chronic obstructive pulmonary disease or asthma requiring daily use of bronchodilator medications
    • Poorly controlled or brittle diabetes mellitus
    • Cognitive impairment, related to ALS or otherwise, sufficient to impair the patient's ability to understand and/or comply with study procedures and provide informed consent

    Female who is pregnant or breastfeeding or with intention of becoming pregnant during the course of the study
    Any impairment or social circumstance that, in the opinion of the Investigator, would render the patient not suitable to participate in the study
    Patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study
    Patient is participating in (or plans to participate in) any other investigational drug trial, or plans to be exposed to any other investigational agent, device and/or procedure, from 30 days prior to Screening through study completion

  • Site Contact Information

    Barrow Neurological Institute
    Phoenix , Arizona 85013
    United States

    Columbia University
    New York, New York 10032
    United States

Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS: Open-Label Extension for Patients Completing Study 3119002 (REFALS-ES)

Study Purpose:

This study provides an opportunity for subjects in the REFALS (3119002; NCT03505021) study to continue treatment with oral levosimendan. The study will also provide more information about long-term safety and effectiveness of oral levosimendan in patients with ALS.

This is an open-label study, so that all eligible subjects that complete the double-blind REFALS study (48-weeks of treatment) will have the opportunity to receive oral levosimendan treatment. The primary objective, in addition to continuing treatment for subjects enrolled in the REFALS study, is to evaluate long-term safety of oral levosimendan in ALS patients. Another important objective is to explore long-term effectiveness of oral levosimendan in the treatment of patients with ALS.

This study is open only to patients taking part in the REFALS study.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase III

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03948178

Neals Affiliated?

No

Coordinating Center Contact Information

Massachusetts General Hospital
Merit E Cudkowicz, MD / .(JavaScript must be enabled to view this email address) / 617-726-2383
.(JavaScript must be enabled to view this email address) Boston, Massachusetts United States

Full Study Summary:

Study Sponsor:

Orion Corporation, Orion Pharma

Participant Duration:

Estimated Enrollment:

450

Estimated Study Start Date:

06/26/2019

Estimated Study Completion Date:

07/31/2022

Posting Last Modified Date:

09/16/2019

Date Study Added to alsconsortium.org:

09/16/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    N/A

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Written or verbal informed consent (IC) for participation in the study
    Subjects who completed 48 weeks of treatment according to the REFALS study protocol
    Able to swallow study treatment capsules at the time of completing 48 weeks dosing in the REFALS study

    Exclusion Criteria:

    Development (or significant worsening from baseline of the REFALS study) of serious cardiovascular disease (e.g.: myocardial infarction, heart failure, arrhythmia, stroke, or second or third degree atrioventricular (AV) block)
    Pulse/heart rate repeatedly >100 bpm after 5-minute rest at baseline. If the pulse/heart rate is >100 bpm in the first recording, then a second recording must be done after another 5 min rest to confirm pulse/heart rate >100 bpm
    Systolic blood pressure (SBP) <90 mmHg
    Severe renal impairment (creatinine clearance < 30ml/min or creatine >170 µmol/l at 48 week visit of the REFALS study, or on dialysis
    Severe hepatic impairment at the discretion of the investigator
    Women of reproductive age without a negative pregnancy test and without a commitment to using a highly effective method of contraception (e.g.: oral hormonal contraceptive associated with inhibition of ovulation, intrauterine devices and long acting progestin agent), if sexually active during the study, and for 1 month after the last dose of the study treatment. Women who are postmenopausal (1 year since last menstrual cycle), surgically sterilised or who have undergone a hysterectomy are considered not to be reproductive and can be included
    Subject judged to be actively suicidal by the investigator
    Any other clinical significant cardiovascular, gastrointestinal, hepatic, renal, neurological or psychiatric disorder or any other major concurrent illness that in the opinion of the investigator could interfere with the interpretation of the study results or constitute a health risk for the subject if he/she took part in the study

  • Site Contact Information

    Hospital Universitari de Bellvitge
    Barcelona, 08207
    Spain

Open Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 Trial

Study Purpose:

A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS)who have completed the ORARIALS-01 trial.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase III

Study Chair(s)/Principal Investigator(s):

Michael Benatar, MD, PhD University of Miami

Clinicaltrials.gov ID (11 digit #):

NCT03836716

Neals Affiliated?

No

Coordinating Center Contact Information


Requests at Orphazyme A/S / .(JavaScript must be enabled to view this email address) / +45 39 17 82 72
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Orphazyme

Participant Duration:

Estimated Enrollment:

231

Estimated Study Start Date:

08/15/2019

Estimated Study Completion Date:

08/01/2022

Posting Last Modified Date:

08/29/2019

Date Study Added to alsconsortium.org:

08/29/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    • Subject is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures, or in the circumstance that the subject is incompetent, informed consent/assent is provided in accordance with local regulation and/or procedures
    • Subject has completed the ORARIALS-01 trial (i.e., met one of the surrogate survival endpoints of tracheostomy or PAV or has completed the 76 weeks randomized treatment period)
    • On treatment with IMP defined as last dose within 2 weeks of the last visit of the blinded ORARIALS-01 trial.
    • Able and willing to travel to the site for the Baseline visit and in the investigator's opinion is expected to be able to attend the clinic for the visit at Week 4.

    Exclusion Criteria:

    • Known or suspected allergy or intolerance to the IMP (Arimoclomol or constituents)
    • Exposure to any other investigational treatment, advanced therapy medicinal product or use of any other prohibited concomitant medications
    • Significant protocol deviation in the blinded ORARIALS-01 trial based on the Investigator's judgement
    • Women who are lactating or pregnant, or men or women unwilling to use a highly effective method of birth control if not surgically sterile (defined as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men) for female participants until 4 weeks after last dose and for male participants until 3 months after last dose. Pre-menopausal women must have a negative pregnancy test prior to dosing with trial medication.
    • Any of the following medically significant conditions:
      Clinically significant renal or hepatic disease, as indicated by clinical laboratory assessment (results ≥ 3 times the upper limit of normal [ULN] for aspartate aminotransferase, alanine aminotransferase, and lactate dehydrogenase, bilirubin ≥ 2 times the ULN, or creatinine ≥ 1.5 times the ULN).
      Any new condition or worsening of existing condition which, in the opinion of the investigator, would make the subject unsuitable for enrolment or could interfere with the subject participating in or completing the trial
    • Any serious adverse event or moderate/severe adverse event from the ORARIALS-01 trial which is ongoing at the time of transitioning to ORARIALS-02 and assessed as probably related to IMP

  • Site Contact Information

    Barrow Neurological Institute
    Nicole Turcotte / .(JavaScript must be enabled to view this email address) / 602-406-4775
    Phoenix, Arizona 85013
    United States

    HonorHealth Neurology
    Mackenzie Steinbach / .(JavaScript must be enabled to view this email address) / 480-882-4916
    Phoenix , Arizona 85018
    United States

    UC Irvine Health ALS and Neuromuscular Center
    Ivonne Turner / .(JavaScript must be enabled to view this email address) / 714-456-7760
    Orange, California 92868
    United States

    University of Miami
    Maria Elena Paredes / .(JavaScript must be enabled to view this email address) / 305-243-7336
    Miami, Florida 33136
    United States

    University of Kansas Medical Center
    Alyssa Lackey / .(JavaScript must be enabled to view this email address) / 913-945-9942
    Kansas City, Kansas 66160
    United States

    Hospital for Special Surgery
    William Mark Richardson / .(JavaScript must be enabled to view this email address) / 646-797-8657
    New York, New York 10021
    United States

    Providence Brain & Spine Institute
    Arlena Cummings / .(JavaScript must be enabled to view this email address) / 503-962-1171
    Portland , Oregon 97213
    United States

    University of Pensylvania
    Kelly Almasy / .(JavaScript must be enabled to view this email address) / 215-829-5041
    Philadelphia, Pennsylvania 19107
    United States

    University of Texas Southwestern Medical Center
    Amruta Joshi / .(JavaScript must be enabled to view this email address) / 213-648-9380
    Dallas, Texas 75390
    United States

    University of Virginia Health System
    Mary Wagoner / .(JavaScript must be enabled to view this email address) / 434-924-5541
    Charlottesville, Virginia 22908
    United States

    Catholic University Leuven
    Leuven,
    Belgium

    London Health Sciences Centre
    Christine Piechowicz / .(JavaScript must be enabled to view this email address) / 519-685-8500 ext 34858
    London, Ontario
    Canada

    Sunnybrook Health Sciences Centre
    Liane Phung / .(JavaScript must be enabled to view this email address) / 16-480-6100 ext 87561
    Toronto, Ontario
    Canada

    Montreal Neurological Institute and Hospital
    Natalie Saunders / .(JavaScript must be enabled to view this email address) / +15143981779
    Montréal, Quebec
    Canada

    Centre Hospitalier Regional Universitaire (CHRU) Montpellier
    Laura Labar / .(JavaScript must be enabled to view this email address) / +330467330281
    Montpellier,
    France

    Groupe Hospitalier Pitie-Salpetriere
    Sylvie Coudoin / .(JavaScript must be enabled to view this email address) / +330142165762
    Paris,
    France

    Charite - Universitaetsmedizin Berlin
    Birgit Koch / .(JavaScript must be enabled to view this email address) / +49 30 450 560028
    Berlin,
    Germany

    Medizinische Hochschule Hannover (MHH)
    Chantal Fischer / .(JavaScript must be enabled to view this email address) / +49 (0) 511 532-8333
    Hannover,
    Germany

    Universitaetsklinikum Ulm
    Johannes Dorst, MD / .(JavaScript must be enabled to view this email address) / +49- 731 177 5285
    Ulm,
    Germany

    Instituti Clinica Scientifici Maugeri
    Riccardo Sideri / .(JavaScript must be enabled to view this email address) / +390250725260
    Milano ,
    Italy

    Azienda Ospedaliero Universitaria (AUO) di Torino
    Gio Fuda / .(JavaScript must be enabled to view this email address) / +390116335439
    Torino,
    Italy

    University Medical Center Utrecht
    Tommy Bunte / .(JavaScript must be enabled to view this email address) / 31887573110
    Utrecht,
    Netherlands

    Centrum Medyczne NeuroProtect
    Marta Biel / .(JavaScript must be enabled to view this email address) / +48 22 468 15 48
    Warsaw,
    Poland

    Citi Clinic
    Adam Ronert / .(JavaScript must be enabled to view this email address) / + 48607616559
    Warsaw ,
    Poland

    Hospital Universitario Vall d'Hebron
    Ana Canovas / .(JavaScript must be enabled to view this email address) / 0034 93274600 ext 2780
    Barcelona,
    Spain

    Hospital Carlos III - Hospital Universitario La Paz
    Saul Marin / .(JavaScript must be enabled to view this email address) / +34 686061101
    Madrid,
    Spain

    Umeå University Hospital
    Erica Stenberg / .(JavaScript must be enabled to view this email address) / +46 72548 74 10
    Umeå,
    Sweden

    Kantonsspital St.Gallen
    Christoph Neuwirth, MD / .(JavaScript must be enabled to view this email address) / +41 714943581
    Saint Gallen,
    Switzerland

    Leonard Wolfson Experimental Neurology Centre
    Anna Bellin / .(JavaScript must be enabled to view this email address) / 2034488014
    London,
    United Kingdom

Phase 2a Study of the Expansion and Infusion of Autologous T-Regulatory Cells in Amyotrophic Lateral Sclerosis

Study Purpose:

This study is a randomized, placebo-controlled, phase 2a trial to study the biological activity, safety, and tolerability of regulatory T Lymphocytes (Tregs) taken and expanded outside of the body and returned back to the same person whose Treg were removed, given back by IV (intravenously) and in combination with low-dose IL-2 in people with Amyotrophic Lateral Sclerosis (ALS).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Stanley H. Appel, MD The Methodist Hospital System
James D. Berry, MD Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):

NCT04055623

Neals Affiliated?

Yes

Coordinating Center Contact Information

Houston Methodist Hospital
Sharon L. Halton, MA,CCRC / .(JavaScript must be enabled to view this email address) / 713-441-3420
Patricia A Mendoza, BSN,CCRC / .(JavaScript must be enabled to view this email address) / 713-441-5855
Houston, Texas 77030 United States

Full Study Summary:

Based on data collected in a previous study with a small group of patients, evidence was found to show that interfering with the immune system using Treg cells slowed ALS disease progression. It is known that Treg cell numbers and function are reduced in patients with ALS and in some patients with lower Treg cells, they have a more marked rapid progression of their ALS. For this study, there are two sites (in Houston, TX and Boston, MA) in which Tregs will be taken from participants, increased or expanded outside the body, and then re-administered back to the participants from which the Tregs came.

This study has two parts:

  1. The first period is a 6-month phase 2a, 2-center, randomized, placebo-controlled clinical trial studying the biological activity, safety, and tolerability of the increased / expanded Tregs administered intravenously (IV) with subcutaneous low-dose Interleukin-2 (IL-2) in 12 adults with ALS. IL2 helps regulate the immune system's white blood cells.
  2. The second period is a 6-month open-label extension in which all participants will receive their own expanded Treg cells administered intravenously in combination with subcutaneous low-dose IL-2.

This study is studying whether the enhancement of Treg numbers and function will slow disease progression.

In the first study of Tregs, we completed a single-center, open-label phase I study of Tregs from people with ALS. Tregs were increased outside the body and returned back to the individual Treg owners in multiple doses every 2 to 4 weeks. This early study provided evidence in a small group of patients that treatment with autologous Tregs may be effective in slowing ALS progression.

Study Sponsor:

The Methodist Hospital System

Participant Duration:

Estimated Enrollment:

12

Estimated Study Start Date:

09/01/2019

Estimated Study Completion Date:

11/30/2023

Posting Last Modified Date:

10/23/2019

Date Study Added to alsconsortium.org:

08/16/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    65

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    • ALS meeting El Escorial criteria for possible, probable, lab-supported probable, or definite ALS.
    • At least 18 years old.
    • Provided informed consent and authorized use of protected health information (PHI) in accordance with national and local patient privacy regulations.
    • Capable of complying with all study procedures, including the study drug delivery procedure, in the Investigator's opinion.
    • On a stable regimen of riluzole for at least 30 days at the time of screening. If not on riluzole at the time of study entry, willing to refrain from initiation of the agent for the duration of the trial.
    • Patients on edaravone willing to refrain from taking edaravone on the same day as they will receive the Tregs infusion for the duration of the trial. If not on edaravone at the time of study entry, willing to refrain from initiation of the agent for the duration of the trial.
    • Medical record documentation of a decline in ALSFRS-R total score of at least two points in the 90 days prior to screening or at least four points over the 180 days prior to screening.
    • Forced vital capacity (FVC) ≥65% of predicted capacity for age, height, and gender at screening.
    • Patient able and willing to undergo leukapheresis.

    Exclusion Criteria:

    • Presence of any of the following clinical conditions that would interfere with the safe conduct of the study, as determined by the Investigator:
      Unstable neurological, cardiovascular, cerebrovascular, pulmonary, renal, hepatic, endocrine, or hematologic disease; active malignancy or infectious disease; or other medical illness.
      Unstable psychiatric illness defined as psychosis (hallucinations or delusions), unstable major depression or substance abuse within 180 days prior to screening.
    • Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 times the upper limit of normal (ULN) at screening.
    • Serum creatinine greater than 1.8 mg/dL or creatinine clearance less than 40 mL/min at screening.
    • History of or positive test result for human immunodeficiency virus (HIV), hepatitis C virus, or hepatitis B virus (i.e., positive for both hepatitis B surface antigen and hepatitis B core antibody) at screening.
    • Tracheostomy.
    • If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or unwilling to use effective contraception for the duration of the trial and for 90 days after treatment.
    • If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 90 days after treatment.
    • Enrollment in any other interventional study.
    • Treatment with another investigational drug, biological agent, or device within 30 days or 5 half-lives of screening, whichever is longer. Patient participation in an observational/non-interventional clinical study is to be discussed with the Medical Monitor.
    • Prior gene or cell therapy treatments for ALS.

  • Site Contact Information

    Massachusetts General Hospital
    Boston, Massachusetts 02114
    United States

    Houston Methodist Hospital
    Sharon Halton / .(JavaScript must be enabled to view this email address) / 713-441-3420
    Patricia Mendoza / .(JavaScript must be enabled to view this email address) / 713-441-5855
    Houston, Texas 77030
    United States

The E-health Application To Modify ORal Energy Intake and Measure Outcomes REmotely in ALS Clinical Trial (EAT MORE2)

Study Purpose:

This is phase IIa feasibility and tolerability study of a mobile health (mHealth) application designed to study the effects of remote dietary counseling on disease progression and quality of life. The study will consist of two phases: Part I will consist of building and beta-testing the ALS Nutrition app and Part II will consist of enrolling a larger cohort of users into the app.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Observational Study

Study Category:

Device

Study Status:

Not yet enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Anne-Marie Alexandra Wills, MD, Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):

NCT04051333

Neals Affiliated?

Yes

Coordinating Center Contact Information

Massachusetts General Hospital
Mansi Sharma / .(JavaScript must be enabled to view this email address) / 617-643-2400
.(JavaScript must be enabled to view this email address) Boston, Massachusetts United States

Full Study Summary:

The investigators' prior work has shown that nutrition is an important modifiable prognostic factor for ALS disease progression and survival. In the recently completed E-Health Application To Measure Outcomes Remotely (EAT MORE) clinical trial (sponsored by the ALS Association), the investigators found that nutritional counseling supported by a mobile health (mHealth) app was associated with 0.5 points/month slower ALSFRS-R progression (p=0.17) and improved quality of life (p=0.09).

The investigators are now designing an ALS-specific app that can be used by everyone with ALS, including those who do not live near ALS Centers. The app would help to address gaps in the delivery of ALS care by providing nutritional counseling, helping patients to manage and track their disease symptoms, and communicating with their providers. After the initial design has been tested by a group of NEALS patients and their caregivers, the study will advertise the app widely to recruit a larger group of ALS patients to measure nutrition and outcomes.

Study Sponsor:

Massachusetts General Hospital

Participant Duration:

Estimated Enrollment:

200

Estimated Study Start Date:

08/31/2019

Estimated Study Completion Date:

12/31/2021

Posting Last Modified Date:

08/12/2019

Date Study Added to alsconsortium.org:

08/12/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    1. Participants with a diagnosis of ALS (self-reported)
    2. Male or female subjects aged 18 years or older.
    3. Participants must be capable of providing informed consent and complying with trial procedures.
    4. Participants must have access to an iOS or Android device to allow the to download the apps.

    Exclusion criteria:

    1. Use of a feeding tube (G-tube or J-tube)
    2. BMI>30 kg/m2 regardless of weight loss history
    3. A history of cardiovascular disease (stroke, myocardial infarction, peripheral vascular disease)
    4. A history of diabetes (self-reported)

  • Site Contact Information

    Massachusetts General Hospital
    Mansi Sharma / .(JavaScript must be enabled to view this email address) / 617-643-2400
    Boston, Massachusetts
    United States

A Multicenter, Randomized, Placebo-Controlled, Double-Blind, Phase 1b Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL747 in Subjects With Amyotrophic Lateral Sclerosis

Study Purpose:

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple oral doses of DNL747 in subjects with Amyotrophic Lateral Sclerosis when administered for 29 days in a cross-over design

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03757351

Neals Affiliated?

No

Coordinating Center Contact Information


Melissa Leedom / .(JavaScript must be enabled to view this email address) / 913-624-4970
.(JavaScript must be enabled to view this email address)

Full Study Summary:

This is a Phase 1b randomized, placebo-controlled, double-blind, crossover study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL747 in subjects with Amyotrophic Lateral Sclerosis (ALS)

Study Sponsor:

Denali Therapeutics Inc.

Participant Duration:

Estimated Enrollment:

16

Estimated Study Start Date:

12/28/2018

Estimated Study Completion Date:

08/31/2019

Posting Last Modified Date:

09/12/2019

Date Study Added to alsconsortium.org:

07/30/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    21

    Maximum Age:

    80

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    <36 months

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    • Women of non-childbearing potential and men, aged 21−80 years
    • Willingness and ability to complete all aspects of the study; participant should be capable of completing assessments either alone or with help of a caregiver
    • Diagnosis of laboratory-supported probable, probable, or definite (sporadic or familial) ALS according to the El Escorial World Federation of Neurology revised research diagnostic criteria
    • Less than 3 years since symptom onset
    • Forced vital capacity (FVC) >50% predicted measured within 30 days of screening
    • If subject is taking approved ALS treatments (riluzole and/or edaravone), doses must be stable for ≥2 months prior to screening and subject is expected to stay on a stable regimen throughout the study

    Exclusion Criteria:

    • History of a clinically significant non-ALS neurologic disorder (other than frontal temporal lobe dementia), including, but not limited to, muscular dystrophy, spinal stenosis, peripheral neuropathy, inherited neuropathies, AD, Parkinson's disease, Lewy body dementia, vascular dementia, Huntington's disease, epilepsy, stroke, multiple sclerosis, brain tumor, or brain infection or abscess
    • Unstable or poorly controlled comorbid disease process of any organ system currently requiring active treatment or likely to require treatment adjustment during the study

  • Site Contact Information

    Bioclinica
    Matthew Bubalo / .(JavaScript must be enabled to view this email address) / Orlando, Florida 32806
    United States

    PRA Health Sciences
    Karena McDonald, RN / .(JavaScript must be enabled to view this email address) / 801-904-4640
    Salt Lake City, Utah 84124
    United States

    CHDR
    Leiden, South Holland
    Netherlands

A Pilot Study to Assess Transmembrane Electromyography (TM-EMG) for the Assessment of Neuromuscular Function in the Oropharynx

Study Purpose:

This is a pilot study to examine the diagnostic utility of a novel transmembrane surface sensor, and compare signals obtained with the transmembrane sensor to conventional needle EMG signals from healthy volunteers to those with documented neurologic pharyngeal muscle dysfunction (ALS and muscular dystrophy) and to those with severe OSA.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Healthy Volunteer, Healthy Volunteer with a Family History of ALS, Obstructive Sleep Apnea, Muscular Dystrophies

Study Type:

Interventional Trial

Study Category:

Device

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Perry Mansfield, MD Perry Mansfield MD Inc.

Clinicaltrials.gov ID (11 digit #):

NCT03986671

Neals Affiliated?

No

Coordinating Center Contact Information

SENTA Clinic
Derrick Cordice / .(JavaScript must be enabled to view this email address) / 619-810-1239
.(JavaScript must be enabled to view this email address) San Diego, California 92108 United States

Full Study Summary:

Transmembrane electromyography (TM-EMG) may be a feasible and valid non-invasive EMG technique for detecting neuromuscular (NM) impairment. This study will assess whether, in healthy volunteers and participants with known obstructive sleep apnea (OSA) and other NM diseases involving the oropharynx, the same characteristic motor unit potentials obtained on conventional needle EMG (NEMG) can be obtained using a TM-EMG sensor. The purpose of this study is to demonstrate whether the TM-EMG sensor can provide the same diagnostic accuracy as the concentric needle electrode for the diagnosis of NM diseases. Having demonstrated diagnostic similarity of TM-EMG to NEMG, the secondary aim of this study is to confirm that NM disturbance of oropharyngeal striated muscles in participants with OSA can be elicited with the TM-EMG sensor.

Study Sponsor:

Powell Mansfield Inc.

Participant Duration:

Estimated Enrollment:

23

Estimated Study Start Date:

04/30/2019

Estimated Study Completion Date:

04/30/2020

Posting Last Modified Date:

07/25/2019

Date Study Added to alsconsortium.org:

07/25/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    70

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?


    Inclusion Criteria:

    • Age: 18-70
    • Must be able to pause use of anticoagulation, NSAID and multi-vitamins for appropriate period prior to study test.
    • Must be willing to stop any type of smoking or vaping 10 days prior to testing

    A cohort of participants with documented neurological disorders involving upper airway striated muscles including ALS and muscular dystrophy with the presence of bulbar symptoms.

    A cohort of participants diagnosed with moderate to severe OSA proven by an in-lab PSG, including the following criteria:

    • AHI > 25
    • Nadir SaO2 < 85%
    • not currently using CPAP

    A cohort of healthy participants that meet the following criteria:

    • Normal craniofacial anatomy
    • BMI < 30

    Exclusion Criteria:

    • Allergy to topical anesthetic
    • 4 or more alcoholic drinks on the same occasion on 5 or more days in the past month
    • Prior cancer, or radiation to the head or neck
    • Craniofacial anatomical disorders
    • Presence of any underlying medical, surgical or psychiatric disorder that would preclude participation as determined by the principal investigator

  • Site Contact Information

    SENTA Clinic
    San Diego, California 92108
    United States

Real World Testing of a Brain-Computer Interface to Operate a Commercial Augmentative and Alternative Communication System

Study Purpose:

The goal of this project is to test a new AAC-BCI device comparing gel and dry electrode headgear used for communication while providing clinical care. Innovative resources will be employed to support the standard of care without considering limitations based on service billing codes. Clinical services will include AAC assessment, AAC-BCI device and treatment to individuals with minimal movement due to amyotrophic lateral sclerosis (ALS), brain stem strokes, severe cerebral palsy, traumatic brain injury (TBI) and their family support person. This is a descriptive study designed to measure and monitor the communication performance of individuals using the AAC-BCI, any other AAC strategies, their user satisfaction and perceptions of communication effectiveness, and the satisfaction of the family support persons.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Brain Stem Stroke, Cerebral Palsy, Traumatic Brain Injury, Speech Disorders

Study Type:

Observational Study

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Katya Hill, PhD University of Pittsburgh

Clinicaltrials.gov ID (11 digit #):

NCT04026581

Neals Affiliated?

No

Coordinating Center Contact Information

University of Pittsburgh
Katya Hill, PhD / .(JavaScript must be enabled to view this email address) / 412-523-6424
.(JavaScript must be enabled to view this email address) Pittsburgh, Pennsylvania 15260 United States

Full Study Summary:

This clinical trial follows a descriptive study design collecting data to measure and monitor variables related to the standard of care in providing speech language pathology augmentative and alternative communication clinical (AAC) assessment and treatment. The study tracks participants through the AAC, speech generating device trial and AAC-BCI trial processes. In addition this study measures and monitors the communication performance of individuals using the AAC-BCI and any other AAC strategies for treatment in the home. Data on communication performance, user satisfaction, and perceptions of communication effectiveness are gathered over monthly visits along with the satisfaction and perceptions of communication effectiveness by the family support persons.

Study Sponsor:

University of Pittsburgh

Participant Duration:

Estimated Enrollment:

20

Estimated Study Start Date:

06/05/2019

Estimated Study Completion Date:

08/31/2023

Posting Last Modified Date:

07/25/2019

Date Study Added to alsconsortium.org:

07/25/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    14

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?


    Inclusion Criteria:

    natural speech does not meet daily communication needs requiring using a speech generating device
    Has a diagnosis resulting in minimal movement interfering with direct selection to a keyboard or AAC display
    age 14 and above
    able to read a standard computer screen
    able to follow instructions
    English as their native language

    Exclusion Criteria:

    history of photosensitive epilepsy
    open sores on the scalp
    history of uncorrectable hearing loss
    unavailable to participate during the times scheduled for the study

  • Site Contact Information

    AAC Institute Clinic (ICAN Talk Clinic)
    Shannon Carney / .(JavaScript must be enabled to view this email address) / 412-489-5527
    Carnegie, Pennsylvania 15102
    United States

    University of Pittsburgh, AAC Performance and Testing Laboratory
    Katya Hill, PhD / .(JavaScript must be enabled to view this email address) / 412-523-6424
    Pittsburgh, Pennsylvania 15260
    United States

TRIAL READY (Clinical Trial Readiness)

Study Purpose:

This study, being conducted under the auspice of the CReATe Consortium, will enroll patients with ALS and related disorders as well as healthy controls, with the goal of facilitating clinical validation of leading biological-fluid based biomarker candidates that may aid therapy development for patients with ALS and related disorders.
 

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Primary Lateral Sclerosis (PLS), Healthy Volunteer

Study Type:

Observational Study

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Michael Benatar, MBChB, MS, DPhil University of Miami

Clinicaltrials.gov ID (11 digit #):

NCT03912987

Neals Affiliated?

No

Coordinating Center Contact Information

University of Miami
Anne Cooley / .(JavaScript must be enabled to view this email address) / 844-837-1031
.(JavaScript must be enabled to view this email address) Miami, Florida 33136 United States

Full Study Summary:

This multi-center study aims to clinically validate leading biological-fluid-based biomarker candidates as potential prognostic and pharmacodynamic biomarkers that have the potential to facilitate therapy development for patients with ALS and related disorders. Biomarker candidates that will be considered include: urinary p75 neurotrophin receptor extracellular domain (p75ECD), blood and cerebrospinal fluid (CSF) phosphorylated neurofilament heavy (pNfH), blood and CSF neurofilament light (NfL) and, in the population with a C9orf72 hexanucleotide repeat expansion, peripheral blood mononuclear cell (PBMC) and CSF levels of the dipeptide repeat protein poly(GP). In pursuit of these goals, the CReATe Consortium is already collecting longitudinal biological samples from patients with ALS and related disorders through the ongoing Phenotype-Genotype-Biomarker (PGB) study. TRIAL READY aims to identify additional patients with the C9orf72 hexanucleotide repeat expansion mutation (HREM), the most common genetic cause of ALS, who may be further followed through the PGB study. This study will also enroll and longitudinally evaluate a cohort of age- and gender-match healthy controls.

Study Sponsor:

University of Miami

Participant Duration:

Estimated Enrollment:

610

Estimated Study Start Date:

01/22/2019

Estimated Study Completion Date:

01/31/2022

Posting Last Modified Date:

05/21/2019

Date Study Added to alsconsortium.org:

05/21/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Study Population
    Patients with ALS or a related neurodegenerative disorder, including FTD, ALS-FTD, PLS and PMA. Individuals never diagnosed with and not at particular risk of developing ALS or a related disorder.
    Criteria

    Inclusion Criteria:

    Member of at least one of the following categories:

    • Individuals with a clinical diagnosis of ALS or a related disorder, including FTD, ALS-FTD, PLS, and PMA.
    • Individuals never diagnosed with and not at particular risk of developing ALS or a related disorder.

    Able and willing to comply with relevant procedures.

    Exclusion Criteria:

    A condition or situation which, in the PI's opinion, could confound the study finding or may interfere significantly with the individual's participation and compliance with the study protocol. This includes (but is not limited to) neurological, psychological and/or medical conditions.

  • Site Contact Information

    University of Miami
    Kristina Reyes / .(JavaScript must be enabled to view this email address) / 305-243-4997
    Miami, Florida 33136
    United States

Speech Analysis in ALS Patients With and Without Cognitive Abnormalities: Evaluation of Sensitivity and Disease Progression

Study Purpose:

The purpose of this study is to find out if changes in speech can signal changes in the ability to think or remember. ALS patients with and without cognitive dysfunction will be followed for one year. Every three months, patients will undergo a series of cognitive and basic clinical outcomes tests. In addition, participants will take home a study-provided tablet on which they will complete weekly speech recording activities.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Observational Study

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Jeremy Shefner, MD Barrow Neurological Institute      
Shafeeq Ladha, MD Barrow Neurological Institute     

Clinicaltrials.gov ID (11 digit #):

NCT03868345

Neals Affiliated?

No

Coordinating Center Contact Information

Barrow Neurological Institute
Kerisa Shelton, PhD / .(JavaScript must be enabled to view this email address) / 602-406-6598
.(JavaScript must be enabled to view this email address) Phoenix, Arizona 85013 United States

Full Study Summary:

Cognitive dysfunction is increasingly recognized as a core feature of amyotrophic lateral sclerosis (ALS). With appropriate testing, up to 50% of ALS patients will show evidence of frontotemporal dysfunction. Approximately 15% of patients meet formal criteria for frontotemporal dementia (FTD). Certain genetic forms of ALS (e.g., mutations in C9orf72) have even higher incidences of FTD. The presence of cognitive abnormalities is an adverse risk factor for survival, and its presence influences the ability of patients to cooperate in clinical trials. However, screening for frontotemporal abnormalities is frequently not performed in ALS clinics, and tools for diagnosing cognitive dysfunction are either time consuming or insensitive. Additionally, the frequently co-existing dysarthria complicates the assessment and may mask more subtle cognitive deficits. Once identified, ways of following progressive decline are also lacking. In an ongoing study, it has been shown that a sophisticated suite of speech and language analytics, developed by two of the investigators, can identify abnormalities in cognitively normal ALS patients without speech symptoms, and predict important functional changes outside of the speech domain. In this study, investigators will evaluate both speech and language in 50 patients with ALS both with and without symptoms of cognitive decline. This evaluation will be paired with two cognitive screening tools frequently used in ALS clinics, the ALS Cognitive Behavioral Screen (ALS-CBS) and the Montreal Cognitive Assessment (MoCA). The investigators will evaluate the extent to which speech and language deficits precede abnormalities as measured by the above tools and determine whether cognitive change can be accurately followed over 12 months using speech and language measures. It is hypothesized that speech and language measures will accurately and sensitively predict cognitive changes. If so, such measures may be very useful in future studies of potential therapeutic agents for ALS-FTD and other dementias.

Study Sponsor:

Barrow Neurological Institute

Participant Duration:

Estimated Enrollment:

50

Estimated Study Start Date:

02/18/2019

Estimated Study Completion Date:

12/31/2019

Posting Last Modified Date:

05/21/2019

Date Study Added to alsconsortium.org:

05/21/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    21

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    male or female, age 21 or older,
    diagnosed with definite, probable, or possible ALS according to the modified El Escorial Criteria,
    a score of 2 or greater on the speech question of the ALSFRS-R (i.e. speech is intelligible with occasional repetition),
    continuous internet access at home,
    willingness and medical ability to comply with scheduled visits and study procedures,
    ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations,
    geographic accessibility to study site,
    for the 25 participants in Group 1, NO noted symptoms of frontotemporal cognitive dysfunction, and
    for the 25 participants in Group 2, MUST have cognitive symptoms as noted either by themselves or a caregiver.

    Exclusion Criteria:

    unwilling or unable to comply with the requirements of this protocol, including the presence of any condition (physical, mental, or social) that is likely to affect the participant's ability to comply with the protocol, and
    any other reasons that, in the opinion of the PI, cause the candidate to be deemed unsuitable for entry into the study.

  • Site Contact Information

    Barrow Neurological Institute
    Jessie Duncan / .(JavaScript must be enabled to view this email address) / Phoenix, Arizona 85013
    United States

Impact of Nuedexta on Bulbar Physiology and Function in ALS

Study Purpose:

Nuedexta is FDA approved for the treatment of pseudobulbar affect in ALS patients and anecdotal reports of improvements in speech, salivation or swallowing have been reported. However, no prospective study has been conducted to comprehensively examine and determine the physiologic impact of Nuedexta on both speech and swallowing physiology in a large group of ALS individuals. These data are needed in order to provide evidence-based guidance to the management of bulbar dysfunction in ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase I ,Phase II

Study Chair(s)/Principal Investigator(s):

Lauren Tabor, PhD Phil Smith Neuroscience Institute at Holy Cross Hospital
Emily Plowman, PhD University of Florida

Clinicaltrials.gov ID (11 digit #):

NCT03883581

Neals Affiliated?

No

Coordinating Center Contact Information

University of Florida
Kelby Magennis, MPH / .(JavaScript must be enabled to view this email address) / 352-273-8632
.(JavaScript must be enabled to view this email address) Gainesville, Florida 32610 United States

Full Study Summary:

Although advances in the management of bulbar dysfunction in ALS have been disappointing, recent interest has surfaced regarding the therapeutic potential of a pharmaceutical agent, Nuedexta (dextromethorphan HBr and quinidine sulfate), for the treatment of bulbar symptomology in individuals with ALS. Although Nuedexta received approval from the Food and Drug Administration (FDA) to target symptoms of pseudobulbar affect (PBA) in ALS; anecdotal reports of improvements in speech, salivation or swallowing were reported from Neurologists treating ALS individuals who were administered Nuedexta. Subsequently, a Phase II clinical trial was conducted that reported improvements in speech, swallowing and salivation following 30-days of Nuedexta treatment. One serious limitation of this study, however, is the fact that the primary outcome employed was a perceptual patient-report scale (PRO) (Center for Neurological Study Bulbar Function Scale, CNS-BFS), with no objective physiologic outcomes to confirm actual change in bulbar physiology. The absence of any objective clinical physiologic outcomes is particularly important when examining effects of Nuedexta, given that it contains selective serotonin reuptake inhibitors (SSRIs), or serotonergic antidepressants, that can impact the regulation of emotional expression, feelings of wellbeing and modulation of depression (all known to impact the response an individual will provide on a PRO measure). Furthermore, findings based on PRO's must be validated with studies that utilize objective physiologic outcomes of speech and swallowing function. Great excitement exists regarding the potential impact of Nuedexta on bulbar function in ALS with many neurologists prescribing Nuedexta to treat these symptoms in ALS patients. To date, however; no data exists to examine and determine the physiologic impact of Nuedexta on speech or swallowing physiology. These data are needed in order to validate the initial patient-reported outcomes of the Phase II clinical trial and to provide evidence-based guidance to the management of bulbar dysfunction in ALS.

Study Sponsor:

University of Florida

Participant Duration:

Estimated Enrollment:

40

Estimated Study Start Date:

07/25/2019

Estimated Study Completion Date:

02/28/2021

Posting Last Modified Date:

09/17/2019

Date Study Added to alsconsortium.org:

05/21/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    90

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Diagnosis of probable-definite ALS (El-Escorial Criterion);
    ALSFRS-R Bulbar subscale score <10
    Bamboo oral reading speaking rate <140 words per minute
    No allergies to barium sulfate.

    Exclusion Criteria:

    Treatment for sialorrhea within the past 3 months that includes either Botox or radiation treatment
    Participation in another disease modifying study targeting bulbar or cough function
    Use of invasive mechanical ventilation/presence of tracheostomy
    Advanced frontotemporal dementia or significant cognitive dysfunction
    Nil per oral status for feeding (i.e., NPO, nothing by mouth)
    Previously prescribed Nuedexta. Additionally, if participants are taking Riluzole or other medications to control sialorrhea, they must be on a stable dose for at least 30 days prior to enrollment in the current study.

  • Site Contact Information

    Phil Smith Neuroscience Institute at Holy Cross Hospital
    Lauren Tabor, PhD / .(JavaScript must be enabled to view this email address) / 954-542-3429
    Fort Lauderdale, Florida 33308
    United States

    University of Florida
    Kelby Magennis, MPH / .(JavaScript must be enabled to view this email address) / 352-273-8632
    Gainesville, Florida 32610
    United States

Amyotrophic Lateral Sclerosis (ALS) Families Project

Study Purpose:

This program provides family members of individuals with familial ALS the opportunity to contribute to research focused on learning more about why motor neuron degeneration begins and how or why it progresses. This study provides genetic counseling and testing to help participants understand and manage their risk and determine if they want to learn their genetic status. This study will follow unaffected ALS gene mutation carriers on an annual basis to gather essential information that will ultimately help researchers develop novel therapies for the prevention and treatment of ALS.

Disease:

Have a first degree relative who had an ALS-spectrum diagnosis with a confirmed ALS-spectrum gene mutation; or already have had genetic testing and have tested positive for an ALS-spectrum gene mutation

Study Type:

Observational Study

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Matthew Harms, MD Columbia University

Clinicaltrials.gov ID (11 digit #):

NCT03865420

Neals Affiliated?

No

Coordinating Center Contact Information

Columbia University
Elizabeth Harrington, MS, CGC / .(JavaScript must be enabled to view this email address) / 347-852-5315
.(JavaScript must be enabled to view this email address) New York, NY 10032 United States

Full Study Summary:

Approximately 10% of people with amyotrophic lateral sclerosis (ALS), or Lou Gehrig's Disease, have a family history of ALS or a related condition called frontotemporal dementia (FTD). In most of these familial cases, and a significant number of "sporadic" patients with no family history, a mutation is present in one of a growing number of genes that have been associated with ALS and/or FTD.

The ALS Families Project will study unaffected carriers of ALS/FTD-associated gene mutations to investigate the first steps in the disease process that leads to motor neuron degeneration, with the goal of identifying early disease targets and points of intervention to slow or stop disease onset and progression.

Unaffected individuals who have either a family member with a known ALS/FTD-associated gene mutation or have a strong family history of ALS and FTD are invited to participate in the ALS Families Project. For those who enroll, research visits will occur every 6-12 months.

Study Sponsor:

Columbia University

Participant Duration:

Estimated Enrollment:

60

Estimated Study Start Date:

09/11/2018

Estimated Study Completion Date:

09/30/2022

Posting Last Modified Date:

05/20/2019

Date Study Added to alsconsortium.org:

05/20/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    105

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


    Inclusion Criteria:

    Men or women of any race or ethnicity aged 18 or older
    No symptoms of ALS or fronto-temporal dementia at enrollment
    Have a first degree relative who had an ALS-spectrum diagnosis with a confirmed ALS-spectrum gene mutation; or already have had genetic testing and have tested positive for an ALS-spectrum gene mutation.
    Willing to undergo genetic testing with option of whether or not to learn results
    Willing to travel to Columbia University Irving Medical Center (CUIMC) every 6-24 months for study procedures
    Capable of providing informed consent and following study procedures, or has a legally authorized representative who is able to consent for the subject.

    Exclusion Criteria:

    Known HIV
    Known hepatitis B
    Known hepatitis C

  • Site Contact Information

    Columbia University
    Elizabeth Harrington, MS, CGC / .(JavaScript must be enabled to view this email address) / 347-852-5315
    New York , New York 10032
    United States

A Phase 2, Pilot Open Label, Sequential Group, Investigator Blinded Study of Magnetic Resonance Spectroscopy (31P-MRS) to Assess the Effects of CNM-Au8 for the Bioenergetic Improvement of Impaired Neuronal Redox State in ALS

Study Purpose:

REPAIR-ALS is a single-center open label pilot, sequential group, investigator and patient blinded study to assess the CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients who have been diagnosed with Amyotrophic Lateral Sclerosis (ALS) within twelve (12) months of Screening. The primary endpoint is the ratio of the oxidized to reduced form of nicotinamide adenine dinucleotide (NAD+:NADH) measured non-invasively by 31phosphorous magnetic resonance spectroscopy (31P-MRS).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not yet enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Jeffery Elliott, MD UT Southwestern

Clinicaltrials.gov ID (11 digit #):

NCT03843710

Neals Affiliated?

No

Coordinating Center Contact Information


Glen Frick, MD / .(JavaScript must be enabled to view this email address) / 801-676-9695
Austin Rynders, RN / .(JavaScript must be enabled to view this email address) / 801-676-9695

Full Study Summary:

This is a single-center open label pilot, sequential group, investigator blinded study of the CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients who have been diagnosed with Amyotrophic Lateral Sclerosis within twelve months of Screening. The Sponsor will select a starting treatment dose of CNM-Au8 for the initial treatment. Investigators and patients will be blinded to each cohort's study dose. Upon completion of the first treatment cohort, the Sponsor will select a single dose or two different doses for the subsequent second cohort from a pre-specified dosing selection plan based on the evaluation of the 31P-Magnetic Resonance Spectroscopy (31P-MRS) changes versus baseline in the first cohort. Up to a total of two treatment cohorts may be studied (n=12 patients/cohort, total n=24 patients). All patients will receive daily oral treatment over twelve consecutive weeks during each cohort's Treatment Period.

There will be three study periods per treatment cohort:

A four-week screening period (Screening Period); A twelve-week treatment period (Treatment Period); A four-week follow-up period (End-of-Study Assessment).

The primary study outcome, CNS metabolic changes, will be assessed based upon each patient's Week 12 study visit versus the pre-treatment baseline. The primary endpoint is the brain metabolic effects of treatment with CNM-Au8 as assessed by an improvement of 31P-MRS assessment of Brain Tissue Cellular Redox Potential defined by the measured tissue ratio of NAD+:NADH concentrations following 12 weeks of once daily treatment.

Study Sponsor:

Clene Nanomedicine

Participant Duration:

Estimated Enrollment:

24

Estimated Study Start Date:

03/31/2019

Estimated Study Completion Date:

06/30/2020

Posting Last Modified Date:

05/20/2019

Date Study Added to alsconsortium.org:

05/20/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    35

    Maximum Age:

    75

    Min Vital Capacity (% predicted normal):

    60

    Time since Symptom Onset:

    <24 months

    Time since Diagnosis:

    <12 months

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Able to understand and give written informed consent.
    Male or female patients aged 35 years or greater (inclusive) and less than 75 years of age at the time of ALS diagnosis.
    Patients with a confirmed ALS diagnosis: "definite ALS" or "probable ALS" or "possible" diagnostic criteria per the revised El Escorial Criteria as determined by a neurologist subspecializing in ALS (e.g., the Principal Investigator by study site).
    Stable background therapy (e.g., stable dosing of riluzole within the prior 6-weeks) per Investigator discretion.
    At the time of Screening disease duration less than or equal to 24-months from symptom onset OR within 12-moths of a confirmed ALS diagnosis.
    Forced vital capacity (FVC) >/= 60% of predicted value as adjusted for gender, height, and age at the Screening Visit.
    Patients who are ambulatory (e.g., normal ambulation, early ambulation difficulties, or walks with assistance) on the ALSFRS-R scale.

    Exclusion Criteria:

    At Screening patients who utilize, or in the Investigator's judgment will be imminently dependent upon during the course of this study:

    Non-invasive ventilation
    Gastrostomy (e.g., use of percutaneous endoscopic gastrostomy tube)
    Use of wheel chair

    Patient who have previously undergone tracheostomy.
    Patient with a history of significant other major medical condition based on the Investigator's judgment.
    Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or study procedures.
    Patient with clinically significant abnormalities in hematology, blood chemistry, ECG, or physical examination not resolved by the Baseline visit which according to Investigator can interfere with study participation.
    Patient participating in any other investigational drug trial or using investigational drug (within 12 weeks prior to screening and thereafter)
    Females who are pregnant or nursing or who plan to get pregnant during the course of this clinical trial or within 6 months of the end of this trial.
    Positive screen for drugs of abuse or known alcohol abuse.
    Women of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control during the study or for 6 months following completion of study participation.
    Women with a positive pregnancy test, are lactating, or are planning to become pregnant during the study.
    Patients with implanted metal objects in their body that may be affected by an MRI procedure.
    Patients who are claustrophobic or otherwise unlikely to be able to complete the MRI scanning procedures.
    Patients with a history of gold allergy.

  • Site Contact Information

    UT Southwestern
    Jeffery Elliott, MD / .(JavaScript must be enabled to view this email address) / 214-648-5437
    Ben Greenberg, MD / .(JavaScript must be enabled to view this email address) / 214-648-5437
    Dallas, Texas 75390
    United States

Non-invasive Brain Stimulation for the Treatment of Depression Symptoms in ALS: A Pilot Study

Study Purpose:

This is an open-label clinical trial to determine the safety of rTMS and efficacy in improving depression symptoms, quality of life and cognition deficits among patients with Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative disorders.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Device

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03373981

Neals Affiliated?

No

Coordinating Center Contact Information

Hospital for Special Surgery
Mona Shahbazi / .(JavaScript must be enabled to view this email address) / shahbazim@hss.edu
Shara Holzberg / .(JavaScript must be enabled to view this email address) New York, New York 10021 United States

Full Study Summary:

he objective of this study is to evaluate the role of repetitive transcranial magnetic stimulation (rTMS) for symptom reduction of depression and cognitive loss among patients with Amyotrophic Lateral Sclerosis (ALS) and other Neurodegenerative Diseases. Behavioral symptoms in ALS have been under scrutiny since its earliest descriptions, nearly 30 years ago (1). The importance of these symptoms among subjects with ALS has been also been under steady scrutiny. Studies have shown that the rates of anxiety and depression are significantly higher among subjects with ALS than the general population (1-23). Anxiety symptoms are related to depression, quality of life, and satisfaction with life (4, 9, 11, 15, 20, 24-26). Depressive symptoms are closely related to the ALS disease process (2).

rTMS has been shown to be a promising tool in modulating mood, memory, and cognitive performance (27). Current approaches to the management of ALS involve addressing symptomatology associated with the disease process. Among patients with ALS and other similar Neurodegenerative disorders, it is therefore important to understand if rTMS as an intervention is capable of:

Symptomatic improvement in mood,
Causing a significant positive change in disease progression or
Helping improve quality of life.

Study Sponsor:

Hospital for Special Surgery, New York

Participant Duration:

Estimated Enrollment:

15

Estimated Study Start Date:

11/29/2017

Estimated Study Completion Date:

11/20/2019

Posting Last Modified Date:

05/15/2019

Date Study Added to alsconsortium.org:

05/15/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    99

    Time since Symptom Onset:

    Time since Diagnosis:

    <24 months

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Diagnosis of familial or sporadic ALS as well as other Neurodegenerative disorders. Verification of the diagnosis will be performed by the Principal Investigator. This diagnosis can include neuro-degenerative disorders of uncertain cause, including ALS, MND, Peripheral neuropathies, Parkinson's and other progressive motor system diseases.
    If subjects has ALS diagnosis, the date of dx should be ≤ 2 years
    Age 18 or older.
    Capable of providing informed consent.
    Minimal speech impairment.
    Ability to comply with study procedures.
    Ability to communicate clearly if the subject wants to withdraw from the procedure at any stage.
    MMSE ≥20

    Female subjects of child bearing potential must engage in abstinence for the duration of the study. If a participant becomes sexually active, she must agree to using the following birth control methods:

    • Hormonal (oral, implanted, injected, etc)
    • Intrauterine device in place for ≥ 3 months
    • Adequate barrier method in conjunction with spermicide
    • Other

    Absence of exclusion criteria.

    Exclusion Criteria

    Unable to provide informed consent
    Significant speech impairment
    Inability to comply with the procedures
    Subjects with ALS diagnosis ≥ 2 years
    Inability to communicate clearly if the subject wants to withdraw from the procedure at any stage
    Seizures or history of seizures
    Patients who have underwent brain surgery for any indication
    Patients with pacemakers, cochlear implants, brain stimulators, infusion pumps, intracardiac lines, metallic clips, other implanted electronic or ferroelectric metallic devices. Dental implants are permitted
    Inability to perform either TMS or NCS studies due to insufficient MEP or CMAP amplitude.
    Patients with uncontrolled hypertension
    Patients with neuro endocrine disorders

    Patients who are withdrawn from the following drugs within 6 months:

    • Barbiturates
    • Benzodiazepines
    • Meprobamate
    • Chloral hydrate

    Patients who have a recent history (within 24h) or chronic history of intake of:

    Cocaine.
    Phencyclidine Phosphate.
    Gamma-Hydroxy Butyrate.

    Amphetamines including N-methylamphetamine, 3,4-Methylenedioxymethamphetamine, 2,5-dimethoxy-4-methylamphetamine, Ephedrine, Cathinone.

    12. Substance abuse+ 13. Excessive use of alcohol # 14. MMSE ≤19 15. Female patients of child bearing potential not practicing contraception 16. Female patients who are pregnant

  • Site Contact Information

    Hospital for Special Surgery
    Mona Shahbazi / .(JavaScript must be enabled to view this email address) / New York, New York 10021
    United States

A Randomised, Double-blind, Single-centre Study on the Safety, Tolerability and Efficacy of Cannabis Based Medicine Extract (CannTrust CBD Oil) in Slowing the Disease Progression in Amyotrophic Lateral Sclerosis or Motor Neurone Disease Patients

Study Purpose:

This is a randomised, double-blind, placebo controlled study on a cannabis-based medicine extract (CannTrust CBD Oil), in patients with Amyotrophic Lateral Sclerosis or Motor Neurone Disease. Participants will be randomised in a 1:1 ratio to receive CannTrust CBD Oil or placebo (both in capsules). The treatment duration is 6 months with one-month safety follow up. Participants will be checked every month either face to face or via telephone and will be assessed to collect data for study objectives such as ALSFRS-R, Forced Vital Capacity, pain and spasticity score, and quality of life. Thirty (30) participants will be randomised.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase III

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03690791

Neals Affiliated?

No

Coordinating Center Contact Information

Gold Coast Hospital and Health Service
Arman Sabet, MD / .(JavaScript must be enabled to view this email address) / +61 1300 744 284
Berzenn Urbi, RN / .(JavaScript must be enabled to view this email address) / +61 1300 744 284
Gold Coast, Queensland Australia

Full Study Summary:

Study Sponsor:

Gold Coast Hospital and Health Service

Participant Duration:

Estimated Enrollment:

30

Estimated Study Start Date:

01/09/2019

Estimated Study Completion Date:

01/31/2021

Posting Last Modified Date:

05/15/2019

Date Study Added to alsconsortium.org:

05/15/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    25

    Maximum Age:

    75

    Min Vital Capacity (% predicted normal):

    70

    Time since Symptom Onset:

    <24 months

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Affected by ALS/MND, either of definite or probable according to the El Escorial revised criteria
    Can provide written informed consent
    Able and willing to comply with all study requirement
    Male or female, ages 25-75 years old
    Onset of first symptom within the last 2 years
    Forced Vital Capacity (FVC) of at least 70% on baseline

    Exclusion Criteria:

    Participants who are bedridden
    Have used or taken cannabis or cannabinoid-based medications within 30 days of study entry
    History of any psychiatric disorder other than depression associated with their underlying condition including immediate family history of schizophrenia
    Heavy consumption of alcohol or use of illicit drug
    Hypersensitivity to cannabinoids or any of the excipients
    Any of the following: eGFR <30 mL/min/1.73m2, ejection fraction <35%, or ASL and ALT >5 X ULN
    Unwillingness of a female participant of child bearing potential, or their partner, to use effective contraception during the study and 30 days thereafter
    Pregnant, lactating mother or female participant planning pregnancy during the course of the study and for 30 days thereafter
    Received any investigational drug or medical device within 30 days prior randomisation
    Any other significant disease or disorder which, in the opinion of the investigator, may either put the participant at risk because of participation in the study, or may influence the result of the study, or the participant's ability to participate in the study
    Inability to cooperate with the study procedures
    Unwilling to stop driving vehicle or operating dangerous machinery whilst on study drug.
    Close affiliation with the study team, e.g. close relative of the investigator

  • Site Contact Information

    Gold Coast Hospital and Health Service
    Arman Sabet, MD / .(JavaScript must be enabled to view this email address) / +61 1300 744 284
    Gold Coast, Queensland
    Australia

A Phase II Pilot Single-arm Safety and Tolerability Study of ILB in Patients With Motor Neurone Disease (MND)/ Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

This is a phase II study to determine the safety and tolerability of ILB , a type of low molecular weight dextran sulfate, in patients with Motor Neurone Disease (MND)/ Amyotrophic Lateral Sclerosis (ALS)

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Venkataramanan Srinivasan, MD, University of Birmingham

Clinicaltrials.gov ID (11 digit #):

NCT03705390

Neals Affiliated?

No

Coordinating Center Contact Information

University Hospitals Birmingham NHS Foundation Trust
Venkataramanan Srinivasan, MD / .(JavaScript must be enabled to view this email address) / 0121 371 6851
Claire Potter / .(JavaScript must be enabled to view this email address) / 0121 371 8492
Birmingham, West Midlands United Kingdom

Full Study Summary:

Amyotrophic Lateral Sclerosis (ALS) belongs to a wider group of disorders known as motor neuron diseases and mainly involves the nerve cells (neurons) in the body. Voluntary muscles produce movements like chewing, walking and talking. ALS is caused by gradual deterioration (degeneration) and death of these motor neurons. The disease is progressive, meaning the symptoms get worse over time and most people with ALS die from respiratory failure, usually within 3 to 5 years from when the symptoms first appear. Currently there is no cure for ALS and no effective treatment to halt or reverse the progression of the disease (National Institute of Neurological Disorders and Stroke, Fact Sheet).

The aim of this study is to explore the safety and acceptability of a type of low molecular weight dextran sulfate called ILB.

The investigators will invite 15 patients to take part from a single centre in the UK. Participants will be closely monitored for any side-effects; for changes in ALS symptoms and on quality of life during and after the study.

The trial period for patient participation is 24 weeks (6 months), ILB injections will be administered once weekly for 10 weeks.

Study Sponsor:

University of Birmingham

Participant Duration:

24 weeks

Estimated Enrollment:

15

Estimated Study Start Date:

03/29/2019

Estimated Study Completion Date:

03/31/2020

Posting Last Modified Date:

05/15/2019

Date Study Added to alsconsortium.org:

05/15/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


    Inclusion Criteria:

    • Patients ≥18 years and who have provided written informed consent to participate in the study
    • Prior to trial entry patients will have a definite diagnosis of ALS according to El Escorial Criteria. All patients will demonstrate either presence of Upper Motor Neuron (UMN) (increased tone, brisk reflexes) as well as Lower Motor Neuron (LMN) (weakness, wasting and fasciculation) signs in the bulbar region and at least two of the other spinal regions (cervical, thoracic or lumbosacral) or presence of UMN and LMN signs in all three spinal regions (cervical, thoracic or lumbosacral)
    • Electrophysiological tests (Electromyography (EMG) / Nerve Conduction Study (NCS)) that supports the diagnosis of Motor Neurone Disease (MND) and to exclude mimic disorders
    • Forced Vital Capacity (FVC) ≥50% of predicted value for gender, height and age at screening and a mean Sniff Nasal Inspiratory Pressure (SNIP) ≥50% of predicted value for age
    • Adequate haematological function (Hb≥10g/dl absolute neutrophil count ≥1.5x109/L and a platelet count ≥60 x109/L
    • International Normalised Ratio (INR) ≤ 1.5, Activated Partial Thromboplastin Time (aPTT) 30 - 40 seconds, Prothrombin Time (PT) 11-13.5 seconds
    • Patient willing and able to comply with schedule visits, treatment plan and other study procedures.
    • Patients taking Riluzole must have discontinued treatment ≥28 days prior to study entry (and following consent to take part in the study)
    • Women Of Child Bearing Potential (WOCBP) who agree to use highly effective means of contraception (as defined in the Heads of Medicines Agencies_Clinical Trials Facilitation Group (HMA_CTFG) guideline (see Appendix 8) and in combination with a barrier contraception method (condom, diaphragm or cap) for the entirety of the study

    Exclusion Criteria:

    • Patients classified as either probable or possible ALS according to El Escorial Criteria.
    • Subjects in whom other causes of neuromuscular weakness have not been excluded
    • Assisted ventilation of any type within 3 months before the screening visit or at screening
    • Patients requiring Radiologically Inserted Gastrostomy (RIG) or Percutaneous Endoscopic Gastroscopy (PEG) feeding
    • Involvement in any other interventional study involving use of another IMP or biological product, within 3 months of screening
    • Any use of antioxidants, edaravone, tirasemtiv or CK-2127107 within 1 month before the screening visit
    • Any botulinum toxin use within 3 months before the screening visit.
    • Any form of stem cell or gene therapy for the treatment of amyotrophic lateral sclerosis (ALS)
    • Neuroimaging of brain and cervical spine with Magnetic Resonance imaging (MRI) indicating compressive myelopathy as an alternate diagnosis
    • Laboratory examinations including Acetylcholine receptor (AChR) antibodies and Muscle Specific Kinase (MuSK) antibodies to exclude Bulbar onset Myasthenia gravis from Bulbar onset Motor neuron disease as an alternate diagnosis and Antinuclear Antibodies (ANA), Anti-neutrophil cytoplasmic antibodies (ANCA), Extractable Nuclear Antigen (ENA) antibodies, Creatine Kinase (CK), electrophoresis and immunoglobulin indicating an alternate diagnosis for muscle disease like Myositis
    • Abnormal liver function defined as Aspartate Transaminase (AST) and/or Alanine Transaminase (ALT) >3 times upper limit of normal
    • Any head trauma, intracranial or spinal surgery within 3 months of trial entry
    • Patients who have had recurrent falls will be excluded to reduce the risk of intracerebral haemorrhage with this IMP
    • Current use of an anticoagulant e.g Warfarin, Aspirin, Clopidogrel, any novel anticoagulants (NOAC)s or low molecular weight subcutaneous heparin
    • Uncontrolled severe hypertension defined as systolic blood pressure (SBP) ≥ 220 mmHg or diastolic blood pressure (DBP) ≥120 mmHg
    • Current or previous history of heparin-induced thrombocytopenia
    • Active peptic ulcer disease
    • Known hypersensitivity to sulphur
    • Severe liver insufficiency 
    • Patients with evidence of major psychiatric illness, significant cognitive impairment or clinically evident dementia that may interfere with the patients' ability to comply with study procedures
    • Pulmonary illness (e.g asthma or Chronic Obstructive Pulmonary Disease (COPD)) requiring regular treatment
    • Patient judged to be actively suicidal by the investigator during 3 months before the screening visit
    • Subjects with a diagnosis of another neurodegenerative disease (e.g. Parkinson`s disease, Alzheimer's disease and Frontotemporal dementia)

  • Site Contact Information

    University Hospitals Birmingham NHS Foundation Trust
    .(JavaScript must be enabled to view this email address) / Birmingham , West Midlands B15 2TH
    United Kingdom

A Phase 1, Double-Blind, Placebo-Controlled, Single-Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adult Participants With ALS

Study Purpose:

The primary objective of this study is to evaluate the safety, tolerability of single-ascending doses of BIIB100 in adults with amyotrophic lateral sclerosis (ALS). The secondary objective of the study is to characterize the pharmacokinetic profile of BIIB100.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03945279

Neals Affiliated?

No

Coordinating Center Contact Information


US Biogen Clinical Trial Center / .(JavaScript must be enabled to view this email address) / 866-633-4636
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Biogen

Participant Duration:

Estimated Enrollment:

40

Estimated Study Start Date:

06/19/2019

Estimated Study Completion Date:

12/31/2020

Posting Last Modified Date:

09/16/2019

Date Study Added to alsconsortium.org:

05/22/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    65

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Key Inclusion Criteria:

    Must meet the laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria.
    Participants taking concomitant riluzole at study entry must be on a stable dose for greater than or equals to (>=) 30 days prior to the first dose of study treatment (Day 1). Participants taking concomitant riluzole must be willing to continue with the same dose regimen throughout the study, unless the Investigator determines that riluzole should be discontinued for medical reasons, in which case it may not be restarted during the study.
    Participants taking concomitant edaravone at study entry must be on a stable dose for >= 60 days prior to the first dose of study treatment (Day 1).
    Adequate respiratory function as indicated by slow vital capacity (SVC) >= 65% of predicted value as adjusted for sex, age, and height (from the sitting position).

    Key Exclusion Criteria:

    Ongoing medical condition (e.g., wasting or cachexia, severe anemia) that would, in the opinion of the Investigator, interfere with the conduct or assessments of the study.
    Significant cognitive impairment or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression less than or equals to (<=) 90 days of Screening, which in the opinion of the Investigator would interfere with the study procedures.
    Treatment with drugs that are transported by Breast Cancer Resistance Protein (BCRP) and P-glycoprotein (P-gp) including, but not limited to, rosuvastatin, sulfasalazine, dabigatran, digoxin and fexofenadine.
    Current enrollment or plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy for investigational use is administered within 30 days or 5 half-lives of the agent, whichever is longer, prior to the Baseline Visit (pre-dose on Day 1). Participation in a noninterventional study focused on ALS natural history may be allowed at the discretion of the Investigator and after consultation with the Sponsor.

  • Site Contact Information

    Barrow Neurological Institute
    .(JavaScript must be enabled to view this email address) / Phoenix, Arizona 85013
    United States

    University of California San Diego
    Rosemarie Previte / .(JavaScript must be enabled to view this email address) / 858-246-1319
    San Diego, California 92121
    United States

    Loma Linda University Medical Center
    Loma Linda, California 92354
    United States

    University of South Florida
    Tampa, Florida 33620
    United States

    Mayo Clinic Hospital
    Jacksonville, Florida 32224
    United States

    Johns Hopkins Hospital
    Baltimore, Maryland 21224
    United States

    Massachusetts General Hospital
    Boston, Massachusetts 02114
    United States

    Washington University
    Saint Louis, Missouri 63110
    United States

    New Orleans Center for Clinical Research
    865-305-9100 ext 302 or 303
    Knoxville, Tennessee 37920
    United States

A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis

Study Purpose:

To determine if long-term topiramate therapy is safe and slows disease progression in patients with ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Study Chair(s)/Principal Investigator(s):

Merit Cudkowicz, Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):

Neals Affiliated?

Yes

Coordinating Center Contact Information


.(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Participant Duration:

Estimated Enrollment:

296

Estimated Study Start Date:

06/01/1999

Estimated Study Completion Date:

08/31/2000

Posting Last Modified Date:

05/09/2019

Date Study Added to alsconsortium.org:

05/09/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    N/A

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


  • Site Contact Information
  • Study Results

    RESULTS:

    Patients treated with topiramate showed a faster decrease in arm strength (33.3%) during 12 months (0.0997 vs 0.0748 unit decline/month, p = 0.012). Topiramate did not significantly alter the decline in FVC and ALSFRS or affect survival. Topiramate was associated with an increased frequency of anorexia, depression, diarrhea, ecchymosis, nausea, kidney calculus, paresthesia, taste perversion, thinking abnormalities, weight loss, and abnormal blood clotting (pulmonary embolism and deep venous thrombosis).

    CONCLUSIONS:

    At the dose studied, topiramate did not have a beneficial effect for patients with ALS. High-dose topiramate treatment was associated with a faster rate of decline in muscle strength as measured by MVIC and with an increased risk for several adverse events in patients with ALS. Given the lack of efficacy and large number of adverse effects, further studies of topiramate at a dose of 800 mg or maximum tolerated dose up to 800 mg/day are not warranted.

    Full Study Summary

A Clinical Trial of Creatine in ALS

Study Purpose:

Mitochondrial dysfunction occurs early in the course of ALS, and the mitochondria may be an important site for therapeutic intervention. Creatine stabilizes the mitochondrial transition pore, and is important in mitochondrial ATP production. In a transgenic mouse model of ALS, administration of creatine prolongs survival and preserves motor function and motor neurons.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Study Chair(s)/Principal Investigator(s):

Jeremy Shefner (SUNY)

Clinicaltrials.gov ID (11 digit #):

Neals Affiliated?

Yes

Coordinating Center Contact Information


.(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Participant Duration:

Estimated Enrollment:

104

Estimated Study Start Date:

09/01/2000

Estimated Study Completion Date:

09/30/2002

Posting Last Modified Date:

05/09/2019

Date Study Added to alsconsortium.org:

05/09/2019
  • Eligibility Criteria

    Gender:

    Neals Affiliated, Diseases, Study Type, Study Category, Study Status, Phase

    Minimum Age:

    N/A

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


  • Site Contact Information
  • Study Results

    RESULTS:

    Creatine was tolerated well, but no benefit of creatine could be demonstrated in any outcome measure. CI analysis showed that the study, although powered to detect a 50% or greater change in rate of decline of muscle strength, actually made an effect size of greater than 23% unlikely. It was also demonstrated that motor unit number estimation was performed with acceptable reproducibility and tolerability, and may be a useful outcome measure in future clinical trials.

    CONCLUSION:

    Any beneficial effect of creatine at 5 g per day in ALS must be small. Other agents should be considered in future studies of therapeutic agents to address mitochondrial dysfunction in ALS. In addition, motor unit number estimation may be a useful outcome measure for future clinical trials in ALS.

    Full Study Summary

Tolerance of high-dose coenzyme Q10 in ALS

Study Purpose:

An open-label dose-escalation trial was performed to assess the safety and tolerability of high doses of coenzyme Q10 (CoQ10) in ALS. CoQ10, a cofactor in mitochondrial electron transfer, may improve the mitochondrial dysfunction in ALS. In this study, CoQ10 was safe and well tolerated in 31 subjects treated with doses as high as 3,000 mg/day for 8 months.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Study Chair(s)/Principal Investigator(s):

Merit Cudkowicz, Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):

Neals Affiliated?

Yes

Coordinating Center Contact Information


.(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Participant Duration:

Estimated Enrollment:

31

Estimated Study Start Date:

06/01/2001

Estimated Study Completion Date:

05/31/2003

Posting Last Modified Date:

05/09/2019

Date Study Added to alsconsortium.org:

05/09/2019

Trial of Celecoxib in Amyotrophic Lateral Sclerosis

Study Purpose:

To determine whether chronic treatment with celecoxib, a cyclooxygenase-2 inhibitor that has been shown to be beneficial in preclinical testing, is safe and effective in amyotrophic lateral sclerosis (ALS).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Study Chair(s)/Principal Investigator(s):

Merit Cudkowicz, Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):

Neals Affiliated?

Yes

Coordinating Center Contact Information


.(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Participant Duration:

12 months

Estimated Enrollment:

300

Estimated Study Start Date:

06/01/2001

Estimated Study Completion Date:

06/30/2003

Posting Last Modified Date:

05/09/2019

Date Study Added to alsconsortium.org:

05/09/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    N/A

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


  • Site Contact Information
  • Study Results

    Celecoxib did not slow the decline in muscle strength, vital capacity, motor unit number estimates, ALS Functional Rating Scale-Revised, or affect survival. Celecoxib was well tolerated and was not associated with an increased frequency of adverse events. Prostaglandin E(2) levels in cerebrospinal fluid were not elevated at baseline and did not decline with treatment.

    Full Study Results

Generation and characterization of cell lines for amyotrophic lateral sclerosis

Study Purpose:

This research is designed to augment the understanding of amyotrophic lateral sclerosis (ALS) by looking at skin cells from people with genetically confirmed familial ALS, as well as those from healthy individuals. Once the skin cells have been grown in the laboratory, they will be used to generate induced pluripotent stem cells, which will be made into nerve cells. Scientists will then be able to study these cells to learn more about ALS and potential treatments.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS

Study Type:

Observational Study

Study Category:

Neals Affiliated , Diseases , Study Type , Study Status , Phase , Gender

Study Status:

Not enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Nicholas Maragakis, MD, Johns Hopkins University
Jeffrey Rothstein, MD, PhD, Johns Hopkins University

Clinicaltrials.gov ID (11 digit #):

Neals Affiliated?

Yes

Coordinating Center Contact Information

Johns Hopkins University
.(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address) Baltimore, Maryland 21287 United States

Full Study Summary:

Study Sponsor:

NIH

Participant Duration:

Estimated Enrollment:

25

Estimated Study Start Date:

09/30/2009

Estimated Study Completion Date:

08/31/2011

Posting Last Modified Date:

05/09/2019

Date Study Added to alsconsortium.org:

05/09/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    N/A

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


  • Site Contact Information

    Johns Hopkins University
    Baltimore, Maryland 21287
    United States

  • Study Results

    Although we do not fully know if disease study of cells in Petri dishes can fully emulate the developmental progression that occurs in human adult neurodegenerative disease like ALS, new described technical ability to generate Induced Pluripotent Cells (iPS) from ALS patients provides an exceptional tool by which we can explore these issues. Many recent insights into the pathophysiology of ALS come from the study of familial forms of this disease. The ability to actually have human cell lines- representing the natural disease in the most relevant cell types- motor neurons and astrocytes- will provide unprecedented tools to 1) study cell- cell interactions responsible for disease pathophysiology and 2) provide critical tools for drug discovery and genetic pathway analysis. Eventually these ALS cell lines will also be useful to compare common and uncommon pathways between ALS and other neurodegenerative iPS models. But - iPS cell biology is exceptionally new and we do not yet have sufficient information about the reliability of the cells generated, their ability to truly reflect human cell biology, recapitulate the protein, genetic and functional characteristics of native motor neurons and astroglia. Before we can embark on extensive use of these cells for basic/translational research- it would be critical to generate a series of cell lines- all produced under identical conditions, from different fALS mutations, to determine how representative they are for cell type specificity and functional biology. The overall proposal will involve four principal investigators, working in tight collaboration, to generate and evaluate familial ALS (fALS) iPS cell lines. Project 1, led by Dr. Eggan will obtain the skin biopsies from FALS and control patients, generate the fibroblast and ultimately the initial iPS lines. We will employ the aid of iZumi, a biotech company to be a central site for uniform protocol iPS cell generation. iPS cell lines with neural/glial characteristics will be sent to the Project 2 Lab- Motor neuron biology, lead by Chris Henderson and to Project 3 lab, Astrocytes- lead by Jeffrey Rothstein. These two projects/labs will determine which of the fALS iPS cell lines have the appropriate characteristics of motor neurons and astroglia, through a series of sequential analyses. Only those cell lines that meet final criteria (as compared to human ES cell and prior work on human astroglia) will then go on for final genetic analysis in the Project 4 lab, lead by Tom Maniatis.

    Click here to read more.

A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB067 Administered to Adult Subjects With ALS and Confirmed Superoxide Dismutase 1 Mutation (VALOR)

Study Purpose:

The primary objective of Parts A and B of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 in adult with ALS. The primary objective of Part C of this study is to evaluate the clinical efficacy of BIIB067 administered to adult participants with ALS and confirmed superoxide dismutase 1 (SOD1) mutation.

The secondary objective of Parts A and B of this study is to evaluate the effects of BIIB067 on levels of SOD1 protein in the cerebrospinal fluid (CSF). The secondary objectives of Part C are to evaluate the safety, tolerability, and pharmacodynamic (PD) effects of BIIB067 administered to adult participants with ALS and confirmed SOD1 mutation.

Disease:

Amyotrophic Lateral Sclerosis (ALS)

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase III

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT02623699

Neals Affiliated?

No

Coordinating Center Contact Information


US Biogen Clinical Trial Center / .(JavaScript must be enabled to view this email address) / 866-633-4636
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

This is a 3-part study to examine the efficacy, safety, tolerability, PK and PD of BIIB067. Part A is the single ascending dose (SAD) component of the study, Part B is the multiple ascending dose (MAD) component of the study and Part C will be the fixed dose component of the study. Hence, the overall phase of development of the study is 1/2/3.

Study Sponsor:

Biogen

Participant Duration:

28 day screening window with a 32 week follow up

Estimated Enrollment:

144

Estimated Study Start Date:

01/20/2016

Estimated Study Completion Date:

05/29/2020

Posting Last Modified Date:

09/16/2019

Date Study Added to alsconsortium.org:

05/08/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Key Inclusion Criteria: Part A and B

    Weakness attributable to ALS and documented SOD1 mutation at Screening Visit 2.
    A forced vital capacity (FVC) ≥50% of predicted value as adjusted for sex, age, and height (from the sitting position). Participants with stable FVC <50% but ≥45%, whose FVC has not declined by more than 5% in the last 6 months may be considered for inclusion, at the discretion of the Investigator.
    If taking riluzole, participant must be on a stable dose for ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.
    Medically able to undergo the study procedures, and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.

    Key Exclusion Criteria: Part A and B

    History of or positive test result for human immunodeficiency virus.
    History of, or positive test result at Screening, for hepatitis C virus antibody.
    Current hepatitis B infection (defined as positive for hepatitis B surface antigen [HBsAg] and/or hepatitis B core antibody [HBcAb]). Participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive hepatitis B surface antibody immunoglobulin G, and positive HBcAb) or vaccination (defined as positive anti-HBs) are eligible to participate in the study.
    Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering ribonucleic acid, stem cell therapy, or gene therapy is allowed.
    Current enrollment in any other interventional study.
    Current or recent (within 1 month) use, or anticipated need, in the opinion of the Investigator, of copper (II) (diacetyl-bis (N4-methylthiosemicarbazone)) or pyrimethamine.
    Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period.

    Key Inclusion Criteria: Part C

    Weakness attributable to ALS and confirmed SOD1 mutation at Screening Visit.
    If taking riluzole, participant must be on a stable dose for ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.
    If taking edaravone, participant must have initiated edaravone ≥60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study. Edaravone may not be administered on dosing days of this study.
    Medically able to undergo the study procedures and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.

    Key Exclusion Criteria: Part C

    History of or positive test result for human immunodeficiency virus.
    History of, or positive test result at Screening, for hepatitis C virus antibody.
    Current hepatitis B infection (defined as positive for HBsAg and/or anti-HBc). participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive IgM anti-HBc, and positive anti-HBc) or vaccination (defined as positive anti-HBs) are eligible to participate in the study.
    Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs), biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering RNA, stem cell therapy, or gene therapy is allowed.
    Current enrollment in any other interventional study.
    Current or recent (within 1 month) use, or anticipated need, in the opinion of the Investigator, of copper (II) (diacetyl-bis(N4-methylthiosemicarbazone)) or pyrimethamine.
    Current or anticipated need, in the opinion of the Investigator, of a DPS during the study period.

  • Site Contact Information

    Barrow Neurological Institute
    Phoenix, Arizona 85013
    United States

    University of California San Diego Medical Center
    La Jolla, California 92093
    United States

    California Pacific Medical Center
    San Francisco, California 94115
    United States

    Bioclinica Research
    Orlando, Florida 32806
    United States

    University of Miami School of Medicine
    Miami, Florida 33136
    United States

    Emory University Hospital
    Atlanta, Georgia 30322
    United States

    Johns Hopkins University
    Baltimore, Maryland 21287
    United States

    Massachusetts General Hospital
    Boston, Massachusetts 02114
    United States

    Washington University School of Medicine
    Saint Louis, Missouri 63110
    United States

    Neurology Associates, P.C.
    Lincoln, Nebraska 68506
    United States

    New Orleans Center for Clinical Research
    Knoxville, Tennessee 37920
    United States

    UZ Leuven
    Leuven, 3000
    Belgium

    Sunnybrook Health Sciences Centre
    Toronto , Ontario M4N 3M5
    Canada

    Montreal Neurological Institute
    Montreal, Quebec H3A 2B4
    Canada

    Hospitalier Pitie-Salpetriere
    Paris, 75651
    France

    Universitaetsklinikum Ulm
    Ulm, Baden Wuerttemberg 89081
    Germany

    Research Site
    Suita-Shi, Osaka-Fu
    Japan

    Research Site
    Bunkyo-Ku, Tokyo-To
    Japan

    Research Site
    Shinjuku-ku, Tokyo-To
    Japan

    Research Site
    Kagoshima City,
    Japan

    Sheffield Institute for Translational Neuroscience
    Sheffield, South Yorkshire S10 2HQ
    United Kingdom

Perampanel Single Ascending Dose Transcranial Magnetic Stimulation Biomarker Study in Amyotrophic Lateral Sclerosis

Study Purpose:

To evaluate if transcranial magnetic stimulation can be used as a biomarker in Amyotrophic Lateral sclerosis (ALS).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Biomarkers/Imaging , transcranial magnetic stimulation

Study Status:

Enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Bjorn E. Oskarsson, MD, Mayo Clinic

Clinicaltrials.gov ID (11 digit #):

NCT03793868

Neals Affiliated?

No

Coordinating Center Contact Information

Mayo Clinic
Carla Palmucci / .(JavaScript must be enabled to view this email address) / 905-953-3182
.(JavaScript must be enabled to view this email address) Jacksonville, Florida 32224 United States

Full Study Summary:

To measure the effect on motor threshold (MT) by transcranial magnetic stimulation (TMS) after a single dose of perampanel at two dose levels.

Study Sponsor:

Mayo Clinic

Participant Duration:

Estimated Enrollment:

24

Estimated Study Start Date:

12/04/2018

Estimated Study Completion Date:

12/01/2019

Posting Last Modified Date:

01/31/2019

Date Study Added to alsconsortium.org:

01/31/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    70

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    A probable laboratory supported, probable or definitive ALS diagnosis by revised El Escorial criteria.
    Sporadic or familial ALS.
    Ages of 18-70.
    Agree to use reliable contraception
    Randomization will occur after a baseline MT has been established; any subject in whom a MT cannot be established will be excluded.
    Caregiver willing to report adverse behavioral events. -

    Exclusion Criteria:

    History of epilepsy.
    Significant laboratory abnormality (AST or alanine aminotransferase >3x upper limit of normal, or glomerular filtration rate <60)
    History of aggressive behavior.
    Subject unwilling to abstain from alcohol for 2 weeks after each dosing.
    History of drug abuse in the last 5 years
    Other severe medical conditions, including psychiatric conditions, which would cause an increased risk in the opinion of the investigator, including but not limited to renal failure and liver failure.
    Skull defect or other physical contraindication for TMS
    Pacemaker or implanted defibrillator
    Inability to take study capsule by mouth

    Females only: Subject is pregnant [as confirmed by a positive serum human chorionic gonadotropin (hCG) test for females of reproductive potential (FRP) only], subject is breastfeeding, or subject is of reproductive potential and does not agree to follow use of reliable contraception.

  • Site Contact Information

    Mayo Clinic
    Jany Paulett / .(JavaScript must be enabled to view this email address) / 904-953-3730
    Jacksonville, Florida 32224
    United States

BHV-0223 Expanded Access Protocol in Patients With Amyotrophic Lateral Sclerosis

Study Purpose:

This is an open label expanded access protocol for the treatment of up to approximately 250 adult patients with amyotrophic lateral sclerosis (ALS) who have difficulty swallowing oral riluzole tablets and may be able to derive benefit from treatment with an alternative oral formulation of riluzole.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Study Category:

Expanded Access - Treatment IND/Protocol

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03537807

Neals Affiliated?

No

Coordinating Center Contact Information


Early Access Care / .(JavaScript must be enabled to view this email address) / 1-888-315-5797 Ext 6
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

Riluzole is indicated in the U.S. for the treatment of patients with amyotrophic lateral sclerosis (ALS). The commercially available dosage form is a 50mg oral tablet. This expanded access protocol (EAP) is designed to provide access to a dissolving tablet formulation of riluzole designed for sublingual (SL) administration, in patients with ALS who, in the opinion and clinical judgement of the treating physician, would benefit from treatment with BHV-0223.

Study Sponsor:

Biohaven Pharmaceuticals, Inc.

Participant Duration:

Estimated Enrollment:

250

Estimated Study Start Date:

Estimated Study Completion Date:

Posting Last Modified Date:

12/19/2018

Date Study Added to alsconsortium.org:

12/19/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Patients with diagnosed ALS of any type or duration
    Current or previous treatment with oral riluzole tablets, or patients who have never taken riluzole oral tablets, or patients who have successfully completed a clinical trial with BHV-0223 and were not withdrawn prematurely due to adverse events
    Swallowing difficulties, or patient or caregiver report choking one or more times per week, or investigator deems appropriate to treat with sublingual BHV-0223 because (s)he deems the patient cannot be satisfactorily treated with Rilutek®
    Adequate hepatic function

    Exclusion Criteria:

    Patient with history of severe hypersensitivity reaction to riluzole oral tablets or BHV-0223
    Patient is known to have any other acute or chronic liver disease

  • Site Contact Information

Acute Intermittent Hypoxia and Breathing in Neuromuscular Disease

Study Purpose:

This project seeks to investigate the effects of a single acute intermittent hypoxia (AIH) session on respiratory and non-respiratory motor function and EMG (electromyography) activity on patients with ALS (amyotrophic lateral sclerosis) and healthy controls.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Healthy Volunteer, Healthy Volunteer with a Family History of ALS

Study Type:

Interventional Trial

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Barbara K Smith, PT, PhD, University of Florida

Clinicaltrials.gov ID (11 digit #):

NCT03645031

Neals Affiliated?

No

Coordinating Center Contact Information

University of Florida
Barbara K Smith, PT, PhD / .(JavaScript must be enabled to view this email address) / 352-294-5315
Jessica Ehrbar, MS / .(JavaScript must be enabled to view this email address) / 352-273-6855
Gainesville, Florida 32611 United States

Full Study Summary:

Most ALS patients survive less than 5 years after diagnosis, and the main cause of death is respiratory failure. The investigators are interested in the therapeutic potential of acute intermittent hypoxia (AIH) for individuals with neuromuscular diseases, such as ALS. More than two decades of research indicates AIH elicits meaningful respiratory and non-respiratory motor recovery. Acute intermittent hypoxia (AIH) consists of alternating periods of breathing mildly hypoxic (lowered oxygen concentration) and normoxic (normal oxygen concentration) air.

The investigators propose to study mechanisms of respiratory plasticity associated with a single presentation of mild AIH. The fundamental hypothesis guiding this proposal is that even a single AIH trial improves respiratory (and non-respiratory) motor function in ALS patients procedure. Participants will then be asked to breathe air with reduced oxygen for short periods of time, for a duration of 45 minutes. The activity of your muscles and your heart function will be monitored throughout the procedure.

Study Sponsor:

University of Florida

Participant Duration:

Estimated Enrollment:

32

Estimated Study Start Date:

10/01/2018

Estimated Study Completion Date:

03/31/2020

Posting Last Modified Date:

12/14/2018

Date Study Added to alsconsortium.org:

12/14/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    30

    Maximum Age:

    65

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    a healthy adult
    clinical diagnosis of ALS
    baseline FVC >50% predicted for age, sex and height.

    Exclusion Criteria:

    pregnant
    diagnosed cardiovascular disease
    a BMI >30 kg/m2
    currently take selective serotonin reuptake inhibitors (SSRI)
    history of seizures
    history of hospitalization for sepsis
    respiratory infection or took antibiotic medications within the past 4 weeks
    use external respiratory support during any waking hours
    participate in a pharmaceutical trial to treat ALS
    have any other medical condition the PI or medical director identify would make it unsuitable to participate.

  • Site Contact Information

    University of Florida
    Gainesville, Florida 32611
    United States

    UF Clinical Research Center
    Gainesville, Florida 32610
    United States

Treatment of FUS-Related ALS With Betamethasone - The TRANSLATE Study

Study Purpose:

By doing this study the investigator hopes to learn more about a potential cause of amyotrophic lateral sclerosis (ALS) called "oxidative stress". Oxidative stress is essentially an imbalance between the production of certain chemicals in the body called "free radicals" and the ability of the body to counteract or detoxify their harmful effects through neutralization by antioxidants. It is thought that factors such as environmental exposure (chemicals and lead), diet, smoking,alcohol consumption, physical activity and psychological stress cause oxidative stress to occur inside the body.

By doing this study, the investigator hopes to learn whether the FDA-approved steroid medication called Betamethasone will restore overall antioxidant activity fALS patients with mutations in the Fused in Sarcoma gene (FUS gene).

Participants who agree to take part in this research study, agree to the following responsibilities:

  • Attend all scheduled visits
  • Notify the study doctor of any illnesses, unexpected or troublesome side effects, or any other medical problems that occur during the study
  • Be completely honest with their answers to all questions
  • Check with the study doctor before taking any new medications, whether prescribed or "over the counter," even vitamins and herbal supplements.

Disease:

Familial ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Edward Kasarskis, MD, University of Kentucky

Clinicaltrials.gov ID (11 digit #):

NCT03707795

Neals Affiliated?

No

Coordinating Center Contact Information

University of Kentucky Medical Center
Meghann Bruno, RN / .(JavaScript must be enabled to view this email address) / 859-218-5064
Meha Joshi / .(JavaScript must be enabled to view this email address) / 859-218-5046
Lexington, Kentucky 40475 United States

Full Study Summary:

This will be a single-blinded, pharmacokinetic and pharmacodynamics study of intramuscular (IM) betamethasone in ALS patients and non-ALS relatives from families with a mutation in the FUS gene. Participants remain blinded as to their genotype.

Betamethasone is a FDA-approved drug and is only available in an IM dosing form (Celestone®). This will be a proof-of-concept translational study built on discoveries made by our research team at the University of Kentucky. All participants will receive active drug.

The research procedures will be conducted at the University of Kentucky (UK) Albert B. Chandler Hospital, Pavilion H, 800 Rose Street, Lexington, KY. Participants will need to come to Center for Clinical and Translational Science (CCTS) on the 3rd Floor (Room C300) of Pavilion H, at UK Albert B. Chandler Hospital, for a Consent/Screening Visit. If found eligible and qualify for the study, participants will be asked to give voluntary written consent to participate.

Following signing the consent form, participants will be admitted to the UK Albert B. Chandler Hospital, CCTS Inpatient Unit, 5th Floor, 5 North Wing of the Hospital for 2 nights (approximately 48 hours). The following tests and procedures will take place:

  • Neurological Exam
  • Vital Signs (blood pressure, heart rate and respiratory rate) will be measured
  • Medical and medication history will be collected
  • Questionnaire
  • blood specimen for Pharmacokinetic and Pharmacodynamic will be collected testing*
  • Participants will also be asked to do some testing of their breathing and physical abilities.

The study drug (betamethasone sodium phosphate/betamethasone acetate [Celestone® Soluspan®]) will be injected into a muscle such as arm or buttock - this will be the first of the four injections of the study drug administered during the study.

At 24 hours after the first injection with the study drug, and while still in the the hospital, the study drug will again be injected into a muscle such as arm or buttock - this will be the second of the four injections of the study drug administered during the study.

Following this second treatment, and after the study doctor determines it is safe, the participant will be discharged from the hospital. They will will stay in a local motel overnight, awaiting the next day's blood draw, assessments, testing and 3rd treatment with study drug.

Participants who do not live close to the hospital, motel accommodations will be provided at no charge, for an overnight stay between the 48 Hour visit, and the 72 Hour scheduled follow-up visit.

At 72 hours after first injection with the study drug, the study drug will again be injected into a muscle such as arm or buttock - this will be the fourth and last of the four injections administered during the study. Following this fourth treatment, and after the study doctor determines if is safe, participants will be allowed to leave.

The 72 Hour (Day 3), 168 Hour (Day 7) and 336 Hour (Day 14) visits will take about 1 hour each. The approximate, total amount of time participants will be asked to volunteer for this study is 51 hours over the 336 hour (14 day) duration of this research study.

Participants or their insurance company, Medicare or Medicaid will be responsible for the costs of all routine medical care and treatment they would normally receive for their condition. The University of Kentucky may not be allowed to bill insurance companies, Medicare or Medicaid for the medical procedures done strictly for research.

Neither the participant or their provider will be charged for costs of any of the procedures performed for the research study.

Study Sponsor:

University of Kentucky

Participant Duration:

Estimated Enrollment:

30

Estimated Study Start Date:

08/21/2017

Estimated Study Completion Date:

12/31/2018

Posting Last Modified Date:

12/14/2018

Date Study Added to alsconsortium.org:

12/14/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    20

    Maximum Age:

    80

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Diagnosis of familial ALS (fALS)
    Relative of a fALS person and carry the FUS gene

    Exclusion Criteria:

    Under 20 years or over 80 years of age
    Cannot tolerate steroids, including betamethasone
    Are unwilling or unable to attend all scheduled research visits
    Currently participating in another clinical drug trial
    Major neurological disease, other than ALS
    Pregnant

  • Site Contact Information

    University of Kentucky Medical Center
    Meha Joshi / .(JavaScript must be enabled to view this email address) / 859-218-5046
    Meghann Bruno / .(JavaScript must be enabled to view this email address) / 859-218-5064
    Lexington, Kentucky 40475
    United States

Investigation on the Cortical Communication (CortiCom) System

Study Purpose:

The CortiCom system consists of 510(k)-cleared components: platinum PMT subdural cortical electrode grids, a Blackrock Microsystems patient pedestal, and an external NeuroPort Neural Signal Processor. Up to two grids will be implanted in the brain, for a total channel count of up to 128 channels, for six months. In each participant, the grid(s) will be implanted over areas of cortex that encode speech and upper extremity movement.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Device

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03567213

Neals Affiliated?

No

Coordinating Center Contact Information


Emily Caporello Bluvas, Ph.D. / .(JavaScript must be enabled to view this email address) / 301-679-0969
.(JavaScript must be enabled to view this email address)

Full Study Summary:

The successful adoption of brain-computer interfaces (BCIs) as assistive technologies (ATs) for disabled populations depends on the ability to elicit rapid, intuitive, and reliable control signals. To date, it is not known which sources of neural information provide the most natural and efficient means of control. This study will directly assess the efficacy of two sources of neural control signals, speech and motor cortex, for BCI control of software and devices using investigators' Cortical Communication (CortiCom) system.

The CortiCom system consists of 510(k)-cleared components: platinum PMT subdural cortical electrode grids, a Blackrock Microsystems patient pedestal, and an external NeuroPort Neural Signal Processor. Up to two grids will be implanted in the brain, for a total channel count of up to 128 channels, for six months. In each participant, the grid(s) will be implanted over areas of cortex that encode speech and upper extremity movement.

Investigators' study will be the first to investigate the efficiency and intuitiveness of two contrasting neural control strategies for BCI: Motor imagery and speech. As the first study to chronically and simultaneously record from human speech and motor regions, this study seeks to achieve the following:

  • demonstrate the ability to decode intended upper extremity movements using information decoded from primary motor cortex arm/hand area;
  • demonstrate the ability to decode intended speech from articulatory speech cortex; and
  • determine the most effective and intuitive user strategies (e.g. imagined speech or imagined movement) and optimal neural sources for brain-computer interactions,

The patient populations targeted in this study are amyotrophic lateral sclerosis (ALS), brainstem stroke, locked-in syndrome (LIS), and tetraplegia. Individuals within these populations may have normal cortical function and cognition while suffering from motor or combined speech and motor deficits.

Based on research by colleagues, as well as investigators' own experience working with participants affected by epilepsy implanted with high density electrocorticographic grids, investigators hypothesize that long-term recording of neural activity from the targeted cortical areas may provide a new communication channel for these clinical populations. The utilization of high-channel-count (up to 128 channel) ECoG grids, in combination with simultaneous coverage of speech and motor cortex, will enable investigations into the performance of speech-mediated and motor-mediated control efficacy as applied to a variety of end effectors, such as computers, tablets, headsets for virtual or augmented reality, smart lights, televisions, and assistive technologies. Additionally, eye-tracking may be utilized in combination with neural commands to improve target selection performance and ease.

Through this study, investigators will assess the performance of speech- and motor-mediated control using chronic, high-channel count ECoG grid neural implants in pursuit of a high-performing, clinically beneficial BCI assistive technology.

Study Sponsor:

Johns Hopkins University

Participant Duration:

Estimated Enrollment:

5

Estimated Study Start Date:

12/31/2018

Estimated Study Completion Date:

06/30/2019

Posting Last Modified Date:

01/30/2019

Date Study Added to alsconsortium.org:

10/19/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    22

    Maximum Age:

    70

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Clinical diagnosis of tetraplegia (quadriplegia), brainstem stroke , amyotrophic lateral sclerosis (ALS) or Locked-in Syndrome (LIS)
    Tetraplegia diagnosis, ALS diagnosis, stroke, or LIS etiology onset occurred at least one year prior to enrollment
    Complete or incomplete tetraplegia (quadriplegia), tetraparesis (quadriparesis), or severe ataxia. In addition, these motor impairments may be combined with severe motor-related speech impairment (dysarthria or anarthria), as in LIS.
    22-70 years
    Meeting surgical safety criteria, including surgical clearance by the participant's primary healthcare provider, study physicians, and any necessary consultants
    Ability to communicate reliably, such as through eye movement
    Willingness and ability to provide informed consent
    Screened by rehabilitation psychologist with a result showing that the participant has a stable psychosocial support system with caregiver capable of monitoring participant throughout the study
    Ability and willingness to travel up to 100 miles to study location up to three days per week for the duration of the study
    Ability to understand and comply with study session instructions
    Participant consents to the study and still wishes to participate at the time of the study

    Exclusion Criteria:

    Performance on formal neuropsychological testing that indicates significant psychiatric conditions or cognitive impairments that would interfere with obtaining informed consent or fully participating in study activities.
    Suicide attempt or persistent suicidal ideation within the past 12 months.
    Implanted devices that are incompatible with MRI, which may include pacemakers, cardiac defibrillators, spinal cord or vagal nerve stimulators, deep brain stimulators, and cochlear implants.
    History of substance abuse, narcotic dependence, or alcohol dependence in past 24 months
    Medical conditions contraindicating surgery of a chronically implanted device (e.g. osteomyelitis, diabetes, hepatitis, any autoimmune disease/disorder, epilepsy, skin disorders causing excessive skin sloughing or poor wound healing, blood or cardiac disorder requiring chronic anti-coagulation)
    Other chronic, unstable medical conditions that could interfere with subject participation.
    Presence of pre-surgical findings in anatomical, functional, and/or vascular neuroimaging that makes achieving implant locations within desired risk levels too challenging (to be decided by neurological and neurosurgical team)
    Prior cranioplasty
    Inability to undergo MRI or anticipated need for an MRI during the study period
    Participants with active infections or unexplained fever
    Participants with other morbid conditions making the implantation of the recording elements unsafe; not limited to: significant pulmonary, cardiovascular, metabolic, or renal impairments making the surgical procedure unsafe
    Pregnancy (confirmation through blood test)
    Nursing an infant, planning to become pregnant, or not using adequate birth control
    Corrected vision poorer than 20/100
    HIV or AIDS infection
    Existing scalp lesions or skin breakdown
    Chronic oral or intravenous use of steroids or immunosuppressive therapy
    Active cancer within the past year or requires chemotherapy
    Uncontrolled autonomic dysreflexia within the past 3 months
    Hydrocephalus with or without an implanted ventricular shunt
    Participants in whom it is medically contraindicated to stop anti-coagulant medications during surgery

  • Site Contact Information

    Johns Hopkins Medicine
    Nathan E. Crone, MD / .(JavaScript must be enabled to view this email address) / 410-955-6772
    Baltimore, Maryland 21205
    United States

A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 Administered Intrathecally to Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis

Study Purpose:

The primary objective of this study is to evaluate the safety and tolerability of BIIB078 in adults with C9ORF72-ALS. The secondary objective of this study is to evaluate the pharmacokinetic profile of BIIB078.

Disease:

Amyotrophic Lateral Sclerosis (ALS)

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Biogen Medical Director

Clinicaltrials.gov ID (11 digit #):

NCT03626012

Neals Affiliated?

No

Coordinating Center Contact Information


US Biogen Medical Information / .(JavaScript must be enabled to view this email address) / 866-633-4636
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Biogen

Participant Duration:

Estimated Enrollment:

59

Estimated Study Start Date:

09/10/2018

Estimated Study Completion Date:

11/29/2020

Posting Last Modified Date:

10/19/2018

Date Study Added to alsconsortium.org:

10/19/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Key Inclusion Criteria:

    Ability of the subject to understand the purpose and risks of the study, to provide signed and dated informed consent, and to authorize the use of confidential health information in accordance with national and local subject privacy regulations; or, in the event of the subject's physical incapacity to sign, to confirm that understanding and consent orally to a legally authorized representative (LAR) for the express purpose of having said informed consent and authorization signed on his/her behalf.
    All subjects of childbearing potential must agree to practice highly effective contraception during the study and be willing and able to continue contraception for 5 months after their last dose of study treatment.
    Must meet the possible, laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria and have documentation of a clinical genetic test demonstrating the presence of a pathogenic mutation in C9ORF72.
    Slow vital capacity (SVC) ≥ 50% of predicted value as adjusted for sex, age, and height (from the sitting position).
    Subjects taking concomitant riluzole at study entry must be on a stable dose for ≥ 30 days prior to the first dose of study treatment (Day 1).
    Subjects taking concomitant edaravone at study entry must be on a stable dose for ≥ 60 days prior to the first dose of study treatment (Day 1).
    ALS Cognitive Behavioral Screen (ALS-CBS) score ≥ 11 for the cognitive portion; ≥ 33 for the behavioral portion.
    Medically able to undergo the study procedures, and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.
    Screening values of coagulation parameters including platelet count, international normalized ratio (INR), prothrombin time (PT), and activated partial thromboplastin time (APTT) should be within normal ranges.
    Has an informant/caregiver who, in the Investigator's judgment, has frequent and sufficient contact with the subject as to be able to provide accurate information about the subject's cognitive and functional abilities at Screening.

    Key Exclusion Criteria:

    History of drug abuse or alcoholism ≤ 6 months of Screening that would limit participation in the study, as determined by the Investigator.
    Tracheostomy.
    History of or positive test result at Screening for human immunodeficiency virus. .
    History of, or positive test result at Screening for, hepatitis C virus antibody.
    Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of study agent, whichever is longer.
    Treatment with anti-platelet or anticoagulant therapy ≤ 14 days before Screening (with the exception of aspirin ≤ 325 mg/day) or anticipated use during the study.
    Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system during the study period.
    Female subjects who are pregnant or currently breastfeeding.

    NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

  • Site Contact Information

    Research Site
    La Jolla, California 92093
    United States

    Research Site
    Miami, Florida 33136
    United States

    Research Site
    Baltimore, Maryland 21205
    United States

    Research Site
    Boston, Massachusetts 02114
    United States

    Research Site
    Saint Louis, Missouri 63110
    United States

    Research Site
    Lincoln, Nebraska 68506
    United States

    Research Site
    Knoxville, Tennessee 37920
    United States

    Research Site
    Montreal, Quebec H3A 2B4
    Canada

Effect of Oral Levosimendan (ODM-109) on Respiratory Function in Patients with ALS (REFALS)

Study Purpose:

The purpose of the study is to evaluate safety and the effectiveness of levosimendan in preserving respiratory function in patients with amyotrophic lateral sclerosis (ALS).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase III

Study Chair(s)/Principal Investigator(s):

Merit Cudkowicz, MD, Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):

NCT03505021

Neals Affiliated?

Yes

Coordinating Center Contact Information

Massachusetts General Hospital
Merit E Cudkowicz, MD / .(JavaScript must be enabled to view this email address) / 617-726-2383
Merja Mäkitalo / .(JavaScript must be enabled to view this email address) / +358 10 426 7737
Boston, Massachusetts 02114 United States

Full Study Summary:

This study will evaluate whether prolonged oral levosimendan can preserve respiratory function more effectively than placebo, resulting in better patient functionality as measured by the ALSFRS-R scale. In this randomized, double-blind, placebo-controlled, parallel-group, multicenter study, subjects are allocated in a 2:1 ratio to receive either levosimendan (1 -2 mg daily) or placebo for 48 weeks. The primary endpoint is slow vital capacity (SVC) at 12 weeks, with the impact on patient function assessed through 48 weeks, adjusted for patient outcome, using ALSFRS-R (combined assessment of function and survival, CAFS). Other important efficacy measures include time to respiratory events, clinical global impression (CGI), assessment of dyspnea using the Borg scale and sleep scales (Pittsburgh sleep quality index and Epworth sleepiness scale). Patient safety is monitored using conventional methods including adverse events, safety laboratory tests, vital signs and 12-lead EKG. Following screening and baseline visits, patients attend the clinic at 2, 4, 8, 12, 24, 36 and 48 weeks, with telephone assessments conducted at weeks 18, 30 and 42. An end of study visit is performed 14-25 days after the last study treatment administration. The study will be monitored by an independent data and safety monitoring board.  A long-term extension study will be available for patients completing the study.

Study Sponsor:

Orion Corporation, Orion Pharma

Participant Duration:

Total study duration for each subject will be 51-52 weeks, including the screening period, 48 weeks treatment and an end-of-study visit.

At screening, the visit length will be approximately 2 – 2.5hrs. 

For visits 2, 3 and 8, the visit length will be approximately 3 – 5 hrs.

For visits 4 to 7, the visit length will be approximately 2 – 3hrs.

There will also be three telephone contacts made in-between sites visits that should not last longer than 30 minutes.

Depending on location of sites, the study participant may be required to travel.  

Estimated Enrollment:

450

Estimated Study Start Date:

06/21/2018

Estimated Study Completion Date:

10/30/2020

Posting Last Modified Date:

01/22/2019

Date Study Added to alsconsortium.org:

07/06/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    60-90

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion criteria

    Written or verbal informed consent (IC) for participation in the study
    Diagnosis of laboratory supported probable, probable or definite ALS according to El Escorial revised criteria.
    Able to swallow study treatment capsules, and in the opinion of the investigator, is expected to continue to do so during the study.
    Disease duration from symptom onset (defined by first muscle weakness or dysarthria) 12-48 months at the time of visit 1 (baseline)
    Able to perform supine SVC in an adequate and reliable way at screening and baseline visits as judged by the investigator.
    Subjects with or without riluzole (up to 100mg/day, stable at least 4 weeks) and/or edaravone (at least one 28-day treatment cycle completed).

    Exclusion criteria

    Other causes of neuromuscular weakness not excluded.
    Diagnosis of another neurodegenerative disease (e.g. Parkinson’s or Alzheimer’s disease).
    Assisted ventilation of any type within 3 months before the screening visit or at screening.
    Any use of a diaphragm pacing system (DPS) within 3 months before the screening visit.
    Any form of stem cell or gene therapy for the treatment of ALS.
    Known hypersensitivity to levosimendan.
    Administration of levosimendan within 3 months before the screening visit or previous participation in oral levosimenan trial.
    Any use of tirasemtiv or reldesemtiv within 1 month before the screening visit.
    Participation in a clinical trial with any experimental treatment within 30 days or within 5 half-lives of that treatment (whichever is longer) before the screening visit.
    Any botulinum toxin use within 3 months before the screening visit.
    Recorded diagnosis or evidence of major psychiatric diagnosis, significant cognitive impairment or clinically evident dementia that may interfere with the patient’s ability to comply with study procedures.
    Pulmonary illness (e.g. asthma or COPD) requiring regular treatment.
    Haemodynamically significant uncorrected valve disease or hypertrophic cardiomyopathy or restrictive cardiomyopathy.
    Any cardiovascular event (e.g. myocardial infarction, HF, arrhythmia or stroke) requiring hospitalisation within 3 months before the screening visit.
    History of Torsades de Pointes (TdP) or diagnosed long QT-syndrome.
    History of life-threatening ventricular arrhythmia, unless treated with reliable measures to prevent recurrence (e.g. with placement of implantable cardioverter defibrillator [ICD] or catheter ablation).
    History of second or third degree atrioventricular (AV) block or sinus node disease at screening, if not treated with pacemaker.
    HR repeatedly > 100 bpm in the 12-lead ECG after a 5-minute rest at screening.
    Systolic blood pressure (SBP) < 90 mmHg at screening.
    Potassium < 3.7 mmol/l or > 5.5 mmol/l at screening.
    Severe renal impairment (creatinine clearance < 30 ml/min at screening), creatinine > 170 μmol/l at screening or on dialysis.
    Blood haemoglobin < 10 g/dl at screening or blood donation or loss of significant amount of blood within 60 days before the screening visit.
    Clinically significant hepatic impairment at the discretion of the investigator.
    Body mass index (BMI) ≤ 18.5kg/m2 (BMI = weight/height2).
    Women who are lactating or of reproductive age without a negative pregnancy test and without a commitment to using a highly effective method of contraception (e.g. oral hormonal contraceptives associated with inhibition of ovulation, intrauterine devices and long acting progestin agents), if sexually active during the study, and for 1 month after the last dose of the study treatment. Women who are postmenopausal (1 year since last menstrual cycle), surgically sterilised or who have undergone a hysterectomy are considered not to be reproductive and can be included.
    Patient judged to be actively suicidal by the investigator during 3 months before the screening visit.
    Patients with known history of human immunodeficiency virus (HIV) infection.
    Any other clinically significant cardiovascular, gastrointestinal, hepatic, renal, neurological or psychiatric disorder or any other major concurrent illness that in the opinion of the investigator could interfere with the interpretation of the study results or constitute a health risk for the subject if he/she took part in the study.

  • Site Contact Information

    Neuromuscular Research Center and Neuromuscular Clinic of Arizona
    Kumaraswamy Sivakumar, MD / .(JavaScript must be enabled to view this email address) / 480-314-1007
    Phoenix, Arizona 85028
    United States

    Phoenix Neurological Associates
    Phoenix, Arizona 85006
    United States

    University of California San Diego
    Rose Previte / .(JavaScript must be enabled to view this email address) / La Jolla, California 92037
    United States

    Cedars-Sinai Medical Center
    Dana Fine / .(JavaScript must be enabled to view this email address) / 310-423-8497
    Los Angeles, California 90048
    United States

    University of California Irvine
    Ivonne Turner / .(JavaScript must be enabled to view this email address) / 714-546-8323
    Orange, California 92868
    United States

    California Pacific Medical Center
    San Francisco, California 94115
    United States

    Colorado Springs Neurological Associates
    Lisa Deschaine / 719-389-1129
    Colorado Springs, Colorado 80907
    United States

    Hospital for Special Care
    Honora Dalamagas / .(JavaScript must be enabled to view this email address) / 860-612-6356
    Stephanie Beck / .(JavaScript must be enabled to view this email address) / 860-827-1958 ext 5753
    New Britain, Connecticut 06053
    United States

    The George Washington Medical Faculty Associates
    Lindsey Covington / .(JavaScript must be enabled to view this email address) / 202-741-2745
    Washington, District of Columbia 20037
    United States

    Georgetown University
    Sasha Warren / 202-444-6941
    Washington, District of Columbia 20007
    United States

    University of Florida Health - Jacksonville
    Lisa Smith / .(JavaScript must be enabled to view this email address) / Jacksonville, Florida 32209
    United States

    University of South Florida
    Brittany Harvey / .(JavaScript must be enabled to view this email address) / 813-974-9413
    Tampa, Florida 33612
    United States

    Holy Cross Medical Center
    Lindita Burba / .(JavaScript must be enabled to view this email address) / 954-489-4314
    Fort Lauderdale, Florida 33308
    United States

    Mayo Clinic
    Arijana Draganovic / .(JavaScript must be enabled to view this email address) / 904-953-6912
    Jacksonville, Florida 32224
    United States

    University of Florida
    Jennifer Steshyn / .(JavaScript must be enabled to view this email address) / Gainesville, Florida 32611
    United States

    Emory University School of Medicine
    Jane Bordeau / .(JavaScript must be enabled to view this email address) / Atlanta, Georgia 30322
    United States

    Augusta University
    Michael H. Rivner, MD / .(JavaScript must be enabled to view this email address) / Augusta, Georgia 30912
    United States

    Northwestern University Feinberg School of Medicine
    Benjamin Joslin / .(JavaScript must be enabled to view this email address) / Chicago, Illinois 60611
    United States

    University of Kentucky Chandler Medical Center
    Meha Joshi / .(JavaScript must be enabled to view this email address) / 859-218-5046
    Lexington, Kentucky 40536
    United States

    Kentucky Neuroscience Research
    Annette Robinson / .(JavaScript must be enabled to view this email address) / 502-540-3585
    Louisville, Kentucky 40202
    United States

    Johns Hopkins Hospital
    Kristen Riley / .(JavaScript must be enabled to view this email address) / Baltimore, Maryland 21205
    United States

    Massachuesetts General Hospital
    Olivia Pijanowski / .(JavaScript must be enabled to view this email address) / 617-643-2522
    Boston, Massachuesetts 02114
    United States

    University of Michigan
    Jayna Duell / .(JavaScript must be enabled to view this email address) / 734-936-8776
    Kalamazoo, Michigan 49007
    United States

    Mayo Clinic - Rochester
    Rochester, Minnesota 55905
    United States

    HealthPartners Specialty Center
    Gaurav Guliani / .(JavaScript must be enabled to view this email address) / 651-495-6363
    Saint Paul, Minnesota 55130
    United States

    Washington University School of Medicine
    Kelly Streckfuss / .(JavaScript must be enabled to view this email address) / Saint Louis, Missouri 63110
    United States

    Neurology Associates
    Ashley Calhoun / .(JavaScript must be enabled to view this email address) / 402-770-7403
    Lincoln, Nebraska 68506
    United States

    Mount Sinai Beth Israel
    Stephen Scelsa, MD / .(JavaScript must be enabled to view this email address) / 212-844-8490
    New York, New York 10003
    United States

    Hospital for Special Surgery
    Shara Holzberg / .(JavaScript must be enabled to view this email address) / 646-797-8592
    New York, New York 10021
    United States

    Columbia Presbyterian Hospital
    Jinsy Andrews, MD / .(JavaScript must be enabled to view this email address) / Meenakshi Rozenstrauch / .(JavaScript must be enabled to view this email address) / 212-305-2233
    New York, New York 10032
    United States

    Neurosciences Institute - Neurology Charlotte
    Cynthia Lary / .(JavaScript must be enabled to view this email address) / Charlotte, North Carolina 28207
    United States

    Duke University Medical Center
    Karen Grace / .(JavaScript must be enabled to view this email address) / 919-668-2844
    Durham, North Carolina 27705
    United States

    Wake Forest University Baptist Medical Center
    James Caress, MD / 336-713-8577
    Winston-Salem, North Carolina 27157
    United States

    The Ohio State University
    Adam Quick / .(JavaScript must be enabled to view this email address) / Rory Eustace / .(JavaScript must be enabled to view this email address) / Columbus, Ohio 43210
    United States

    Providence Brain and Spine Institute
    Arlena Cummings / .(JavaScript must be enabled to view this email address) / 503-962-1171
    Portland, Oregon 97213
    United States

    Oregon Health and Science University
    Anneka Sonstroem / .(JavaScript must be enabled to view this email address) / 503-494-7394
    Portland, Oregon 97201
    United States

    University of Pittsburgh Medical Center
    Danielle Rowlands / .(JavaScript must be enabled to view this email address) / 412-864-2873
    Pittsburgh, Pennsylvania 15213
    United States

    University of Pennsylvania
    Kelly Almasy / .(JavaScript must be enabled to view this email address) / 215-829-5041
    Philadelphia, Pennsylvania 19107
    United States

    Allegheny General Hospital
    Sandeep Rana / .(JavaScript must be enabled to view this email address) / Pittsburgh, Pennsylvania 15212
    United States

    Temple University School of Medicine
    Kathleen Hatala / .(JavaScript must be enabled to view this email address) / 215-707-4171
    Theresa Ropars / .(JavaScript must be enabled to view this email address) / 215-707-5440
    Philadelphia, Pennsylvania 19140
    United States

    Nerve and Muscle Center of Texas
    Zinah Rasheed / .(JavaScript must be enabled to view this email address) / 713-795-0033
    Houston, Texas 77030
    United States

    Texas Neurology
    Daragh Heitzman / 214-827-3610
    Dallas, Texas 75214
    United States

    University of Utah
    Mark Bromberg / 801-585-7575
    Salt Lake City, Utah 84108
    United States

    University of Vermont Medical Center
    Shannon Lucy / .(JavaScript must be enabled to view this email address) / 802-656-4582
    Burlington, Vermont 05401
    United States

    Swedish Neuroscience Institute
    Robin Smith / .(JavaScript must be enabled to view this email address) / 206-320-2334
    Seattle, Washington 98122
    United States

    University of Washington Medical Center
    Laura Sissons-Ross / .(JavaScript must be enabled to view this email address) / 206-543-0081
    Seattle, Washington 98195
    United States

    Froedtert Hospital
    Lynn Wheeler / .(JavaScript must be enabled to view this email address) / 414-805-9307
    Milwaukee, Wisconsin 53226
    United States

    Brain and Mind Centre
    Eleanor Ramsey / 02 9351 0976
    Camperdown, New South Wales 2050
    Australia

    Perron Institute for Neurological and Translational Science
    Joanne Borrelli / .(JavaScript must be enabled to view this email address) / Murdoch, Western Australia 6150
    Australia

    Calvary Health Care Bethlehem
    Emma Windebank / .(JavaScript must be enabled to view this email address) / Caulfield South, Victoria 3162
    Australia

    Flinders Medical Centre
    David Schultz / 61 8 8204 4187
    Bedford Park, South Australia 5042
    Australia

    Royal Brisbane and Women's Hospital
    Susan Heggie / .(JavaScript must be enabled to view this email address) / 07 3646 8111
    Brisbane, Queensland 4029
    Australia

    Universität Innsbruck
    Wolfgang Löscher / .(JavaScript must be enabled to view this email address) / Marina Peball / .(JavaScript must be enabled to view this email address) / 43-512-504-25810
    Innsbruck, Tyrol 6020
    Austria

    Salzkammergut-Klinikum Vöcklabruck
    Vöcklabruck, Upper Austria 4840
    Austria

    Medizinische Universität Wien
    Wien, 1090
    Austria

    Universitaire Ziekenhuis Leuven
    Philip Van Damme / 016 344280
    Leuven, Flemish 3000
    Belgium

    Algemeen Ziekenhuis St. Lucas Gent
    Jan De Bleeker / 32-9-2246530
    Gent, Oost-Vlaanderen 9000
    Belgium

    Centre Hospitalier Régional de la Citadelle
    Stephanie Delstanche / .(JavaScript must be enabled to view this email address) / 00 32 4 321 82 24
    Liège, Liege 4000
    Belgium

    Stan Cassidy Centre for Rehabilitation
    Colleen O'Connell / .(JavaScript must be enabled to view this email address) / Susan McCully / .(JavaScript must be enabled to view this email address) / Fredericton, New Brunswick E3B 0C7
    Canada

    Centre De Recherche Du Centre Hospitalier de l'Universite de Montreal - Hopital Notre-Dame
    Geneviève Matte / 514-890-8000 ext 27208
    Montreal, Quebec H2L 4M1
    Canada

    Alberta Health Services - Neuromuscular Clinic
    Jose Martinez / .(JavaScript must be enabled to view this email address) / 403-210-7009
    Janet Petrillo / .(JavaScript must be enabled to view this email address) / 403-210-7006
    Calgary, Alberta T3M 1M4
    Canada

    Montreal Neurological Institute and Hospital
    Angela Genge, MD / .(JavaScript must be enabled to view this email address) / 514-298-3868
    Natalie Saunders / .(JavaScript must be enabled to view this email address) / 514-398-6526
    Montreal, Quebec H3A 2B4
    Canada

    University of Alberta
    Wendy Johnston / 780-248-1089
    Kelsey Tymkow / 780-248-1329
    Edmondton, Alberta T6G 2G3
    Canada

    Moncton Hospital, Southeast Regional Health Authority
    Cynthia Lary / .(JavaScript must be enabled to view this email address) / Moncton, New Brunswick E1C 2Z3
    Canada

    Sunnybrook Health Sciences Centre
    Adrienne Sulistyo / .(JavaScript must be enabled to view this email address) / 416-480-6100 ext 87561
    Toronto, Ontario M4N 3M5
    Canada

    McMaster University Medical Centre
    Daniela Trapsa / .(JavaScript must be enabled to view this email address) / Hamilton, Ontario L8N 3Z5
    Canada

    Centre Hospitalier Affilie Universitaire de Quebec
    Alexandra Simard / 418-649-0252
    Quebec, Quebec G1J 1Z4
    Canada

    Etelä-Karjalan keskussairaala
    Lappeenranta, 53130
    Finland

    Turku University Hospital
    Turku, 20521
    Finland

    Neurologian Poliklinikka - Meilahden Tornisairaala 3
    Helsinki, 00029
    Finland

    Universitätsklinikum Jena
    Julian Großkreutz / .(JavaScript must be enabled to view this email address) / 4936419323428
    Jena, Thuringen 07747
    Germany

    Deutsche Klinik für Diagnostik
    Martina Müller / .(JavaScript must be enabled to view this email address) / 49 611-577652
    Wiesbaden, Hessen 65191
    Germany

    Universitätsklinikum Münster
    Matthias Boentert / .(JavaScript must be enabled to view this email address) / +49 251-834-8196
    Münster, Nordrhein-Westfalen 48149
    Germany

    Universitätsklinikum Carl Gustav Carus
    Andreas Hermann / R. Guenther / 493514582524
    Dresden, Sachsen 01307
    Germany

    Charité Universitätsmedizin Berlin - Campus Virchow-Klinikum
    Thomas Meyer / 0049 30 450 560 028
    Berlin, 13353
    Germany

    Universitätsklinikum Ulm
    A. C. Ludolph / .(JavaScript must be enabled to view this email address) / Ulm, Baden-Württemberg 89081
    Germany

    Universitätsmedizin Rostock
    Rostock, Mecklenburg-western-pommerania 18147
    Germany

    Medizinische Hochschule Hannover
    Susanne Petri / .(JavaScript must be enabled to view this email address) / Hannover, Niedersachsen 30625
    Germany

    Alfried Krupp Krankenhaus Rüttenscheid
    Essen, Nordrhein-westfalen 45131
    Germany

    Beaumont Hospital - Ireland
    Orla Hardiman / .(JavaScript must be enabled to view this email address) / Dublin, 9
    Ireland

    Azienda Ospedaliera - Universitaria Città della Salute e della Scienza di Torino
    Adriano Chio / .(JavaScript must be enabled to view this email address) / 39 0116709170
    Torino, 10126
    Italy

    Azienda Ospedaliera Universitaria-Maggiore della Carità di Novara
    Latizia Mazzini / .(JavaScript must be enabled to view this email address) / Novara, 28100
    Italy

    ICS Maugeri Spa SB
    Gabriele Mora / .(JavaScript must be enabled to view this email address) / Pavia, 27100
    Italy

    Azienda Ospedaliero-Universitaria Pisana Ospedale Santa Chiara
    Pisa, 56126
    Italy

    Policlinico Umberto I di Roma
    Roma, 0016
    Italy

    Universitair Medisch Centrum Utrecht - Rudolf Magnus Instituut voor Neurowetenschappen
    Leonard Van den Berg / .(JavaScript must be enabled to view this email address) / 020 5668227
    Utrecht, 3584 CG
    Netherlands

    Academisch Medisch Centrum
    Marije Voermans / .(JavaScript must be enabled to view this email address) / 20 5668227
    Amsterdam, Noord-Holland 1105 AZ
    Netherlands

    Hospital San Rafael - Madrid
    Jesus S Mora Pardina / .(JavaScript must be enabled to view this email address) / Madrid, 28016
    Spain

    Hospital Universitari de Bellvitge
    Berta Blanco Burguet / 34932607586
    Barcelona, 08207
    Spain

    Hospital Universitario y Politécnico de La Fe
    Juan Francisco Vázquez / .(JavaScript must be enabled to view this email address) / Paula Lizandra / .(JavaScript must be enabled to view this email address) / Valencia, 46026
    Spain

    Hospital Universitario Reina Sofia
    Córdoba, 14011
    Spain

    Hospital de Basurto
    Luis Verona / .(JavaScript must be enabled to view this email address) / Bilbao, Vizcaya 48013
    Spain

    Norrlands Universitetssjukhus
    Peter M Andersen / .(JavaScript must be enabled to view this email address) / Umeå, 907
    Sweden

    Karolinska Universitetssjukhuset
    Karolina Palmback / 08 51771231
    Stockholm, 14186
    Sweden

    Centralsjukhuset Karlstad
    Karlstad, 651 85
    Sweden

    The Walton Centre NHS Foundation Trust
    C A Young / 0151 529-5666
    Liverpool, L9 7LJ
    United Kingdom

    Barts Health NHS Trust
    Aleksandar Radunovic / Kimberley Allen-Phillbey / 02035940637/8
    London, England E1 1BB
    United Kingdom

    King's College Hospital NHS Foundation Trust
    London , England SE5 9RS
    United Kingdom

A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)

Study Purpose:

The causes of ALS are largely unknown. However, mitochondrial dysfunction, resulting in impaired energy production, oxidative stress and apoptosis, may play a key role in ALS progression. Triheptanoin can improve mitochondrial function and energy production and therefore has potential for slowing ALS progression. Indeed, triheptanoin slowed motor neuron loss and delayed the onset of weakness in a mutant SOD1 model of ALS. This pilot trial will determine if Triheptanoin is safe tolerable, alters biomarkers of brain energy metabolism and oxidative stress, and slows functional decline in people with ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase I ,Phase II

Study Chair(s)/Principal Investigator(s):

Richard S. Bedlack, MD, PhD, Duke ALS Clinic

Clinicaltrials.gov ID (11 digit #):

NCT03506425

Neals Affiliated?

No

Coordinating Center Contact Information

Duke University
Richard S. Bedlack, MD, PhD / .(JavaScript must be enabled to view this email address) / 919-668-2839
Karen Grace, RN / .(JavaScript must be enabled to view this email address) / 919-668-2844
Durham, North Carolina 27705 United States

Full Study Summary:

Study Sponsor:

Richard Bedlack, M.D., Ph.D.

Participant Duration:

Estimated Enrollment:

10

Estimated Study Start Date:

06/21/2018

Estimated Study Completion Date:

02/28/2019

Posting Last Modified Date:

12/14/2018

Date Study Added to alsconsortium.org:

09/04/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Lab supported probable or more definite ALS by El Escorial Criteria
    Age greater than or equal to 18 years
    Willing and able to provide informed consent
    On riluzole at a stable dose for at least 30d or not taking this
    On Radicava at a stable dose for at least 30d or not taking this
    Life expectancy at least 6 months
    Currently managed on a reasonably stable diet, avoidance of fasting, carnitine or medium chain triglyceride (MCT) oils
    Must stop any other experimental ALS treatment for at least 30 days prior to screening
    If sexually active, must agree to use contraceptive or abstinence for duration of treatment with triheptanoin
    Females of child bearing age must have negative pregnancy test at screening

    Exclusion Criteria:

    Unwilling or unable to provide informed consent
    Previous intolerance or adverse reaction to triheptanoin or MCT
    Conditions that will prohibit MRI scanning (metal in eye, some surgical implants, claustrophobia, inability to lie supine)
    Have any other co-morbid conditions that in the opinion of the study investigator, places the participant at increased risk of complications, interferes with study participation or compliance, or confounds study objectives

  • Site Contact Information

    Duke University
    Durham, North Carolina 27705
    United States

Remote Pulmonary Function Testing in Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

The specific objective of this study is to validate the practice of remote pulmonary function testing (rPFT) conducted in the home through the use of connected mobile health devices and the Penn State Hershey ALS Telemanagement program.

The second part is a comparison of respiratory outcomes in patients receiving telemedicine-guided respiratory assessments monthly vs. those receiving standard of care. This is a randomized controlled study which assesses the effects rPFT on respiratory outcomes, quality of life, and survival.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Device

Study Status:

Not enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Zachary Simmons, Milton S. Hershey Medical Center

Clinicaltrials.gov ID (11 digit #):

NCT03214224

Neals Affiliated?

No

Coordinating Center Contact Information


Andrew Geronimo, PhD / .(JavaScript must be enabled to view this email address) / 717-531-0003 ext 282576
Anne Morris, MPH / .(JavaScript must be enabled to view this email address) / 717-531-0003 ext 289123
Hershey, Pennsylvania United States

Full Study Summary:

The specific objective of this study is to validate the practice of remote pulmonary function testing (rPFT) conducted in the home through the use of connected mobile health devices and the Penn State Hershey ALS Telemanagement program. The central hypothesis is that guided home assessment of respiratory function is a valid method for detecting respiratory insufficiency leading to noninvasive ventilation (NIV) recommendation. This study has the potential to transform the current practice of conducting breathing assessments every three months, resulting in timelier detection of respiratory insufficiency, thereby staining quality of life and lengthening survival. This protocol has the potential to demonstrate telemanagement exceeding the standards of ALS care.

The study has two parts, which patients can participate in either or both. The first part is a self-controlled study which will enroll 40 patients from the ALS clinic. On the day of their clinical visit, study participants will perform both a standard PFT as well as a simulated rPFT, both generating three valid repetitions of forced vital capacity (FVC) and maximal inspiratory pressure (MIP) procedures. The simulated rPFT will mimic the practice of home telemonitoring by having patients be instructed by a respiratory therapist over the telemanagement portal while in a research room within the ALS clinic. The primary hypothesis is that there is no difference in the results of PFT and rPFT for respiratory assessment of FVC and MIP.

The second part is a comparison of respiratory outcomes in patients receiving telemedicine-guided respiratory assessments monthly vs. those receiving standard of care. This is a randomized controlled study which assesses the effects rPFT on respiratory outcomes, quality of life, and survival. Forty patients with ALS will be randomized into two arms, both receiving standard respiratory assessments for pulmonary function every three months, and the experimental group additionally receiving rPFTs in the interim months over the course of a year. The primary hypothesis is that home respiratory assessment enables thresholds for NIV recommendation to be met in the experimental group significantly closer to the initiation criteria, at a time when quality of life is higher, compared to the control group.

Study Sponsor:

Milton S. Hershey Medical Center

Participant Duration:

Estimated Enrollment:

83

Estimated Study Start Date:

09/01/2017

Estimated Study Completion Date:

10/31/2019

Posting Last Modified Date:

05/10/2019

Date Study Added to alsconsortium.org:

08/15/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Part 1

    Patients:

    Possess a diagnosis of definite, probable, probable laboratory-supported, or possible ALS by revised El Escorial research criteria [Brooks2000].
    Be 18 years of age or older.
    Have a caregiver available to participate in the study

    Caregivers:

    Be 18 years of age or older, of either gender.
    Be able and willing to provide informed consent.

    Respiratory Therapist

    Be a member of the Hershey Medical Center ALS multidisciplinary care team.
    Be able and willing to provide verbal informed consent after receiving a summary explanation of research (SER).

    Part 2 imposes additional inclusion criteria for patients only.

    Patients:

    4) Symptom onset within the last three years. 5) Intent to attend the Penn State Hershey ALS clinic every three months for the next year.

    6) Have home wireless internet service sufficient for engaging in telemedicine sessions.

    Exclusion Criteria:

    Exclusion criteria are the same for both parts of the study.

    Patients:

    Use of NIV or diaphragm pacer at time of obtaining informed consent.
    FVC ≤50% predicted or MIP > -60 cm H2O.
    ALS Functional Rating Scale (ALSFRS-R) [Cedarbaum1999] score on day of screening of ≥2 on items for speech, swallowing, and salivation. These items are indicators of bulbar dysfunction, which limits the reliability of PFT administration.
    Cognitive impairment, as judged by the ALS clinic neurologist, that prevents participation in the study.

    Caregivers: None

    Respiratory Therapists: None

  • Site Contact Information

Understanding Communication and Cognitive Impairments in Neurodegenerative Disorders

Study Purpose:

The goal is to improve the fundamental knowledge about articulatory motor performance in people with Lou Gehrig's disease (also known as ALS) and Parkinson's disease (PD), in order to develop more sensitive assessments for progressive speech loss, which may lead to the improved timing of speech therapies.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Healthy Volunteer, Parkinson Disease

Study Type:

Interventional Trial

Study Category:

Behavioral

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Mili Kuruvilla-Dugdale, PhD, University of Missouri-Columbia

Clinicaltrials.gov ID (11 digit #):

NCT03613038

Neals Affiliated?

No

Coordinating Center Contact Information

University of Missouri-Columbia
Mili Kuruvilla-Dugdale / .(JavaScript must be enabled to view this email address) / 573-882-2910
.(JavaScript must be enabled to view this email address) Columbia, Missouri 65211 United States

Full Study Summary:

The long-term goal is to optimize dysarthria assessment by improving the early detection and tracking of articulatory performance in progressive dysarthrias. The short-term goal of the proposed cross-sectional study is to focus on ALS and PD and quantify articulatory kinematic performance as a function of phonetic complexity, which is experimentally manipulated based on theoretical principles of speech motor development. The research strategy is to use 3D electromagnetic articulography to examine phonetic complexity effects of single word stimuli at the articulatory kinematic level in 15 talkers each with preclinical, mild, and moderate dysarthria, relative to 45 controls. The central hypothesis is that as dysarthria severity increases the discrepancy in articulatory performance, indexed by movement speed, distance, coordination, and variability, between people with dysarthria and typical controls will significantly increase at a lower phonetic complexity level.

Study Sponsor:

University of Missouri-Columbia

Participant Duration:

Estimated Enrollment:

150

Estimated Study Start Date:

07/15/2017

Estimated Study Completion Date:

07/14/2020

Posting Last Modified Date:

08/06/2018

Date Study Added to alsconsortium.org:

08/06/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    19

    Maximum Age:

    90

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    May or may not have a neurological impairment.
    Age range of 19-90 years.
    Male or female.
    Provide written consent before any study specific procedures are performed.
    Have ability to comply with basic instructions.
    Monolingual English speaker.
    Have ability to partake in a 90 minute data collection.

    Exclusion Criteria:

    Any speech, language, cognition, or hearing impairment prior to diagnosis of a neurodegenerative disease.
    Anyone not appropriate for study participation, as deemed by the principal investigator.

  • Site Contact Information

    University of Kansas Medical Center
    Richard Barohn, MD / .(JavaScript must be enabled to view this email address) / 913-558-6970
    Andra Lahner, RN / .(JavaScript must be enabled to view this email address) / 913-588-0972
    Fairway, Kansas 66205
    United States

    University of Missouri-Columbia
    Mili Kuruvilla-Dugdale, PhD / .(JavaScript must be enabled to view this email address) / 573-882-2910
    Columbia, Missouri 65211
    United States

Safety and Efficacy of Ranolazine for the Treatment of Amyotrophic Lateral Sclerosis

Study Purpose:

The purpose of this research study is to evaluate the safety and effectiveness of Ranolazine, and how well it is tolerated in patients with Amyotrophic Lateral Sclerosis (ALS). Ranolazine is an FDA approved drug that is used for decreasing chest pain.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Jeffrey Statland, MD, University of Kansas Medical Center

Clinicaltrials.gov ID (11 digit #):

NCT03472950

Neals Affiliated?

No

Coordinating Center Contact Information

University of Kansas Medical Center
Laura Herbelin / .(JavaScript must be enabled to view this email address) / 913-588-5095
.(JavaScript must be enabled to view this email address) Kansas City, Kansas 66160 United States

Full Study Summary:

Amyotrophic Lateral Sclerosis (ALS) is a progressive debilitating and fatal neurodegenerative disease involving the motor neurons in the primary motor cortex, corticospinal tracts, brainstem and spinal cord with 5,000 newly diagnosed patients per year in the USA. There is a pressing need for additional therapies, as the only two FDA-approved drugs for ALS, riluzole and edaravone, showed prolongation of median survival of only two to three months and only a modest benefit in daily functioning, respectively. The ability to identify FDA approved drugs which can be repurposed to ALS, and which may slow disease progression, alleviate symptoms, or prolong survival will have an immediate positive impact of the lives of patients with ALS and their family members. Hypothesis: Ranolazine, an FDA approved drug for angina which inhibits the late Na+ current and intracellular Ca2+ accumulation may be neuroprotective in ALS by reducing neuronal hyperexcitability, may slow disease progression and reduce cramp frequency.

Study Sponsor:

University of Kansas Medical Center

Participant Duration:

Estimated Enrollment:

20

Estimated Study Start Date:

06/11/2018

Estimated Study Completion Date:

06/11/2019

Posting Last Modified Date:

08/06/2018

Date Study Added to alsconsortium.org:

08/06/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Patients with clinically definite, probable, laboratory supported probable, or possible ALS per revised El Escorial criteria
    Cramp frequency greater than 4 cramps per week during 2 week run in
    ALS functional rating scale-revised (ALSFRS-R) score of greater than 24
    Able to lie on back for study procedures

    Exclusion Criteria:

    Tracheostomy invasive ventilation, or use of non-invasive ventilation greater than 12 hours per day
    Pregnant or lactating
    Participation in a prior experimental drug trial less than 30 days prior to screening
    Patients taking ranolazine
    Patients taking medications which are contraindicated for use with ranolazine such as strong CYP3 inhibitors (ketoconazole, clarithromycin, nelfinavir), and CYP3 inducers (rifampin, phenobarbital)
    Patients with clinically significant medical comorbidities (hepatic, renal, cardiac, etc)
    Patients with baseline QT interval prolongation on Electrocardiography (ECG)
    Patients pre-disposed to secondary QT prolongation for other health conditions like family history of congenital long QT syndrome, heart failure, bradycardia, or cardiomyopathies

  • Site Contact Information

    University of Kansas Medical Center
    Kansas City, Kansas 66160
    United States

Tolerability and Efficacy of L-Serine in Patients With Amyotrophic Lateral Sclerosis: A Phase IIa Study

Study Purpose:

The purpose of this study is to determine the tolerability of L-Serine oral doses for ALS patients and assess preliminary indications of efficacy

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Elijah W Stommel, MD,PHD, Dartmouth-Htichcock Medical Center

Clinicaltrials.gov ID (11 digit #):

NCT03580616

Neals Affiliated?

No

Coordinating Center Contact Information

Dartmouth-Htichcock Medical Center
Catherine L Andrews, RN, CCRN / .(JavaScript must be enabled to view this email address) / 603-650-4633
.(JavaScript must be enabled to view this email address) New Hampshire United States

Full Study Summary:

All patients will receive the same dose of the study treatment over 6 months. For each participant the study will last approximately one year with follow up visits after the treatment period of 6 months is completed. The visits will include blood draws, electromyography (EMG), vital sign checks, neurological and physical exams, pulmonary testing with forced vital capacity (FVC), and questionnaires.

Study Sponsor:

Elijah W. Stommel

Participant Duration:

Estimated Enrollment:

50

Estimated Study Start Date:

07/31/2018

Estimated Study Completion Date:

06/30/2022

Posting Last Modified Date:

12/14/2018

Date Study Added to alsconsortium.org:

07/10/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    60

    Time since Symptom Onset:

    Time since Diagnosis:

    <36 months

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Diagnosis of probable or definite ALS
    ALSFRS-R score >25 and FVC score ≥ 60% predicted
    If currently taking Riluzole and Edaravone/Radicava for 3 months prior to Baseline/Screening or not at all

    Exclusion Criteria:

    Diagnosis of probable or definite ALS more than 3 years prior to study enrollment
    Diagnosis or previous history of ischemic stroke, brain tumor, uncontrolled diabetes, renal insufficiency, or severe hypertension.
    Diagnosis or previous history of peripheral neuropathy of any other comorbid progressive neurodegenerative disease such as Alzheimer's disease, Parkinson's disease, Lewy Body disease, Pick's disease, Huntington's disease, Progressive Supranuclear palsy. ALS patients diagnosed with frontotemporal dementia will not be excluded from this study.
    Undergoing any chemotherapy or radiation therapy for any cancer
    Any medical condition likely to interfere with the conduct of the trial or survival of the patient during this study period
    Pregnant women or women who are breast feeding
    Has taken L-Serine supplement within 30 days prior to start of study drug

  • Site Contact Information

    Dartmouth-Hitchcock Medical Center
    Catherine L Andrews, RN / .(JavaScript must be enabled to view this email address) / 603-650-4633
    Lebanon, New Hampshire 03756
    United States

Open-Label Study to Evaluate Safety, Tolerability and Pharmacokinetics of Multiple Doses of BHV-0223 in Subjects With Amyotrophic Lateral Sclerosis

Study Purpose:

Phase 1, open-label study of BHV-0223 in ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03520517

Neals Affiliated?

No

Coordinating Center Contact Information


Robert Berman, MD / .(JavaScript must be enabled to view this email address) / 203-404-0410
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

This is a phase 1, open-label, single arm study to evaluate the safety, tolerability and pharmacokinetics of multiple doses of BHV-0223 in subjects with ALS.

Study Sponsor:

Biohaven Pharmaceutical Holding Company Ltd.

Participant Duration:

Estimated Enrollment:

20

Estimated Study Start Date:

02/02/2018

Estimated Study Completion Date:

07/31/2018

Posting Last Modified Date:

08/06/2018

Date Study Added to alsconsortium.org:

05/29/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Subjects with diagnosed ALS by the revised El Escorial diagnostic criteria, including laboratory supported probable, probable, or definite ALS;
    Subjects who have never taken riluzole tablets, OR Subjects who previously took riluzole tablets but discontinued at least 1 month prior to the screening visit. Subjects must not have had ALT or AST > 5 x ULN while taking riluzole tablets, or any other clinically significant tolerability issues (e.g., hypersensitivity reactions) in the judgement of the investigator;
    Subjects determined by the investigator to be medically stable;
    Subjects determined by the investigator to be willing and physically able to complete the study as designed, with or without caregiver assistance.

    Exclusion Criteria:

    Target Disease Exceptions

    Medical History Exceptions

    • Subject is known to have a current diagnosis of acute or chronic viral hepatitis;
    • Subject is known to have any other acute or chronic liver disease that is clinically significant in the investigator's judgment;
    • Subject has a history of a clinically significant medical condition that would interfere with the subject's ability to comply with study instructions, would place the subject at increased risk, or might confound the interpretation of the study results in the investigator's judgment;
    • Any other sound medical, psychiatric and/or social reason in the investigator's judgment;

    Physical and Laboratory Test Findings

    • Positive urine pregnancy test in WOCBP at screening;
    • Subject has evidence of organ dysfunction or any clinically significant deviation from normal in physical examination, vital signs, or other determinations beyond what is consistent with the target population, in the investigator's judgment;
    • Subject has liver function testing abnormalities (ALT, AST , or total bilirubin) that are > 1 x ULN;

    Other Exclusions a. Subjects who are unable to be compliant with the visit schedule or protocol procedures.

  • Site Contact Information

    Holy Cross Neuroscience Research Institute
    Franco A Salas, RMA / .(JavaScript must be enabled to view this email address) / 954-229-7965
    Fort Lauderdale, Florida 33334
    United States

    Somnos/Neurology Associates Clinical Research
    Desi Eschiti, LPN / .(JavaScript must be enabled to view this email address) / 402-486-3410
    Kelli Ferguson / .(JavaScript must be enabled to view this email address) / 402-486-3410
    Lincoln, Nebraska 68506
    United States

    Neurosciences Institute, Neurology
    Lisa H Ranziger, RN / .(JavaScript must be enabled to view this email address) / 704-446-0803
    Charlotte, North Carolina 28207
    United States

    Wesley Neurology Clinic
    Cindy Benzel, LPN / .(JavaScript must be enabled to view this email address) / 901-752-3784
    Cordova, Tennessee 38018
    United States

    Texas Neurology
    Todd Morgan / .(JavaScript must be enabled to view this email address) / 214-827-3610
    Dallas, Texas 75214
    United States

Noninvasive Cervical Electrical Stimulation for ALS: Mechanistic and Safety Study

Study Purpose:

Veterans are at higher risk than non-Veterans of falling ill with amyotrophic lateral sclerosis (ALS). ALS causes degeneration of motor neurons in both the brain and the spinal cord. Evidence from studies in people with spinal cord injury suggests that activating spared nerve circuits with electromagnetic stimulation improves nerve transmission.

With this goal, the investigators have developed a novel method of noninvasive cervical (neck) electrical stimulation (CES). In this study, the investigators will investigate CES for its potential to strengthen nerve circuits to the hands in ALS.

To the investigators' knowledge, electrical spinal stimulation for ALS has never been tested previously. This study will be performed in two stages: First, basic experiments will be performed to better understand how CES interacts with other types of electrical and magnetic stimulations over the brain and peripheral nerves. Second, experiments will be performed to determine the types of CES that can facilitate active arm and hand movements.

These experiments will improve understanding of electrical stimulation in ALS, and may set the table for future treatments.

Both United States Veterans and non-Veterans are eligible to participate in this study.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Device

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Noam Y. Harel, MDm PhD, VA Office of Research and Development

Clinicaltrials.gov ID (11 digit #):

NCT03411863

Neals Affiliated?

No

Coordinating Center Contact Information


Sana Saeed, BS / .(JavaScript must be enabled to view this email address) / 718-584-9000 ext 3123
Noam Y Harel, MD PhD / .(JavaScript must be enabled to view this email address) / 718-584-9000 ext 1742
Bronx, New York 10468 United States

Full Study Summary:

Amyotrophic lateral sclerosis (ALS) reduces connections between the cortical motor neurons that initiate movement and the spinal motor neurons that direct muscles to execute movement. This situation shares many key features with incomplete spinal cord injury (SCI). Accumulating evidence in SCI suggests that externally activating spared nerve circuits with electromagnetic stimulation augments neural transmission.

With this goal, the investigators developed a novel method of noninvasive cervical electrical stimulation (CES). CES activates multiple muscles on both upper limbs by triggering afferent sensory or efferent motor nerve roots depending on stimulus intensity. This study will investigate CES for its potential to strengthen residual circuits to the hands in ALS.

To the investigators' knowledge, electrical spinal stimulation for ALS has never been tested or applied previously. Therefore, a pilot study is essential. This study will be performed in two stages:

Map CES circuit and synaptic targets: The experiments share a common structure comprising conditioning and test stimuli delivered at a range of intensities, sites, and interstimulus intervals.
Determine parameters for combining CES with volitional movement: volitional limb movements depend on the same corticospinal and motor neuron circuits as those activated by TMS and F-waves. Since preliminary data shows that subthreshold CES facilitates TMS responses, CES may also be able to facilitate volitional limb movements.

Successful completion of these experiments will: mechanistically elucidate CES circuit interactions; investigate the potential for CES to enhance concurrent volitional muscle activation; and establish CES as safe and feasible in the ALS population. Given the limited treatment options for ALS, any amount of progress would represent a meaningful step forward. Moreover, results of this pilot study could lead to direct translation for lasting clinical benefit by combining repetitive subthreshold CES with repetitive task-oriented physical exercise training in subsequent studies. CES would be compatible with other interventions, including medications and cell-based treatments.

Study Sponsor:

VA Office of Research and Development

Participant Duration:

Estimated Enrollment:

50

Estimated Study Start Date:

01/04/2018

Estimated Study Completion Date:

12/31/2019

Posting Last Modified Date:

05/29/2020

Date Study Added to alsconsortium.org:

05/29/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    21

    Maximum Age:

    65

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Age between 21 and 65 years
    Diagnosis of probable or definite ALS (or non-disabled volunteer)
    Incomplete weakness of left or right wrist or hand muscles:

    • score of 2, 3, or 4 (out of 5) on manual muscle testing of wrist extension
    • wrist flexion
    • finger extension
    • finger flexion
    • or finger abduction

    Detectable F-wave responses of the left or right APB to median nerve stimulation and/or ADM to ulnar nerve stimulation
    US Veteran or non-Veteran

    Exclusion Criteria:

    History of other serious injury or disease of central or peripheral nervous system
    History of seizures
    Ventilator dependence or patent tracheostomy site
    Use of medications that significantly lower seizure threshold
    History of head trauma with evidence of brain contusion or hemorrhage or depressed skull fracture on prior imaging

    History of implanted:

    • brain/spine/nerve stimulators
    • aneurysm clips
    • ferromagnetic metallic implants
    • or cardiac pacemaker/defibrillator

    Significant coronary artery or cardiac conduction disease
    History of bipolar disorder or suicide attempt or active psychosis
    Heavy alcohol consumption (> equivalent of 5 oz of liquor) within previous 48 hours
    Open skin lesions over the face, neck, shoulders, or arms
    Pregnancy
    Unsuitable for study participation as determined by study physician

  • Site Contact Information

    James J. Peters VA Medical Center
    Sana Saeed, BS / .(JavaScript must be enabled to view this email address) / 718-584-9000 ext 3123
    Bronx, New York 10468
    United States

An Open Label Study to Evaluate the Safety and Efficacy of FLX-787-ODT for Treatment of Fasciculations in the Tongue and One Appendicular Muscle in Adult Subjects With Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

The FLX-787-107 study will determine how well FLX-787-ODT works to reduce fasciculations in patients with Amyotrophic Lateral Sclerosis (ALS). The study will measure how often fasciculations occur, if tongue and muscle strength, speech, and swallowing are affected, and monitor any side effects that might develop while taking the investigational product. Participants will be assessed before and after taking a single dose of FLX-787-ODT. Approximately 15 people will take part in this study at one center in the United States. Participants will be in the study for a single clinic visit and receive a telephone call 7 days later to monitor for side effects.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03334786

Neals Affiliated?

No

Coordinating Center Contact Information


David Golod, PhD / .(JavaScript must be enabled to view this email address) / 617-721-1988
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

Study Sponsor:

Flex Pharma, Inc.

Participant Duration:

Estimated Enrollment:

15

Estimated Study Start Date:

04/05/2018

Estimated Study Completion Date:

08/31/2018

Posting Last Modified Date:

09/11/2018

Date Study Added to alsconsortium.org:

04/10/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Documented diagnosis of ALS diagnosis of less than 5 years.
    Greater than 6 fasciculations per minute noted at least in the tongue by clinical, ultrasound, or EMG evaluation.
    Normal oral cavity exam at screening.

    Exclusion Criteria:

    Presence of clinically significant or unstable condition that would result in an increased risk of study participation or difficulty in interpretation of the study results.
    Tremor or other movement disorder that would interfere with recording.
    Presence of major gastrointestinal disorders, such as inflammatory bowel disease, diverticulitis, active peptic ulcer disease, or significant gastroesophageal reflux disease (i.e., not well-controlled on antacids or proton pump inhibitors), or oral or esophageal lesions/ulcers.
    Presence of laryngospasm or significant swallowing problems.
    Inability to tolerate a spicy sensation in the mouth or stomach.
    Actively using illicit drugs or history of chronic substance abuse within the past year prior to screening, including abuse of alcohol.
    Participated in a clinical study (except natural history studies without administration of an investigational product) within 30 days prior to screening.
    Pregnant, breastfeeding, or planning to become pregnant.
    Blood pressure of ≥160 mmHg systolic and/or ≥100 mmHg diastolic.
    Clinically significant abnormalities in laboratory findings (including screening complete electrolyte panel, complete blood count, liver function tests).

  • Site Contact Information

    Beth Israel Deaconess Medical Center
    Hilda Guttierez / .(JavaScript must be enabled to view this email address) / 617-667-8130
    Maria Martucci / .(JavaScript must be enabled to view this email address) / 617-667-8130
    Boston, Massachusetts 02215
    United States

A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis

Study Purpose:

A multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotropic lateral sclerosis (ALS)

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase III

Study Chair(s)/Principal Investigator(s):

Michael Benatar, MD PhD University of Miami

Clinicaltrials.gov ID (11 digit #):

NCT03491462

Neals Affiliated?

No

Coordinating Center Contact Information


Requests at Orphazyme A/S / .(JavaScript must be enabled to view this email address) / +45 51 71 68 37
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Orphazyme

Participant Duration:

Screening of up to 4 weeks Treatment of up to 76 weeks

Estimated Enrollment:

231

Estimated Study Start Date:

07/31/2018

Estimated Study Completion Date:

12/31/2020

Posting Last Modified Date:

05/02/2019

Date Study Added to alsconsortium.org:

04/10/2018
  • More Information
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    80

    Time since Symptom Onset:

    <18 months

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    • Subject meets revised El Escorial criteria for clinically possible, clinically probable / Clinically probable ALS laboratory-supported or clinically definite ALS, or familial ALS
    • 18 months or less since first appearance of weakness
    • ALSFRS-R equal to or above 35 and erect SVC% predicted equal to or above 80% at screening

    Exclusion Criteria:

    • Tracheostomy or use of non-invasive ventilation for more than 2 hours during waking hours at the time of screening or baseline
    • pregnant or breast-feeding
    • current or anticipated use of diaphragmatic pacing
    • Any other relevant medically significant condition which could present risk to the subject or interfere with the assessment of safety or has an increased risk of causing death during the trial

  • Site Contact Information

    Barrow Neurological Institute
    Nicole Turcotte / .(JavaScript must be enabled to view this email address) / 602-406-4775
    Phoenix , Arizona 85013
    United States

    HonorHealth Neurology
    Mackenzie Steinbach / .(JavaScript must be enabled to view this email address) / 480-882-4916
    Phoenix, Arizona 85018
    United States

    UC Irvine Health ALS and Neuromuscular Center
    Ivonne Turner / .(JavaScript must be enabled to view this email address) / 714-456-7760
    Veronica Martin / .(JavaScript must be enabled to view this email address) / 714-456-7760
    Orange, California 92868
    United States

    University of Miami
    Maria Elena Paredes / .(JavaScript must be enabled to view this email address) / 305-243-7336
    Miami, Florida 33136
    United States

    The University of Iowa
    Jeri Sieren / .(JavaScript must be enabled to view this email address) / 319-356-8744
    Iowa City , Iowa 52242
    United States

    University of Kansas Medical Center
    Alyssa Lackey / .(JavaScript must be enabled to view this email address) / 913-945-9942
    Kansas City, Kansas 66160
    United States

    Hospital for Special Surgery
    William Mark Richardson / .(JavaScript must be enabled to view this email address) / 646-797-8657
    New York, New York 10021
    United States

    Providence Brain & Spine Institute
    Arlena Cummings / .(JavaScript must be enabled to view this email address) / 503-962-1171
    Portland , Oregon 97213
    United States

    University of Pensylvania
    Kelly Almasy / .(JavaScript must be enabled to view this email address) / 215-829-5041
    Philadelphia, Pennsylvania 19107
    United States

    University of Texas Southwestern Medical Center
    Amruta Joshi / .(JavaScript must be enabled to view this email address) / 213-648-9380
    Dallas, Texas 75390
    United States

    University of Virginia Health System
    Mary Wagoner / .(JavaScript must be enabled to view this email address) / 434-924-5541
    Charlottesville, Virginia 22908
    United States

    Catholic University Leuven
    Leuven, 3000
    Belgium

    London Health Sciences Centre
    Christine Piechowicz / .(JavaScript must be enabled to view this email address) / 519-685-8500 ext 34858
    London, Ontario N6A 5A5
    Canada

    Sunnybrook Health Sciences Centre
    Liane Phung / .(JavaScript must be enabled to view this email address) / 16-480-6100 ext 87561
    Toronto, Ontario M4N 3M5
    Canada

    Montreal Neurological Institute and Hospital
    Montréal, Quebec H3A 2B4
    Canada

    Neuroligisk afdeling Aarhus Universitets Hospital
    Charlotte Odgaard / .(JavaScript must be enabled to view this email address) / Aarhus, 8000
    Denmark

    Neurologisk afdeling Bispebjerg Hospital
    Copenhagen, 2400
    Denmark

    Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac
    Montpellier, 34295
    France

    Groupe Hospitalier Pitie-Salpetriere
    Paris, 75013
    France

    Charite - Universitaetsmedizin Berlin
    Birgit Koch / .(JavaScript must be enabled to view this email address) / +49 30 450 560028
    Berlin, 13353
    Germany

    Medizinische Hochschule Hannover (MHH)
    Chantal Fischer / .(JavaScript must be enabled to view this email address) / +49 (0) 511 532-8333
    Hannover, 30625
    Germany

    Universitaetsklinikum Ulm
    Johannes Dorst / .(JavaScript must be enabled to view this email address) / +49- 731 177 5285
    Ulrike Weiland / .(JavaScript must be enabled to view this email address) / +49- 731 177 5285
    Ulm, 89081
    Germany

    Instituti Clinica Scientifici Maugeri
    Milano, 20138
    Italy

    Azienda Ospedaliero Universitaria (AUO) di Torino
    Torino, 10126
    Italy

    University Medical Center Utrecht
    Tommy Bunte / .(JavaScript must be enabled to view this email address) / 31887573110
    Utrecht , 3584CX
    Netherlands

    Centrum Medyczne NeuroProtect
    Marta Biel / .(JavaScript must be enabled to view this email address) / +48 22 468 15 48
    Warsaw, 01-684
    Poland

    Citi Clinic
    Adam R Ronert / .(JavaScript must be enabled to view this email address) / 48607616559
    Warsaw, 02-473
    Poland

    Hospital Universitario Vall d'Hebron
    Ana Canovas / .(JavaScript must be enabled to view this email address) / 0034 932746000 ext 2780
    Barcelona, 08035
    Spain

    Hospital Carlos III - Hospital Universitario La Paz
    Saul Marin / .(JavaScript must be enabled to view this email address) / +34 686061101
    Madrid, 28046
    Spain

    Umeå University Hospital
    Erica Stenberg / .(JavaScript must be enabled to view this email address) / +46 72548 74 10
    Umeå , 90737
    Sweden

    Kantonsspital St.Gallen
    Christoph Neuwirth / .(JavaScript must be enabled to view this email address) / +41 714943581
    Saint Gallen, 9007
    Switzerland

    Leonard Wolfson Experimental Neurology Centre
    Anna Bellin / .(JavaScript must be enabled to view this email address) / London , WC1N 3BG
    United Kingdom

Open Label Extension Study of AMX0035 in Patients With ALS (CENTAUR-OLE)

Study Purpose:

This study will provide extended access to patients and assess longer-term outcomes on patients who have completed the Centaur study.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03488524

Neals Affiliated?

No

Coordinating Center Contact Information

Massachusetts General Hospital
Andrea Ariza / .(JavaScript must be enabled to view this email address) / 617-724-3871
.(JavaScript must be enabled to view this email address) Boston, Massachusetts 02114 United States

Full Study Summary:

The Centaur Open Label Extension Study (CENTAUR-OLE) is designed to provide longer term access to AMX0035 for patients with ALS who participated in the CENTAUR study. The study will assess longer term safety and therapeutic potential of AMX0035.

Study Sponsor:

Amylyx Pharmaceuticals Inc.

Participant Duration:

Estimated Enrollment:

132

Estimated Study Start Date:

03/29/2018

Estimated Study Completion Date:

01/01/2020

Posting Last Modified Date:

09/17/2019

Date Study Added to alsconsortium.org:

04/10/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    N/A

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Completion of all visits in the randomized, double blind AMX0035 study. Subjects that receive tracheostomy or PAV during the course of the main study will still be followed as ITT until the week 24 visit before enrollment in the OLE.
    Must enroll in the OLE within 28 days of the Week 24 visit of the main study.
    Signed informed consent to enter the open label extension phase.

    Exclusion Criteria:

    Discontinued study drug prematurely in the double-blind phase of the study for reasons other than tracheostomy or PAV.
    Exposure to or anticipated requirement for any disallowed medication listed below.
    Any ongoing adverse events that in the opinion of the Site Investigator are clear contraindications to the study drug.
    Unstable cardiac or other life-threatening disease emergent during the randomized, double blind study
    Any major medical condition that in the opinion of the Site Investigator would interfere with the study and place the subject at increased risk.

  • Site Contact Information

    Massachusetts General Hospital
    Boston, Massachusetts 02114
    United States

Clinical Procedures to Support Research (CAPTURE)

Study Purpose:

The purpose of the Clinical Procedures To Support Research (CAPTURE) study is to utilize information collected in the medical record to learn more about a disease called amyotrophic lateral sclerosis (ALS) and related disorders.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Primary Lateral Sclerosis (PLS)

Study Type:

Observational Study

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Michael Benatar, University of Miami

Clinicaltrials.gov ID (11 digit #):

NCT03489278

Neals Affiliated?

No

Coordinating Center Contact Information

University of Miami
Michael Benatar, MBChB, MS, DPhil / .(JavaScript must be enabled to view this email address) / 844-837-1031
.(JavaScript must be enabled to view this email address) Miami, Florida 33136 United States

Full Study Summary:

The study will consent patients with ALS or related disorders that are receiving care at a clinical center in the CReATe consortium that uses Epic as its electronic health record (EHR) system. The study aims to systematically gather a clinical dataset through the EHR using a standardized approach to characterize the natural history of ALS and related diseases.
 

Study Sponsor:

University of Miami

Participant Duration:

Estimated Enrollment:

1200

Estimated Study Start Date:

02/15/2018

Estimated Study Completion Date:

01/31/2021

Posting Last Modified Date:

08/12/2019

Date Study Added to alsconsortium.org:

04/10/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Diagnosis of ALS or a related disorder (e.g. primary lateral sclerosis, progressive muscular atrophy).
    Receiving care at a clinical center that uses Epic as its EHR.
    Able and willing to provide informed consent (or informed consent obtainable from a designated proxy).

    Exclusion Criteria:

    Inability to understand English and/or Spanish

  • Site Contact Information

    California Pacific Medical Center
    Jennifer Milan / .(JavaScript must be enabled to view this email address) / 415-600-5764
    San Francisco, California 94115
    United States

    University of Miami
    Sumaira Hussain / .(JavaScript must be enabled to view this email address) / 844-837-1031
    Miami, Florida 33136
    United States

Glial Activation Measured by PBR28-PET in People with Neurogenerative Disease

Study Purpose:

The purpose of the study is to learn more about inflammation in the brains of people with Motor Neuron Disease (MND) using combined Magnetic Resonance Imaging (MRI) and Positron Emission Tomography (PET).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Primary Lateral Sclerosis (PLS), Hereditary Spastic Paraplegia (HSP), Healthy Volunteer, Healthy Volunteer with a Family History of ALS

Study Type:

Observational Study

Study Category:

Biomarkers/Imaging

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Suma Babu, Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):

N/A

Neals Affiliated?

No

Coordinating Center Contact Information

Massachusetts General Hospital
Catherine Cebulla / .(JavaScript must be enabled to view this email address) / 617-643 6252
.(JavaScript must be enabled to view this email address) 165 Cambridge St.
Suite 600
Boston, Massachusetts 02114 United States

Full Study Summary:

Massachusetts General Hospital is seeking individuals to participate in a research study enrolling under the direction of Nazem Atassi, MD, MMSc. The purpose of the study is to learn more about inflammation in the brains of people with Motor Neuron Disease (MND) using combined Magnetic Resonance Imaging (MRI) and Positron Emission Tomography (PET). Our study will examine whether particular cells, called microglia, are hyperactive in the nervous system of people with MND, such as those individuals with Amyotrophic Lateral Sclerosis (ALS). This information could help improve the diagnosis and development of treatments for other patients with MND in the future.

This study aims to enroll the following cohorts of individuals:

  • Individuals with a diagnosis of MND: ALS, Primary Lateral Sclerosis (PLS), Hereditary Spastic Paraplegia (HSP)
  • Healthy individuals who are known carriers of an ALS gene such as chromosome 9 open reading frame 72 (C9orf72) or superoxide dismutase 1 (SOD1)
  • Individuals with a diagnosis of Frontotemporal Dementia (FTD)
  • Healthy individuals without any neurodegenerative disease and no family history of MND

Study participation involves two visits to MGH over a maximum of three months (these visits can also be completed in one day). During the visit(s), participants will undergo a 90-minute MR-PET scan. Participants with MND will also undergo a breathing test, muscle test and questionnaires, and may choose to participate in an optional lumbar puncture (not to be completed on the same day as the scan). Individuals with MND may elect to participate for up to four years in up to 9 optional follow-up scans approximately every three months for the first year and every six months for the following three years. Individuals who do not have MND but are known ALS gene carriers may elect to participate in up to 7 optional follow-up scans approximately every six months for up to four years.

Participants must be between the ages of 18 and 80, be medically safe to undergo an MRI scan (i.e., no metallic particles in the body), and be able to safely lie flat for at least 90 minutes. Additionally, participants cannot be taking any immunosuppressive medications or have a diaphragm pacing system and cannot have a diagnosis of Parkinson’s disease, Alzheimer’s disease, unstable psychiatric disease, or renal failure.

All participants will be reimbursed for parking and receive compensation of $150 upon completion of each MR-PET scan. There will be additional compensation of $100 for each lumbar puncture completed by individuals with MND.

Study Sponsor:

Muscular Dystrophy Association, NIH, Voyager

Participant Duration:

Study participation involves two visits to MGH over a maximum of three months (these visits can also be completed in one day). During the visit(s), participants will undergo a 90-minute MR-PET scan. Participants with MND will also undergo a breathing test, muscle test and questionnaires, and may choose to participate in an optional lumbar puncture (not to be completed on the same day as the scan). Individuals with MND may elect to participate for up to four years in up to 9 optional follow-up scans approximately every three months for the first year and every six months for the following three years. Individuals who do not have MND but are known ALS gene carriers may elect to participate in up to 7 optional follow-up scans approximately every six months for up to four years. 

Estimated Enrollment:

300

Estimated Study Start Date:

02/28/2018

Estimated Study Completion Date:

Posting Last Modified Date:

02/05/2019

Date Study Added to alsconsortium.org:

04/10/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


  • Site Contact Information

    Massachusetts General Hospital
    Catherine Cebulla / .(JavaScript must be enabled to view this email address) / 617-643 6252
    165 Cambridge St.
    Boston, Massachusetts 02114
    United States

A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

This is a study of transplantation of Astrocytes derived from human embryonic stem cells, in patients with Amyotrophic Lateral Sclerosis (ALS).

There will be no change in the routine ALS treatment of the patients enrolled into the study. Treatment will be administered in addition to the appropriate standard of care treatment.

The study hypothesis is that transplantation of Astrocyte(AstroRx) cells can compensate for the malfunctioning of patients' own astrocytes by restoring physiological capabilities like the reuptake of excessive glutamate, reducing oxidative stress, reducing other toxic compounds, as well as by secreting different neuroprotective factors

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Stem Cell

Study Status:

Enrolling

Phase:

Phase I ,Phase II

Study Chair(s)/Principal Investigator(s):

Marc Gotkine, MD, Hadassah Ein Kerem Medical Center

Clinicaltrials.gov ID (11 digit #):

NCT03482050

Neals Affiliated?

No

Coordinating Center Contact Information

Hadassah Ein Kerem Medical Center
Yael Feinsod / .(JavaScript must be enabled to view this email address) / +972-2-6779372
.(JavaScript must be enabled to view this email address) Jerusalem, Israel

Full Study Summary:

Study Sponsor:

Kadimastem

Participant Duration:

Estimated Enrollment:

21

Estimated Study Start Date:

03/31/2018

Estimated Study Completion Date:

07/31/2020

Posting Last Modified Date:

05/29/2018

Date Study Added to alsconsortium.org:

04/04/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    70

    Min Vital Capacity (% predicted normal):

    70

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    <24 months

    Can participants use Riluzole?

    Yes


    Main Inclusion Criteria:

    El Escorial criteria for probable or definite ALS
    Males and and non pregnant females between 18 and 70 years of age
    Patients with an ALS-FRS-R score of at least 30 with an ALS diagnosis of two years or less
    No history of active psychiatric disorder.
    Patient has a good understanding of the study and nature of the procedure
    Patient provides written informed consent prior to any study procedure
    Patients should either be on a stable dose of Riluzole and/or Radicava® (if applicable) for 3 months, or not be treated with Riluzole or Radicava®
    Patient is medically able to tolerate immunosuppression regimen
    Presence of a willing and able caregiver who understands the need to attend all follow-up visits, even if mobility declines

    Main Exclusion Criteria:

    Patient has a past infection or a positive test for HBV,HCV or HIV
    Patient is in need of respiratory support
    Patient has a lower than 10/12 in ALS-FRS-R respiratory parameters or below 70% of predicted slow vital capacity (SVC)
    Patient has renal failure
    Patient has impaired hepatic function
    Patient has a Body Mass Index (BMI) of <18.5 or > 30
    Patient suffers from significant cardiac disease, diabetes, autoimmune diseases, chronic severe infection, malignant disease or any other disease or condition that may risk the patient or interfere with the ability to interpret the study results
    Patient has systemic inflammation or active infections
    Patient has been treated previously with any stem cell therapy
    Current use of immunosuppressant medication or use of such medication within 6 weeks of Screening visit (Visit 0)
    Patient has participated in another clinical treatment trial or received other experimental medications outside of a clinical trial within 1 month prior to start of this study
    Any known immunodeficiency syndrome
    Any concomitant disease or condition limiting patient safety to participate

  • Site Contact Information

    Hadassah Ein Kerem Medical Center
    Yael Feinsod / .(JavaScript must be enabled to view this email address) / +972-2-6779372
    Jerusalem,
    Israel

A Multi-Center, Open Label Study to Assess the Safety and Tolerability of Riluzole Oral Soluble Film in Subjects With Amyotrophic Lateral Sclerosis Over 12 Weeks of Twice Daily Treatment

Study Purpose:

The primary objective of this study is to assess the safety and tolerability, with emphasis on the oral cavity, of ROSF (containing riluzole 50mg) in subjects with amyotrophic lateral sclerosis (ALS) administered twice daily for 12 weeks. Secondary objectives include (1) to record the subject's assessment of any difficulty taking riluzole administered as ROSF and any difficulty taking riluzole in the tablet formulation and (2) to record the relative preference, if any, of subjects and caretakers, for riluzole administered as ROSF vs. the riluzole tablet.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Emily Plowman, PhD, CCC-SLP University of Florida
James Wymer, MD, PhD University of Florida

Clinicaltrials.gov ID (11 digit #):

NCT03457753

Neals Affiliated?

No

Coordinating Center Contact Information


Carla Buan, BS Pharmacy / .(JavaScript must be enabled to view this email address) / 908-941-1896
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

The total study participant time is expected to be approximately 14 weeks from time of screening to completion of study. Subjects will be instructed on the use of ROSF and receive the first dose of ROSF under supervision of the investigator during Visit 1.Subjects will then continue on ROSF 50mg twice daily for 12 weeks.

Study Sponsor:

Aquestive Therapeutics

Participant Duration:

14 weeks

Estimated Enrollment:

25

Estimated Study Start Date:

03/31/2018

Estimated Study Completion Date:

10/31/2018

Posting Last Modified Date:

12/14/2018

Date Study Added to alsconsortium.org:

03/21/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Male or female subjects, between 18-80 years of age, inclusive.
    Subjects having a diagnosis of probable or definite ALS in accordance with the Revisited El-Escorial Criteria.
    Subjects must have no known allergy to riluzole or inactive ingredients* in ROSF.
    Subjects or subject's legally authorized representative must be willing and able to complete informed consent/assent and HIPAA authorization.
    Ability to comprehend and be informed of the nature of the study, as assessed by the Primary or Sub-Investigator.
    Subjects prescribed to take riluzole at or before the time of first dose. (The study is open to subjects currently taking riluzole at screening, subjects who are not currently taking riluzole at screening but who have taken riluzole in the past, and subjects to be newly started on riluzole (given as ROSF in the course of this study).
    Availability to volunteer for the entire study duration and willing to adhere to all protocol requirements.
    Female subjects of childbearing potential must have a negative urine pregnancy test at Screening and Visit 1-3. Female subjects of childbearing potential (i.e. not surgically sterile, not 2 years postmenopausal, or not with a sterile partner) must have a negative pregnancy test at screening and Visit 1-3, agree to abstinence, practicing double barrier contraception or using an FDA approved barrier method contraceptive (e.g., licensed hormonal or barrier methods) for greater than 2 months prior to screening visit and commit to an acceptable form of birth control for the duration of the study and for 30 days after participation in the study.
    Subjects, in the judgment of the investigator, must be suitable candidates for administration of ROSF (riluzole oral soluble film).

    Exclusion Criteria:

    Subjects with a history of clinically significant liver disease, renal disease, or any other medical condition judged to be exclusionary by the investigator.
    Subjects who are unwilling to sign informed consent or subjects who for any other reason in the judgment of investigator are unable to complete the study.
    Female subjects who have a positive urine pregnancy test (βhCG) at screening or visit 1, are trying to become pregnant or are breastfeeding.
    Subjects with active cancer within the previous 2 years, except treated basal cell carcinoma of the skin.
    Subjects who have taken any experimental drug within 30 days prior to enrollment or within 5 half-lives of the investigational drug -whichever is the longer period. However, subjects who have previously completed other Aquestive sponsored ROSF clinical studies within the last 30 days prior to enrollment may be eligible for consideration for entry into this study.
    Subjects with known history or presence of moderate or severe renal impairment as defined by a calculated creatinine clearance of ≤50 mL/minute.
    Subjects currently taking riluzole with alanine aminotransferase (ALT) levels greater than 5 times upper limit of normal or with evidence of clinical jaundice. (Riluzole should be discontinued in these patients.)
    Subjects who will be receiving riluzole for the first time who exhibit baseline elevations of several liver function tests (especially elevated bilirubin). (These findings at baseline should preclude the use of riluzole including ROSF.)
    Use of potentially hepatotoxic drugs: (e.g., allopurinol, methyldopa, sulfasalazine).
    Subjects with clinically significant abnormal laboratory values in the judgment of the investigator.
    Use of strong or moderate CYP1A2 inhibitors (e.g., ciprofloxacin, enoxacin, fluvoxamine, methoxsalen, mexiletine, thiabendazole, vemurafenib, zileuton) and CYP1A2 inducers (e.g. rifampin and barbiturates) in the previous 30 days before first drug administration.
    Employee or immediate relative of an employee of the investigator, MonoSol Rx LLC, any of its affiliates or partners, or inVentiv Health.
    Anything else that, in the opinion of the investigator, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.

  • Site Contact Information

    University of Florida Medical Center
    Julie Segura / .(JavaScript must be enabled to view this email address) / 352-273-5566
    Gainesville, Florida 32610
    United States

    Neurology Associates, P.C.
    402-483-7226
    Lincoln, Nebraska 68506
    United States

    Texas Neurology, P.A.
    214-827-3610
    Dallas, Texas 75214
    United States

The Effect of RNS60 on ALS Biomarkers

Study Purpose:

Amyotrophic Lateral Sclerosis (ALS) is a rare lethal neurodegenerative disease involving inflammation. Riluzole, the only drug for ALS, improves median survival by 3 months. This prompts new treatments of ALS. RNS60 is an experimental drug with favorable effects in preclinical studies of neuroinflammation and neurodegeneration. Based on significant efficacy demonstrated in preclinical studies and its excellent clinical safety profile, RNS60 is a promising candidate for a drug to treat ALS. Developing a pharmacodynamic marker will be a first and important step for dose finding and exploration of the mechanism of action in human, and pave the way to trials measuring drug efficacy.

The Investigator propose a multicenter, randomized, double-blind, placebo-controlled, parallel group, Phase II trial. The study centers will be located in Italy and at Massachusetts General Hospital (MGH) in Boston. A total of 142 ALS patients will be randomly assigned to RNS60 or placebo (administered by intravenous infusion once/week and inhaled via nebulization every morning for 24 weeks). All participants will also take riluzole (50-mg tablet twice/day). Blood samples for biomarker analysis (protein, RNA) will be collected in the screening period, on day 1, week 4,12 and 24. Both safety and potential therapeutic effects of RNS60 will be also assessed.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Ettore Beghi, MDIRCCS Istituto di ricerche farmacologiche Mario Negri di Milano

Clinicaltrials.gov ID (11 digit #):

NCT03456882

Neals Affiliated?

No

Coordinating Center Contact Information

IRCCS Istituto di ricerche farmacologiche Mario Negri di Milano
Ettore Beghi, MD / .(JavaScript must be enabled to view this email address) / 0039 02 39014542
Elisabetta Pupillo, PharmD, PhD / .(JavaScript must be enabled to view this email address) / 0039 02 39014605
Milano, Italy

Full Study Summary:

Study Sponsor:

Mario Negri Institute for Pharmacological Research

Participant Duration:

Estimated Enrollment:

142

Estimated Study Start Date:

11/18/2016

Estimated Study Completion Date:

04/30/2019

Posting Last Modified Date:

03/21/2018

Date Study Added to alsconsortium.org:

03/21/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    70

    Min Vital Capacity (% predicted normal):

    80

    Time since Symptom Onset:

    <24 months

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Age 18 through 70 years inclusive;
    Geographically accessible to the site and able to come to the site once a week for 24 weeks;
    Definite or probable ALS diagnosis according to the revised El Escorial criteria; 4) Disease duration 6 to 24 months from symptom onset;

    5) Self sufficiency: Satisfactory bulbar and spinal function (score 3+ on the ALSFRS-R for swallowing, cutting food and handling utensils, and walking); 6) Satisfactory respiratory function (FVC ≥80% of predicted); 7) Documented progression of symptoms in the last three months, as measured by the ALSFRS-R scale; 8) Ability to understand and comply with the study requirements and to give written informed consent personally or via a legally authorized representative; 9) Treatment with riluzole 50 mg twice/day for at least 1 month prior to screening visit.

    Self sufficiency: this term reflect independence in daily living activities. It is an intuitive parameter to indicate preservation of key functional activities, and - not least - it has shown to be a valid and reliable measure

    Exclusion Criteria:

    History of HIV, clinically significant chronic hepatitis, antecedent polio infection, or other active infection;
    Motor neuron disease (MND) other than ALS;
    Involvement of systems other than motor possibly determining a functional impairment (as measured by the end-points) for the entire duration of the study;
    Other severe clinical conditions (e.g., cardiovascular disorders, neoplasms) with impact on survival or functional disability in the next 12 months;
    Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of normal;
    Poor compliance with previous treatments;
    Other experimental treatments in the preceding 3 months;
    Women who are lactating or able to become pregnant (e.g. who are not post menopausal, surgically sterile, or using inadequate birth control) and men unable to practice contraception for the duration of the treatment and 3 months after its completion;
    Unwillingness or inability to take riluzole; 10) Poor capability to use an inhalation device;
    Abnormal liver function defined as AST and/or ALT > 3 times the upper limit of the normal.

  • Site Contact Information

    Massachusetts General Hospital
    Michael Doyle / .(JavaScript must be enabled to view this email address) / 617-724-7398
    Boston, Massachusetts 02114
    United States

    Ospedale San Raffaele
    Riva / 0226433838
    Miano,
    Italy

    Centro Clinico NEMO - Fondazione Serena Onlus
    Lunetta / 02 914 3371
    Milano,
    Italy

A Multicenter, 18-week Open Label Safety and Efficacy Trial of Dalfampridine in Primary Lateral Sclerosis

Study Purpose:

This study will comprise an 18-week open label safety and tolerability trial. In this study, a total of 35 subjects with primary lateral sclerosis PLS or upper motor neuron predominate ALS will be enrolled. At the initial screening evaluation, a baseline T25FW will be obtained. This baseline test will be repeated at weeks 2, 4, 6, 10, 14 18. The validity of this measure was shown in MS studies when compared to the MSWS-12 (12 item walking scale) and CGI (clinical global impression) scales (35-37). A consistent responder will be defined as improvement in 3 of 4 Timed 25Foot Wwalk while on medication, compared with the baseline results while off medication.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Primary Lateral Sclerosis (PLS)

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Dr. Dale J. Lange, Weill Medical College of Cornell University

Clinicaltrials.gov ID (11 digit #):

NCT02868567

Neals Affiliated?

No

Coordinating Center Contact Information

Hospital for Special Surgery
Mona Shahbazi, NP / .(JavaScript must be enabled to view this email address) / 212-774-2361
Shara Holzberg, MS / .(JavaScript must be enabled to view this email address) / 646-797-8592
New York, New York 10021 United States

Full Study Summary:

Study Sponsor:

Weill Medical College of Cornell University

Participant Duration:

18 weeks

Estimated Enrollment:

35

Estimated Study Start Date:

03/01/2016

Estimated Study Completion Date:

01/31/2019

Posting Last Modified Date:

02/26/2018

Date Study Added to alsconsortium.org:

02/26/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    99

    Min Vital Capacity (% predicted normal):

    60

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Male or female, aged 18-99;
    Diagnosis of upper motor neuron disease, compatible with PLS but may include upper motor neuron (UMN) predominant ALS, defined as only upper motor neuron (UMN) features in at least 2 body regions on examination.
    EMG within 3 months of enrollment with minimal or no evidence of lower motor neuron disease,
    Time from symptom onset > 18 months
    No previous allergy to dalfampradine
    No current or exposure to any therapeutic agent targeting PLS or ALS within 30 days of enrollment.
    Must have a forced vital capacity (FVC) ≥ 60% of expected
    Written informed consent prior to screening is present.
    Subjects on a stable dose of or have not taken Riluzole for at least thirty days
    Impaired walking as measured by a Hauser Index of greater than 1 and less than 7 (2 to 6, inclusive);
    Mini Mental Status Score > 22 and deemed by the PI of being capable of providing informed consent and following trial procedures.
    Geographically accessible to the site.
    Women must not be able to become pregnant (e.g., post-menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and three months after study completion. Adequate contraception includes: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal contraception, for example patch or contraceptive ring), intrauterine device (IUD) in place for ≥ 3 months, barrier method in conjunction with spermicide, or another adequate method.
    Elicitable Motor evoked potentials (MEP) recorded at 75% of the stimulator output, using a monophasic, reverse direction, normal current mode pulse.

    Exclusion Criteria:

    History of clinically significant liver disease, renal disease, peripheral neuropathy, serious peripheral vascular disease, known HSP or + C9orf72 or SPG4 mutation, or any other medical condition felt to be exclusionary by the investigator;
    Unwillingness to sign informed consent or any other reasons for which the investigator feels the subject cannot complete the study;
    Women who are pregnant, breastfeeding, or trying to become pregnant;
    Active cancer within the previous 2 years, except treated basal cell carcinoma of the skin;
    Subjects taking any other experimental drugs within 30 days prior to enrollment;
    Patient has any history of seizures; brain surgery, brain implants, any metallic implants above the neck, cardiac pacemakers, cochlear implants, piercing or body modification above the neck, known history of TMS related complications or side-effects, tinnitus.
    Patient has moderate or severe renal impairment as defined by a calculated creatinine clearance of ≤50 mL/minute;
    Patient has been administered botulinum toxin in the lower extremities within 6 months prior to the screening visit and/or is expected to receive botulinum toxin in the lower extremities during the course of the study;
    Patient has a known allergy to pyridine-containing substances or any of the inactive ingredients of the dalfampridine tablet (colloidal silicon dioxide, hydroxypropyl methylcellulose, magnesium stearate, microcrystalline cellulose, polyethylene glycol, and titanium dioxide);
    Patient has a history of drug or alcohol abuse within the past year;
    Patient has clinically significant abnormal laboratory values.
    Anything else that, in the opinion of the SI, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.
    Inability to record an MEP at 75% of the stimulator output using a monophasic, reverse direction, normal current mode pulse due to either an in-excitable motor cortex, co-existing conditions that prevent TMS to be performed in a study subject, inability to tolerate the procedure, or any other cause.

  • Site Contact Information

    Hospital for Special Surgery
    Mona Shahbazi, NP / .(JavaScript must be enabled to view this email address) / 212-774-2361

    New York, New York 10021
    United States

Genetic Characterization of Movement Disorders and Dementias

Study Purpose:

Background:
There are two basic types of movement disorders. Some cause excessive movement, some cause slowness or lack of movement. Some of these are caused by mutations in genes. On the other hand, dementia is a condition of declining mental abilities, especially memory. Dementia can occur at any age but becomes more frequent with age. Researchers want to study the genes of families with a history of movement disorders or dementia. They hope to find a genetic cause of these disorders. This can help them better understand and treat the diseases. This study will not be limited to a particular disorder, but will study all movement disorders or dementias in general. This study will perform genetic testing to identify the genetic causes of movement disorders and dementia. Today, genetic testing can be done to analyze multiple genes at the same time. This increases the chances of finding the genetic cause of movement disorders and dementias.

Objectives:
To learn more about movement disorders and dementia, their causes, and treatments.

Eligibility:
Adults and children with a movement disorder or dementia, and their family members.
Healthy volunteers.

Design:
Participants will be screened with medical history and blood tests. Some will have physical exam.

Participants will give a blood sample by a needle in the arm. This can be done at the clinic, by their own doctor, or at home. Alternatively, a saliva sample may be provided if a blood sample cannot be obtained.

Participants can opt to send an extra blood sample to a repository for future study. Genetic test will be done on these samples. The samples will be coded. The key to the code will remain at NIA. Only NIA investigators will have access to the code key. Participants can request to receive results of the tests.

Participation is generally a single visit. Participants may be called back for extra

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Healthy Volunteer, Healthy Volunteer with a Family History of ALS, Ataxia, Dystonia, Parkinson's Disease, Corticobasal Degeneration, Multiple System Atrophy, Alzheimer's Disease, Lewy Body Dementia, Parkinson Disease-Dementia, Dentatorubral-pallidoluysian Atrophy

Study Type:

Observational Study

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Bryan J Traynor, M.D., National Institute on Aging (NIA)

Clinicaltrials.gov ID (11 digit #):

NCT02014246

Neals Affiliated?

No

Coordinating Center Contact Information

National Institute of Aging
Cynthia D Crews / .(JavaScript must be enabled to view this email address) / 301-451-3826
Bryan J Traynor, M.D. / .(JavaScript must be enabled to view this email address) / 301-451-7606
United States

Full Study Summary:

Objective
The objective of this study is to ascertain individuals with a clinical diagnosis of a movement disorder or dementia, their affected and unaffected family members, and unrelated, healthy individuals (to provide control samples); to characterize their phenotypes; and to identify and further characterize genetic contributions to etiology by collecting blood samples, saliva samples, and/or skin biopsies on these individuals for DNA and induced Pluripotent stem (iPs) cell line preparation.

Study population
Up to 10,000 persons with a diagnosis of a movement disorder or dementia, 1,000 asymptomatic persons who are family members/related to individuals with a diagnosis of movement disorder or dementia, and 1,000 unrelated, healthy control individuals.

Design
This study usually requires one outpatient visit to the NIH Clinical Center. Participant visits may also take place when they are an inpatient at the NIH Clinical Center. Those who are unable to travel to NIH may have study procedures performed at a site near their home, such as hospital facilities, private physician offices, nursing homes, assisted living facilities, local community centers, or participant homes. Participants will undergo medical record review, a physical examination and biospecimen collection including blood draw, saliva collection and/or skin biopsy at the enrollment visit.

Additional visits may be scheduled to collect additional phenotype information or to collect additional biospecimens.

Outcome measures
The primary outcome measure of this study is the identification of pathogenic genetic variants that are causative for the movement disorder or dementia that the patient has been diagnosed with. These disease-causing variants are often inherited.

The secondary outcome measure of this study is the identification of genetic variants that alter susceptibility/risk for the movement disorder or dementia that the patient has been diagnosed with. These genetic risk factors are associated with disease that can be apparently sporadic in nature.

Study Sponsor:

National Institute on Aging (NIA)

Participant Duration:

Participation is generally a single visit

Estimated Enrollment:

12000

Estimated Study Start Date:

02/12/2003

Estimated Study Completion Date:

12/31/2018

Posting Last Modified Date:

01/11/2018

Date Study Added to alsconsortium.org:

01/11/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    2

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    INCLUSION CRITERIA

    For Patients:

    Diagnosis of a movement disorder or dementia by a neurologist or other qualified professional and accompanied by sufficient clinical and/or laboratory evidence to support the diagnosis
    Confirmation of a movement disorder or dementia by study investigators or a qualified clinician by physical examination and/or review of medical records
    Ages 2 or older (age 18 or older to participate via mailer kit)
    Able to provide consent or, in the case of minors, or cognitive impairment, have a legally-authorized representative to provide consent
    Able to understand and participate in study procedures

    For unaffected family members of patients:

    Unaffected relative of a patient diagnosed with a movement disorder or dementia enrolled in this protocol. For these purposes, we define a family member as an individual for which there is a demonstrable relationship with the proband in the pedigree. This is a standard approach used in family-based studies. Furthermore, the related patient (defined as a family member diagnosed with the disease of interest) must be enrolled in the study.
    Ages 2 or older (age 18 or older to participate via mailer kit)
    Able to provide consent or, in the case of minors or cognitive impairment, have a legally-authorized representative to provide consent
    Able to understand and participate in study procedures

    For unrelated healthy control individuals:

    Be in good general health
    Have no known movement disorder or dementia, or family member with a movement disorder or dementia
    Age 18 and above
    Able to provide consent
    Able to understand and participate in study procedures

    EXCLUSION CRITERIA

    For patients:

    -An identifiable, non-genetic etiology for the movement disorder or dementia, such as a specific environmental exposure, birth injury, metabolic disorder, or brain infection such as encephalitis

    For all participants:

    Clinically significant anemia that would make phlebotomy unsafe, and participant unwilling to provide saliva sample.
    Clinically significant bleeding that would make phlebotomy unsafe, and participant unwilling to provide saliva sample.
    Any medical condition that would make phlebotomy unsafe or undesirable, such as a serious medical illness like unstable heart disease, or unstable chronic obstructive pulmonary disease, and participant unwilling to provide saliva sample.

  • Site Contact Information

    National Institute of Aging, Clinical Research Unit
    Baltimore, Maryland 21224
    United States

An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults With Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation

Study Purpose:

The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 in participants with Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation (SOD1-ALS). The secondary objective is to evaluate the pharmacokinetic (PK) profile of BIIB067 in participants with SOD1-ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03070119

Neals Affiliated?

No

Coordinating Center Contact Information


.(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Biogen

Participant Duration:

Estimated Enrollment:

48

Estimated Study Start Date:

03/08/2017

Estimated Study Completion Date:

01/01/2020

Posting Last Modified Date:

01/11/2018

Date Study Added to alsconsortium.org:

01/11/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Key Inclusion Criteria:

    Must have diagnosis of SOD1-ALS, and must have completed Part A and/or Part B of Study 233AS101 (NCT02623699) (i.e., were not withdrawn and did not miss more than 1 dose of study drug).
    If taking riluzole, must be receiving a stable dose for ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.
    For participants of childbearing potential must agree to practice effective contraception during the study and for 5 months after their last dose of study treatment.
    Medically able to undergo the study procedures, and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.

    Key Exclusion Criteria:

    History of or positive test result for human immunodeficiency virus.
    History of or positive test result for hepatitis C virus antibody or hepatitis B virus (defined as positive for both hepatitis B surface antigen and hepatitis B core antibody).
    History of allergies to a broad range of anesthetics.
    Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally and could place a participant at an increased risk for bleeding during or after a Lumbar Puncture (LP) procedure. These risks could include, but are not limited to, anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms, or other abnormalities) and underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., hemophilia, Von Willebrand's disease, liver disease).
    Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter.
    Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell therapy, or gene therapy.
    Treatment with another investigational drug, biological agent (excluding BIIB067), or device within 1 month or 5 half-lives of study agent, whichever is longer.
    Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period.
    Female participants who are pregnant or currently breastfeeding.
    Current enrollment in any other interventional study.
    Current or recent (within 1 month) use, or anticipated need, in the opinion of the Investigator, of copper (II) (diacetyl-bis(N4-methylthiosemicarbazone)) or pyrimethamine.

    NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

  • Site Contact Information

Novel MRI Biomarkers for Monitoring Disease Progression in ALS

Study Purpose:

Routine MRI is normal in motor neuron diseases such as ALS. However, advanced MRI techniques can provide an objective measure of degeneration (a "biomarker") by examining brain structure, wiring, chemistry, and function. We will develop and evaluate novel MRI techniques that could improve our understanding of ALS and provide a means to diagnose it sooner and monitor its progression. Importantly, we expect these techniques to improve how new drugs are tested, which may lead to the more rapid discovery of a treatment for ALS. Each participant will have 3 MRI scans over a period of 8 months, along with neurological and cognitive evaluations. Study visits will take 2 - 3 hours. MRI is a safe technique that does not involve radiation.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Primary Lateral Sclerosis (PLS), Healthy Volunteer

Study Type:

Observational Study

Study Category:

Biomarkers/Imaging

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Sanjay Kalra, MDFRCPC, University of Alberta

Clinicaltrials.gov ID (11 digit #):

NCT03362658

Neals Affiliated?

No

Coordinating Center Contact Information

University of Alberta
Dennell M Mah, BA / .(JavaScript must be enabled to view this email address) / 780-248-1805
.(JavaScript must be enabled to view this email address) Canada

Full Study Summary:

Current clinical measures of disease burden have suboptimal sensitivity to disease progression in ALS. A biomarker would play an essential role in the evaluation of novel therapeutics, leading to the realization of effective treatments faster. Magnetic resonance imaging (MRI) holds promise as a non-invasive source of biomarkers in ALS. In this study data is collected from a national imaging platform (the Canadian ALS Neuroimaging Consortium [CALSNIC]) using standardized MRI and clinical protocols.

CALSNIC was founded with the objective to validate MRI biomarkers on a standardized multi-centre platform. CALSNIC is a multidisciplinary group of scientists at 7 centres across Canada. The first CALSNIC study entitled "MRI Biomarkers in ALS" (CALSNIC-1) is ongoing and slated to finish recruitment in 2017.

This study ("Novel MRI Biomarkers for Monitoring Disease Progression in ALS", CALSNIC-2) is a new project that will evaluate novel MRI biomarkers using advanced imaging acquisition and processing methods. The specific aims of CALSNIC-2 are 1) to establish a standardized MRI and clinical protocol across the 7 centres, and 2) to validate MRI measures with clinical measures of disease burden and progression.

It is anticipated that the project will lead to the discovery of MR-based biomarkers of cerebral degeneration that can be applied across different centres and hence, can assist with drug development. Secondly, this project will expand CALSNIC to include more centres and provide opportunities for collaborative and multidisciplinary translational research on a national scale.

Study Sponsor:

University of Alberta

Participant Duration:

8 months

Estimated Enrollment:

700

Estimated Study Start Date:

10/01/2016

Estimated Study Completion Date:

12/31/2020

Posting Last Modified Date:

01/05/2018

Date Study Added to alsconsortium.org:

01/05/2018
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Patients with a diagnosis as described in Study Populations
    For those with a diagnosis of ALS, patients will be considered with an El Escorial classification of suspected, possible, probable, probable lab-supported, and definite ALS.
    Patients 18 years of age or older
    Healthy controls over the age of 40.
    Be able to lie in an MRI machine for approximately 60 minutes

    Exclusion Criteria:

    Subjects with psychiatric/CNS illnesses such as Major Depressive Disorder, Schizophrenia, and Bipolar disorder.
    Subjects with significant head injury or other neurological disease (stroke, brain tumour).
    Subjects ineligible for MRI investigation due to a pacemaker or other metallic foreign body.

  • Site Contact Information

    University of Calgary / Heritage Medical Research Clinic
    Victoria Hodgkinson / .(JavaScript must be enabled to view this email address) / 403-210-7303
    Calgary, Alberta T2N 4Z6
    Canada

    University of Alberta
    Dennell Mah, BA / .(JavaScript must be enabled to view this email address) / 780-248-1805
    Edmonton, Alberta T6G 2B7
    Canada

    University of British Columbia / GF Strong Rehab Centre
    Marife Fabros, RN / .(JavaScript must be enabled to view this email address) / 604-737-6319
    Vancouver, British Columbia V5Z 2G9
    Canada

    Western University / London Health Sciences Centre
    Christine Piechowicz / .(JavaScript must be enabled to view this email address) / 519-685-8500 ext 34858
    London, Ontario N6A 5A5
    Canada

    University of Toronto / Sunnybrook Health Sciences Centre
    Shirley Pham / .(JavaScript must be enabled to view this email address) / 416-480-5618
    Toronto, Ontario M4N 3M5
    Canada

    McGill University / Montreal Neurological Institute and Hospital
    Natalie Saunders / .(JavaScript must be enabled to view this email address) / 514-398-6526
    Montreal, Quebec H3A 2B4
    Canada

    Laval University
    Alexandra Simard, BSc / .(JavaScript must be enabled to view this email address) / 418-649-0252 ext 63559
    Quebec City, Quebec G1V 0A6
    Canada

An Open Label Study to Evaluate the Safety and Efficacy of FLX-787-ODT for Treatment of Fasciculations in the Tongue and Upper or Lower Extremity Muscles Most Affected in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

The FLX-787-106 study will determine how well FLX-787-ODT works to reduce fasciculations in patients with Amyotrophic Lateral Sclerosis (ALS). The study will measure how often fasciculations occur, and monitor any side effects that might develop while taking the investigational product. Participants will be assessed before and after taking a single dose of FLX-787-ODT. Approximately 15 people will take part in this study at one center in the United States. Participants will be in the study for a single clinic visit and receive a telephone call 7 days later to monitor for side effects.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase I ,Phase II

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03338114

Neals Affiliated?

No

Coordinating Center Contact Information


David Golod, PhD / .(JavaScript must be enabled to view this email address) / 617-721-1988
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

Study Sponsor:

Flex Pharma, Inc.

Participant Duration:

Estimated Enrollment:

15

Estimated Study Start Date:

11/30/2017

Estimated Study Completion Date:

08/31/2018

Posting Last Modified Date:

01/05/2018

Date Study Added to alsconsortium.org:

01/05/2018
  • More Information

    This study was withdrawn (sponsor decision)

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    <36 months

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Documented diagnosis of ALS diagnosis of less than 3 years.
    Expected survival > 6 months
    Frequent fasciculations noted during clinical examination of any single muscle (>6 visible fasciculations per minute) observed in Part I
    Normal oral cavity exam at screening
    Proficient in English
    Male subjects will either be surgically sterile or agree to use a medically acceptable method of contraception during sexual contact with females of childbearing potential, and will refrain from sperm donation for 45 days following the study
    Male subjects will either be surgically sterile or agree to use a medically acceptable method of contraception during sexual contact with females of childbearing potential, and will refrain from sperm donation for 45 days following the study

    Exclusion Criteria:

    Presence of clinically significant or unstable condition that would result in an increased risk of study participation or difficulty in interpretation of the study results
    Tremor or other movement disorder that would interfere with recording
    Inability to lie flat
    Presence of major gastrointestinal disorders, such as inflammatory bowel disease, diverticulitis, active peptic ulcer disease, or significant gastroesophageal reflux disease (i.e., not well-controlled on antacids or proton pump inhibitors), or oral or esophageal lesions/ulcers
    Presence of laryngospasm or significant swallowing problems
    Presence of percutaneous endoscopic gastrostomy, esophagogastroduodenoscopy, or G-tube
    Inability to tolerate a spicy sensation in the mouth or stomach
    Actively using illicit drugs or history of chronic substance abuse within the past year prior to screening, including abuse of alcohol
    Participated in a clinical study (except natural history studies without administration of an investigational product) within 30 days prior to screening
    Pregnant, breastfeeding, or planning to become pregnant
    Blood pressure of ≥160 mmHg systolic and/or ≥100 mmHg diastolic
    Clinically significant abnormalities in laboratory findings (including screening complete electrolyte panel, complete blood count, liver function tests).

  • Site Contact Information

    Wake Forest University Health Sciences
    James Caress, MD / .(JavaScript must be enabled to view this email address) / 336-716-2323
    Winston-Salem, North Carolina 27157
    United States

A Phase 3 Study to Compare the Efficacy and Safety of Masitinib in Combination With Riluzole Versus Placebo in Combination With Riluzole in the Treatment of Patients Suffering From ALS

Study Purpose:

The objective is to compare the efficacy and safety of masitinib in combination with riluzole versus corresponding placebo in combination with riluzole in the treatment of patients diagnosed with Amyotrophic Lateral Sclerosis (ALS).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not yet enrolling

Phase:

Phase III

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03127267

Neals Affiliated?

No

Coordinating Center Contact Information

McGill University
Angela Genge / .(JavaScript must be enabled to view this email address) / 514-398-3868
Alain Moussy / .(JavaScript must be enabled to view this email address) Canada

Full Study Summary:

Study Sponsor:

AB Science

Participant Duration:

Estimated Enrollment:

406

Estimated Study Start Date:

09/01/2017

Estimated Study Completion Date:

09/01/2019

Posting Last Modified Date:

11/08/2017

Date Study Added to alsconsortium.org:

11/08/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    81

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Patient diagnosed with laboratory supported, clinically probable or definite ALS
    Patient treated with a stable dose of riluzole (100 mg/day) for at least 30 days prior to screening
    Patient with adequate organ function at screening

    Exclusion Criteria:

    Patient who underwent tracheostomy and /or gastrostomy

  • Site Contact Information

A Phase II Study of Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis

Study Purpose:

The purpose of this study is to determine the safety and efficacy of intrathecal treatment delivered to the cerebrospinal fluid (CSF) of mesenchymal stem cells in ALS patients every 3 months for a total of 4 injections over 12 months.

Mesenchymal stem cells (MSCs) are a type of stem cell that can be grown into a number of different kinds of cells. In this study, MSCs will be taken from the subject's body fat and grown. CSF is the fluid surrounding the spine.

The use of mesenchymal stem cells is considered investigational, which means it has not been approved by the Food and Drug Administration (FDA) for routine clinical use. However, the FDA has allowed the use of mesenchymal stem cells in this research study.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Stem Cell

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Nathan P Staff, MD, PhD, Mayo Clinic
Anthony J Windebank, MD, Mayo Clinic

Clinicaltrials.gov ID (11 digit #):

NCT03268603

Neals Affiliated?

No

Coordinating Center Contact Information

Mayo Clinic in Rochester
Michelle Turner / .(JavaScript must be enabled to view this email address)
Carol Denny / .(JavaScript must be enabled to view this email address) Rochester, Minnesota 55905 United States

Full Study Summary:

The Goal of the Proposed Study is to perform an open label, 60 subject, Phase II multi-site clinical trial to investigate the safety and efficacy of intrathecal treatment of aaMSCs in ALS. Patients will be treated with 1 x 10^8 aaMSCs every 3 months for a total of 4 intrathecal injections over 12 months. Reduced dose treatments will be allowed based on specific adverse events. Multiple biomarkers will be tracked throughout the clinical trial and correlated with response to treatment. This study will initially be performed at Mayo Clinic in Rochester. Subsequently, the study will expand to the two other Mayo Clinic sites in Arizona and Florida as the manufacturing capabilities are finalized.

Study Sponsor:

Mayo Clinic

Participant Duration:

Estimated Enrollment:

60

Estimated Study Start Date:

09/01/2017

Estimated Study Completion Date:

12/31/2019

Posting Last Modified Date:

10/31/2017

Date Study Added to alsconsortium.org:

10/31/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    65

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    All patients will have ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by the World Federation of Neurology criteria for the diagnosis of ALS.
    Examination and neurophysiological testing confirm a pure motor syndrome compatible with the diagnosis of ALS. All other possible causes of weakness have been excluded by extensive investigations.
    Age greater than 18 years, if female, must be post-menopausal, had a hysterectomy, or agree to two forms of birth control.
    Permanent resident or citizen of the United States.
    Geographic accessibility to the study site and willingness and ability to comply with follow-up.
    History of a chronic onset of a progressive motor weakness of less than two years duration.
    Subjects must be taking a stable dose of riluzole for at least 30 days prior to enrolment or not be on riluzole, and not have been on it for at least 30 days prior to enrolment (riluzole-naïve subjects are permitted in the study).
    Able to comply with protocol requirements, including MRI testing.
    Can provide written informed consent.

    Exclusion Criteria:

    Any clinically significant medical condition (e.g., within six months of baseline, had myocardial infarction, angina pectoris, and/or congestive heart failure) that, in the opinion of the investigator, would compromise the safety of patient.
    Pulmonary Slow Vital Capacity (SVC) less than 65% of predicted for age, gender, and body type.
    Autoimmunity, including Crohn's disease or rheumatoid arthritis
    Current use of immunosuppressant medication or use of such medication within 4 weeks of Screening visit (Visit 1).
    Malignancy 5 years prior to enrollment, including melanoma,with the exception of localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline).
    Active systemic or local infection near the lumbar puncture site.
    Inability to lie flat for the duration of intrathecal cell transplantation, or inability to tolerate study procedures for any other reason.
    Other active systemic disease as defined by laboratory abnormalities delineated in Appendix IV.
    Use of herbal medications or other unapproved drugs
    Enrolled in an investigational drug trial within 30 days of baseline visit
    Prior stem cell therapy of any kind
    Kokmen Short Test of Mental Status score <32
    Presence of a tracheostomy
    Ventilator dependent
    Pregnancy
    Men or women of childbearing potential who are unwilling to employ adequate contraception

  • Site Contact Information

    Mayo Clinic in Rochester
    Michelle Turner / .(JavaScript must be enabled to view this email address) / Rochester, Minnesota 55905
    United States

SOD1 Kinetics Measurements in ALS Patients

Study Purpose:

Washington University in St. Louis is seeking participants with ALS for a study to determine the half-life of the protein SOD1 in the cerebral spinal fluid. Mutations in the SOD1 gene are known to cause some forms of familial ALS. Researchers are developing a treatment to reduce the level of SOD1 in familial ALS, but need to know more about how long SOD1 stays in the body ("half-life") to help determine if the new treatment is effective.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Observational Study

Study Category:

Biomarkers/Imaging

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Timothy Miller, MD, PhD, Washington University (St. Louis)

Clinicaltrials.gov ID (11 digit #):

NCT03449212

Neals Affiliated?

No

Coordinating Center Contact Information

Washington University in St. Louis
Jennifer Balsarotti / .(JavaScript must be enabled to view this email address) / 314-362-6159
.(JavaScript must be enabled to view this email address) Dept. of Neurology, Campus Box 8111
660 S Euclid Ave.
St. Louis , Missouri 63110 United States

Full Study Summary:

Background: Novel targeted therapeutic strategies are being developed for genetic subsets of ALS, such as those caused by dominantly inherited mutations in the superoxide dismutase 1 gene (SOD1). Investigators have developed an antisense oligonucleotide (ASO) inhibitor of SOD1 biosynthesis for ALS patients who carry mutations in SOD1. This ASO is now ready for clinical trial (ClinicalTrials.gov #NCT02623699). The initial success of the ASO will depend on showing a pharmacodynamics result on SOD1 in participants, and thus a key challenge in applying this targeted therapy involves rigorous examination of pharmacodynamics markers.

The Investigator's previous data suggest that SOD1 in the cerebral spinal fluid (CSF) will be an excellent pharmacodynamics marker for an SOD1-focused therapeutic approach. However, one of the central missing components in understanding SOD1 as a marker is SOD1 CSF half-life data. The half-life of this protein will aid in clinical trial planning since half-life influences the amount of SOD1 protein reduction by ASO and thus dictates the optimal timing of drug administration and CSF collection for pharmacodynamics measures.

Objectives:

  • Enroll a total of 86 ALS participants
  • Determine the kinetics for total SOD1 protein, as well as the wild type and mutant protein separately
  • Determine this in patients with known SOD1 mutation as well as sporadic ALS patients Eligibility
  • Adults over age 18
  • fALS with confirmed genetic testing showing a mutation in the SOD1 gene; asymptomatic SOD1 gene carriers and sporadic ALS patients.

Measures: The key outcome of this study is to determine the half-life of the SOD1 protein in symptomatic and asymptomatic ALS patients which will provide critical information to inform future therapeutic studies in ALS. For ALS patients, The Investigators will also perform Slow Vital Capacity testing and the ALSFRS-R at the screening visit and at each lumbar puncture visit.

Measures: Participants will have up to 7 in-person visits over 4 months. The study involves labeling or marking SOD1 with a special type of leucine. Leucine is an essential amino acid that is found in the foods we eat. This method involves an overnight stay for a 16 hour intravenous infusion of labeled leucine along with a collection blood and urine followed by 5 lumbar punctures scheduled over the period of 4 months.

At each subsequent visit, subjects will undergo a blood draw, urine collection, lumbar puncture, a questionnaire (ALS Functional Rating Scale) which measures motor function, and a breathing test to determine Slow Vital Capacity (SVC) measurements.

Analysis: In addition to determining the half-life of the SOD1 protein in ALS patients, the Investigators will also analyze the kinetics of wild type SOD1 protein separately from mutant SOD1 protein to determine differences in half life. The Investigators will also compare CSF Tau half-life between ALS patients and controls as a disease specificity control. The Investigators hope to correlate this data with clinical measures which may reveal other important hypotheses regarding SOD1 kinetic rates and disease manifestations.

Study Sponsor:

ALS Finding A Cure/NIH

Participant Duration:

1 overnight stay for leucine infusion. 4-5 lumbar punctures performed over a period of 3-4 months at Washington University. Each lumbar puncture visit takes approximately three hours.

Estimated Enrollment:

86

Estimated Study Start Date:

12/01/2012

Estimated Study Completion Date:

10/01/2019

Posting Last Modified Date:

04/10/2018

Date Study Added to alsconsortium.org:

10/30/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


  • Site Contact Information

    Massachusetts General Hospital
    165 Cambridge Street
    Boston, Massachusetts 20114
    United States

    Washington University
    Saint Louis, Missouri 63110
    United States

Cognition, Behavior and Caregiver Burden in Amyotrophic Lateral Sclerosis

Study Purpose:

The focus of this study is to investigate risk factors for development of problems with thinking, behavior and mood in Veterans with ALS and whether these problems affect their caregivers.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Primary Lateral Sclerosis (PLS), 

Study Type:

Observational Study

Study Category:

Epidemiology

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Christopher Brady, PhD, VA Boston Healthcare Systems

Clinicaltrials.gov ID (11 digit #):

Neals Affiliated?

No

Coordinating Center Contact Information

VA Boston Healthcare System
Nazifa Abdul Rauf / .(JavaScript must be enabled to view this email address) / 857-364-2136
.(JavaScript must be enabled to view this email address) 150 South Huntington Ave. (151C)
Boston, Massachusetts 02130 United States

Full Study Summary:

A new ALS research study, titled, "Cognition, Behavior, and Caregiver Burden in Amyotrophic Lateral Sclerosis" is being conducted by the Department of Veterans Affairs (VA). This study is designed to investigate whether there are risk factors for the development of problems with thinking, behavior and mood in Persons with Amyotrophic Lateral Sclerosis (PALS), and whether these problems affect their caregivers (e.g., spouse, adult child)

Questions We Want to Answer 

  1. How does ALS affect changes in your thinking, behavior and mood? Are there risk factors for the development of these changes?
  2. How do these changes affect your caregiver? Do caregivers have special needs that your healthcare provider should be aware of?

How ALS Can Affect Your Thinking and Behavior

This study is investigating whether amyotrophic lateral sclerosis (ALS) can cause problems in thinking, behavior, and mood. We are also interested in learning whether these problems affect caregivers. A caregiver is anyone who regularly cares for a person with ALS, and can be a spouse,  an adult child, or other family members.

We will ask you to complete tasks that will help us measure your thinking (such as attention, concentration, and memory), behavior, and mood. Additionally, we will ask your caregiver to complete surveys about you, his/her mood, and the challenges that he/she may face as a caregiver. These tasks and surveys will help us to understand whether there are different patterns of thinking and behavior problems that occur in people with ALS. We will also examine whether thinking and behavior problems are related to greater caregiver burden.

What can I expect if I take part in this study?

In order to take part in this study, you and your caregiver will both need to participate. After enrollment, you and your caregiver will complete some assessments by telephone and by mail. This may take up to an hour of your time for each of you. About 6 months after the initial assessment, we will ask your caregiver to answer some questions over the phone and complete some brief questionnaires. Your caregiver will answer questions regarding their observations of your thinking, behavior, and mood, as well as whether they are experiencing any challenges or burdens as a result of caregiving.

A year after enrollment, you and your caregiver will have another telephone interview and be mailed questionnaires similar to the initial assessment. These annual and 6-month follow-ups (caregivers only) will continue for up to 3 years.

Study Sponsor:

VA BLR&D

Participant Duration:

Any Veteran with ALS (VALS) and their caregiver may be eligible to participate in the study. As this is a VALS/caregiver study, both VALS and their primary caregiver need to participate in order to take part in the study. Participants will be asked to complete surveys by mail and telephone semi-annually for up to three years. This may take up to one hour of their time for each follow up. This study does not require any travel, as all assessments are done by phone and mail.

Estimated Enrollment:

100

Estimated Study Start Date:

Estimated Study Completion Date:

Posting Last Modified Date:

12/17/2018

Date Study Added to alsconsortium.org:

10/16/2017

A Phase 3, Randomized Double-Blind, Placebo-Controlled Multicenter Study to Evaluate Efficacy and Safety of Repeated Administration of NurOwn® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors) in Participants With ALS

Study Purpose:

This study will evaluate the safety and efficacy of repeated administration of NurOwn® (MSC-NTF cells) therapy, which is based on transplantation of autologous bone marrow derived mesenchymal stromal cells (MSC), which are enriched from the patient's own bone marrow, propagated ex vivo and induced to secrete Neurotrophic factors (NTFs).

The autologous NurOwn® (MSC-NTF cells) are back-transplanted into the patient intrathecally by standard lumbar puncture where neurons and glial cells are expected to take up the neurotrophic factors secreted by the transplanted cells

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Stem Cell

Study Status:

Not enrolling

Phase:

Phase III

Study Chair(s)/Principal Investigator(s):

Merit E. Cudkowicz, MD Massachusetts General Hospital
Robert H. Brown, MD, PhD UMass Medical School
Anthony J. Windebank, MD Mayo Clinic
Namita A. Goyal, MD UC Irvine
Robert G. Miller, MD California Pacific Medical Center (CPM) Research Institute

Clinicaltrials.gov ID (11 digit #):

NCT03280056

Neals Affiliated?

No

Coordinating Center Contact Information


Ralph Z. Kern, MD / .(JavaScript must be enabled to view this email address) / 201-488-0460
Yael D. Gothelf, Ph.D. / .(JavaScript must be enabled to view this email address) Israel

Full Study Summary:

Neurotrophic factors (NTFs) are potent survival factors for embryonic, neonatal, and adult neurons and are considered potential therapeutic candidates for ALS. Delivery of multiple NTFs to the immediate environment of afflicted neurons in ALS patients is expected to improve their survival and thus slow down disease progression and alleviate symptoms. NTF-secreting mesenchymal stromal cells (MSC-NTF cells) are a novel cell-therapeutic approach aimed at effectively delivering NTFs directly to the site of damage in ALS patients.

Participants meeting the inclusion and exclusion criteria will be randomized and will undergo bone-marrow aspiration. MSC of the participants randomized to the treatment group will be induced into MSC-NTF cells. Participants will undergo a total of three intrathecal (IT) transplantations with NurOwn® (MSC-NTF cells) or matching placebo at three bi-monthly intervals.

Study Sponsor:

Brainstorm-Cell Therapeutics

Participant Duration:

Estimated Enrollment:

200

Estimated Study Start Date:

08/28/2017

Estimated Study Completion Date:

07/30/2019

Posting Last Modified Date:

03/21/2018

Date Study Added to alsconsortium.org:

09/18/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    60

    Min Vital Capacity (% predicted normal):

    65

    Time since Symptom Onset:

    <24 months

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria.
    Having onset of ALS disease symptoms, including limb weakness within 24 months at the Screening Visit.
    ALSFRS-R ≥ 25 at the screening Visit.
    Upright slow vital capacity (SVC) measure ≥ 65% of predicted for gender, height, and age at the screening Visit.
    Participants taking a stable dose of Riluzole are permitted in the study

    Exclusion Criteria:

    Prior stem cell therapy of any kind
    History of autoimmune or other serious disease (including malignancy and immune deficiency)
    Current use of immunosuppressant medication or anticoagulants (per Investigator discretion)
    Exposure to any other experimental agent or participation in an ALS clinical trial within 30 days prior to Screening Visit
    Use of RADICAVA (edaravone injection) within 30 days of screening or intent to use edaravone at any time during the course of the study including the follow up period
    Use of non-invasive ventilation (BIPAP), diaphragm pacing system or invasive ventilation (tracheostomy)
    Feeding tube
    Pregnant women or women currently breastfeeding

  • Site Contact Information

    University of California Irvine Alpha Stem Cell Clinic
    Robert Zhou / .(JavaScript must be enabled to view this email address) / 949-824-3990
    Irvine, California 92697
    United States

    Cedars-Sinai Medical Center
    Carolyn Prina / .(JavaScript must be enabled to view this email address) / 310-423-1713
    Los Angeles, California 90048
    United States

    California Pacific Medical Center
    Dallas Forshew, RN / .(JavaScript must be enabled to view this email address) / 415-309-5178
    San Francisco, California 94115
    United States

    Massachusetts General Hospital
    Taylor Mezoian / .(JavaScript must be enabled to view this email address) / 617-643-0312
    Boston, Massachusetts 02115
    United States

    University of Massachusetts Medical School
    Diane McKenna-Yasek / .(JavaScript must be enabled to view this email address) / 508-856-4697
    Worcester, Massachusetts 01655
    United States

    Mayo Clinic
    Carol Denny / .(JavaScript must be enabled to view this email address) / 507-284-2676
    Rochester, Minnesota 55905
    United States

Expanded Access Use of microRNA Delivered Intrathecally via Adeno-Associated Virus rh10 as Therapy for Mutant SOD1 Mediated Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

The purpose of this study is to provide compassionate use of virally delivered miRNA against SOD1 to two subjects with ALS caused by mutations in the SOD1 gene. 

Disease:

Familial ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial , Gene Therapy

Study Status:

Not enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Robert Brown, D. Phil, M.D., UMass
James Berry, M.D., MGH

Clinicaltrials.gov ID (11 digit #):

Neals Affiliated?

Yes

Coordinating Center Contact Information

Massachusetts General Hospital
Lindsay Pothier / .(JavaScript must be enabled to view this email address) / 617-643-5582
.(JavaScript must be enabled to view this email address) Boston, Massachusetts United States

Full Study Summary:

This is an open-label, investigational protocol for intrathecal administration of anti-SOD1 miRNA (amiR- SOD1) as an emergency therapeutic in two protocol defined subjects with the SOD1 gene mutations. The intention is to deliver drug into the intrathecal space using adeno- associated virus rhesus strain 10 (AAVrh10). The primary outcome for our study will be clinical status of the patients as assessed by the ALSFRS-R. The safety of anti-SOD1 RNAi will be evaluated using vital signs, weight, clinical laboratory determinations, physical and neurological examinations, ECG’s, AEs, (including death and other SAEs), and use of concomitant medications.

Study Sponsor:

Robert H. Brown Jr., D. Phil, M.D.

Participant Duration:

The study volunteers will be followed at weekly (+/- 2 days) intervals for one month post infusion and then monthly (28 days +/- 5 days) until one year post-infusion. Thereafter visits will be every three months (84 days +/- 7 days) up to 24 months post infusion, then every 6 months (168 days +/- 14 days) thereafter indefinitely.

Estimated Enrollment:

2

Estimated Study Start Date:

11/01/2016

Estimated Study Completion Date:

11/01/2020

Posting Last Modified Date:

04/18/2018

Date Study Added to alsconsortium.org:

09/18/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    N/A

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


  • Site Contact Information

    Massachusetts General Hospital
    Boston, Massachusetts
    United States

    University of Massachusetts
    Worcester, Massachusetts
    United States

The DIALS (Dominant Inherited ALS) Network

Study Purpose:

The purpose of this study is to better understand the clinical and biological changes that may happen in adults who have an ALS causative gene and are asymptomatic (showing no symptoms).

Disease:

Volunteer with first-degree relatives of patients with inherited forms of ALS

Study Type:

Observational Study

Study Category:

Biomarkers/Imaging , Genetic Testing

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Katharine Nicholson, MD, Massachusetts General Hospital
Timothy Miller, MD, PhD, Washington University School of Medicine

Clinicaltrials.gov ID (11 digit #):

N/A

Neals Affiliated?

Yes

Coordinating Center Contact Information

Massachusetts General Hospital; NCRI
Isabel Anez / .(JavaScript must be enabled to view this email address) / 617-643-2499
.(JavaScript must be enabled to view this email address) 165 Cambridge Street
Boston, Massachusetts 02114 United States

Full Study Summary:

Overall Aim: Establish a multicenter research platform to evaluate people at high risk for ALS to identify early diagnostic signs and characterize biomarkers at the time of symptom conversion.

This project aims to establish a mechanism for genetic testing and research involvement of people at risk for inherited forms of ALS.

Aim 1: Provide a platform for the testing of known ALS causative genes in people at risk for familial ALS. 

Substantial barriers prevent the development of a cohort of asymptomatic ALS gene carriers, including the cost and availability of genetic testing and counseling, and insurance risks of putting genetic information into the clinical chart. This study will break down these barriers by using a funded research platform to perform CLIA-certified genetic testing at the New York Genome Center (NYGC) and develop a standardized approach to genetic counseling in ALS.

Hypothesis: Establishing a cohort of asymptomatic ALS gene carriers will provide the opportunity to target people in the early stages of ALS to maximize treatment effects, similar to other neurodegenerative diseases (6).

Aim 2: Perform longitudinal evaluation of people at risk for inherited forms of ALS in order to identify biomarkers of disease initiation (i.e. molecular, electrophysiologic) and novel measures of clinical disease onset (i.e. ATLIS, IOPI).

Upon development of an asymptomatic gene carrier cohort, this study will routinely characterize these patients with leading ALS biomarker candidates and quantitative clinical outcomes to provide a means to identify disease onset earlier than ever before. Biomarker evaluation will include blood and optional CSF collection. Specifically, this project will evaluate the following candidate biomarkers: CSF SOD1, repeat-associated non-ATG (RAN) dipeptides in C9ORF72, phosphorylated neurofilament heavy (pNfH) and neurofilament light (NfL) chain.

Hypothesis:  Longitudinal evaluation of asymptomatic ALS gene carriers will provide critical insight into the earliest clinical and biological changes associated with ALS, to facilitate early diagnosis and better define disease conversion in ALS. Comparison of candidate biomarkers in asymptomatic ALS gene carriers to biobanked specimens from existing protocols of symptomatic people with sporadic and familial ALS and healthy controls will help to characterize the earliest biomarker in ALS.

This study is looking to enroll about 55 asymptomatic first-degree relatives of known C9ORF72 and SOD1-positive patients with ALS from 2 ALS centers in the US. Study participants will be followed for 5 years regardless of gene status, with gene-negative participants providing important comparative information for leading candidate biomarker and outcome development. Study visits for collection of clinical data and biofluid biomarkers will occur every 6 months from screening.

During the informed consent process, study participants will indicate preference to learn the results of genetic testing (i.e. disclosure group).  It is anticipated that at least 70% (28 out of 40) of study participants who undergo genetic testing will opt for gene disclosure, based on prior research initiatives with disclosure and non-disclosure groups in asymptomatic ALS gene carriers. Study participants will undergo genetic counseling with medically-licensed study staff (physician site investigator or genetic counselor) as a part of disclosure group determination, to ensure participants are fully informed of the risk of life and long-term care insurance denial if a positive genetic result is known to them.

During the informed consent process, participants within the disclosure group will have the option to identify a designee to receive the study participant’s genetic results should the participant become unexpectedly incapacitated or pass away from a medical condition other than ALS. The designee will be an adult (i.e. over the age of 18) who is related to or known to the subject. Identification of a designee will be made in conjunction with genetic counseling with medically licensed study staff (physician site investigator or genetic counselor). By indicating preference for non-disclosure, genetic results will not be returned to the study participant or a designee. Individuals who initially elect non-disclosure can decide later to undergo genetic counseling and learn their genetic results at any time. Upon electing for disclosure, participants may then identify a designee to receive the study participant’s genetic results should the participant become incapacitated or pass away.

Study Sponsor:

Target ALS, ALSFAC, Private Donor

Participant Duration:

Approximately, 5 years with up to 14 visits approximately 3-6 months apart, 2 of these will be telephone visits.

Estimated Enrollment:

85

Estimated Study Start Date:

04/01/2017

Estimated Study Completion Date:

04/01/2022

Posting Last Modified Date:

05/02/2019

Date Study Added to alsconsortium.org:

09/18/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


  • Site Contact Information

    Massachusetts General Hospital; NCRI
    Tiina Xu / .(JavaScript must be enabled to view this email address) / 617-643-7428
    165 Cambridge Street
    Boston, Massachusetts 02114
    United States

    Washington University, Department of Neurology
    Jennifer Jockel-Balsarotti / .(JavaScript must be enabled to view this email address) / 314-362-8624
    4523 Clayton Road
    St. Louis, Missouri 63110
    United States

Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia

Study Purpose:

Objectives
The primary objective is to screen patients who are referred with a diagnosis of frontotemporal dementia, motor neuron disorder, or related adult-onset neurodegenerative disorder to assess patient eligibility for ongoing protocols. The secondary objective is to develop and maintain a registry of characterized patients who may be eligible for future trials. An exploratory objective is to obtain biospecimens from clinically characterized patients to carry out laboratory-based studies aimed at understanding the molecular pathways and genetic overlap between these neurodegenerative disorders.

Study population
Adults referred with clinical diagnoses of frontotemporal dementia, motor neuron disorder, or related adult-onset neurodegenerative disorder.

Design
Participants will all undergo diagnostic screening tests, a standard battery of tests to measure cognitive and motor function, and blood for clinical testing and research. Additional diagnostic and research testing, including magnetic resonance imaging, electromyography, neuropsychological testing, lumbar puncture, and skin biopsy will be obtained in selected patients.

Outcome measures
Patients will be screened for eligibility for current research protocol or entered into a registry future research protocols

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Primary Lateral Sclerosis (PLS), 

Study Type:

Observational Study

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Mary Kay Floeter, M.D., National Institute of Neurological Disorders and Stroke (NINDS)

Clinicaltrials.gov ID (11 digit #):

NCT03225144

Neals Affiliated?

No

Coordinating Center Contact Information


Carol H. Hoffman / .(JavaScript must be enabled to view this email address) / 301-451-1229
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

Objectives
The primary objective is to screen patients who are referred with a diagnosis of frontotemporal dementia, motor neuron disorder, or related adult-onset neurodegenerative disorder to assess patient eligibility for ongoing protocols. The secondary objective is to develop and maintain a registry of characterized patients who may be eligible for future trials. An exploratory objective is to obtain biospecimens from clinically characterized patients to carry out laboratory-based studies aimed at understanding the molecular pathways and genetic overlap between these neurodegenerative disorders.

Study population
Adults referred with clinical diagnoses of frontotemporal dementia, motor neuron disorder, or related adult-onset neurodegenerative disorder.

Design
Participants will all undergo diagnostic screening tests, a standard battery of tests to measure cognitive and motor function, and blood for clinical testing and research. Additional diagnostic and research testing, including magnetic resonance imaging, electromyography, neuropsychological testing, lumbar puncture, and skin biopsy will be obtained in selected patients.

Outcome measures
Patients will be screened for eligibility for current research protocol or entered into a registry future research protocols

Study Sponsor:

National Institute of Neurological Disorders and Stroke (NINDS)

Participant Duration:

Estimated Enrollment:

200

Estimated Study Start Date:

09/19/2017

Estimated Study Completion Date:

01/01/2030

Posting Last Modified Date:

09/14/2017

Date Study Added to alsconsortium.org:

09/14/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    INCLUSION CRITERIA:

    Patients will be included if they

    Are age 18 or older
    Have been given a diagnosis by a neurologist of frontotemporal dementia, frontotemporal lobar degeneration, primary progressive aphasia, semantic dementia, motor neuron disorder, amyotrophic lateral sclerosis, primary lateral sclerosis, progressive bulbar palsy, corticobasal syndrome, Huntington disease or other related adult-onset neurodegenerative Disorder

    EXCLUSION CRITERIA:

    Patients will be excluded if they

    have other major neurological or medical diseases that may cause progressive weakness or cognitive dysfunction, such as structural brain or spinal cord disease, metabolic diseases, paraneoplastic syndromes, infectious diseases, peripheral neuropathy or radiculopathy or other significant neurological abnormalities.
    Have an unstable medical condition that, in the opinion of the investigators, makes participation unsafe
    require daytime ventilator support at the time of study entry
    are unable to travel to NIH
    Patients with pacemakers or other implanted electrical devices, brain stimulators, dental implants, aneurysm clips (metal clips on the wall of a large artery), metallic prostheses (including metal pins and rods, heart valves, and cochlear implants), permanent eyeliner, implanted delivery pumps, or shrapnel fragments, metal fragments in the eye) will not be excluded but will not undergo magnetic resonance imaging.

  • Site Contact Information

    National Institutes of Health Clinical Center
    Robin Godwin, M.S.N. / .(JavaScript must be enabled to view this email address) / 301-451-6733
    Bethesda, Maryland 20892
    United States

A Population-Based Ohio ALS Repository and a Case-Controlled Study of ALS Risk Factors

Study Purpose:

The proposed research is particularly relevant to the National ALS Registry and public environmental health issues because it addresses the potential environmental causes of sporadic ALS. The research will develop an ALS surveillance program in Ohio that can be compared with the national and State-Metro Surveillance Programs of the National ALS Registry, and novel methodologies to determine the role of the cyanobacterial toxin, BMAA, and other environmental toxicants as risk factors for ALS. This work will advance the mission of the CDC ATSDR National ALS Registry by offering data on ALS cases in Ohio that address public health concerns over the effects of chronic exposure to cyanobacterial blooms in Lake Erie.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Observational Study

Study Category:

Patient Registry

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Erik Pioro, MD, PhD   

Clinicaltrials.gov ID (11 digit #):

NCT02916966

Neals Affiliated?

No

Coordinating Center Contact Information

Cleveland Clinic Foundation
Elijah W Stommel, MD, PhD / .(JavaScript must be enabled to view this email address) / 866-894-8131
Dominic Facciponte / .(JavaScript must be enabled to view this email address) / 603-650-4205
Cleveland, Ohio 44195 United States

Full Study Summary:

Study Sponsor:

Dartmouth-Hitchcock Medical Center

Participant Duration:

Estimated Enrollment:

1000

Estimated Study Start Date:

10/01/2016

Estimated Study Completion Date:

10/01/2018

Posting Last Modified Date:

09/01/2017

Date Study Added to alsconsortium.org:

09/01/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Study Population

    ALS patients in case-control at CCF
    Non-neurodegenerative patients in case control at CCF
    Non-neurodegenerative general population in Ohio
    ALS patient registry in Ohio

    Inclusion Criteria:

    ALS
    Non-neurodegenerative controls
    Can willingly give consent
    Over 18 years of age

    Exclusion Criteria:

    Cannot give consent
    Under 18 years of age

  • Site Contact Information

    Cleveland Clinic Foundation
    Cleveland, Ohio 44195
    United States

An Online Mindfulness Intervention for People With ALS and Their Caregivers

Study Purpose:

The psychological impact of ALS on both patients and caregivers is high and affects their quality of life (QOL). However, there is minimal research about psychological interventions to improve QOL in the ALS scientific literature.

Recent advances in clinical treatments aimed at improving the health of people with chronic disorders are based on the concept of mindfulness. Mindfulness can be defined as a flexible state of mind resulting from the simple act of actively noticing new things, as opposed to mindlessness, the human tendency to operate on" autopilot".

Preliminary data suggests that mindfulness may promote a better QOL for people with ALS and their caregivers. The investigators also found that a mindful attitude was associated with slower disease progression.

This project's goal is to develop an innovative, web-based online mindfulness training program and intervention, customized for people with ALS and their primary caregivers. It is an active learning intervention, with cognitive exercises and lectures that increase participants' mindfulness. The efficacy of this program for improving QOL, and for reducing anxiety and depression in people with ALS and their caregivers, will be tested with a randomized clinical trial. Assessments immediately post-treatment as well as 3 and 6 months after recruitment will be conducted, comparing subjects undergoing the mindfulness intervention to a control group.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Behavioral: Mindfulness

Study Status:

Not enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Ellen Langer, PhD, Professor of Psychology

Clinicaltrials.gov ID (11 digit #):

NCT03095989

Neals Affiliated?

No

Coordinating Center Contact Information


Ellen Langer, PhD / .(JavaScript must be enabled to view this email address)
Deborah Phillips, PhD / .(JavaScript must be enabled to view this email address)

Full Study Summary:

Quality of Life in people with ALS is not related to physical function, but is related to psychological, existential, and support factors. ALS caregivers demonstrate considerable burden and low QOL. It has been proposed by the investigators that it is time for the ALS field to develop "best practices" that use psychological intervention to improve QOL, reducing psychological distress in people with ALS and their caregivers. A disease that is as inexorably progressive and physically devastating as ALS also inevitably has an immense psychological impact on caregivers, both at the time of diagnosis and throughout the course of the disease. Yet, there is scant research on psychological interventions for ALS patients and caregivers.

Over 35 years of research demonstrate that mindfulness-based interventions comprise some of the most promising behavioral treatments in QOL improvement for people with chronic disorders.This work is based on socio-cognitive aspects of mindfulness, conceptualized as having a flexible state of mind that incorporates active engagement in the present and being sensitive to context and perspective. In over 35 years of research it has been shown that a higher state of mindfulness correlates with an improved QOL. In addition, mindfulness may lead to an improvement in some physiological as well as psychological measures. The literature on mindfulness in other disorders, combined with preliminary data on mindfulness in ALS patients and their caregivers, provides a strong evidence-based platform for proposing a careful study of the effects of mindfulness on psychological and physiological measures of ALS patients and their caregivers. The investigators conducted an exploratory investigation on a large sample (100+) of people with ALS about the influence of mindfulness on QOL and the course of the disease. The investigators found that mindfulness predicted positive QOL and psychological well-being, reducing anxiety and depression. That was quite expected. However, another finding was that mindfulness positively influenced the changes in physical symptoms: subjects with higher mindfulness experienced a slower progression of the disease, as measured by the self- administered ALS Functional Rating Scale (SA-ALSFRS). Furthermore, mindfulness was a significant predictor of positive QOL and psychological well-being in ALS caregivers, with a protective effect against anxiety and depression.

Fifty (50) participants with ALS, together with each subject's primary caregiver (i.e., an expected overall sample of 100 subjects), will be directly recruited from the Hershey Medical Center ALS Clinic at Penn State University. Participants will sign IRS-approved informed consent documents. Both patients and caregivers may be recruited even if a component of the dyad (patient-caregiver) opts not to join the study (i.e. patients and caregivers alone may be recruited). ALS patient-caregiver couples will be considered as a single subject for randomisation purposes (meaning that both the patient and the caregiver are randomly allocated to the same group). Subjects will be randomized into two groups (employing true randomization from atmospheric noise, www.random.org): an intervention group and a control group. Subjects in the intervention group will participate in the online mindfulness program, in addition to standard clinical care.

The intervention for each patient-caregiver pair will commence immediately after recruitment, on a rolling basis. Participants from the control group will be placed on a waiting list and will receive the standard care that they would receive if not in the study. Participants from both groups will be assessed 4 times: at recruitment (T1, baseline); after completing the intervention, or 5 weeks after recruitment for the control group (T2, post- treatment); three months after recruitment (T3); six months after recruitment (T4).

The primary outcome measure will be QOL (assessed with the ALS-Specific Quality of Life-Revised instrument, the ALSSQOL-R) in ALS subjects. Secondary outcomes from ALS subjects will be: depression and anxiety (assessed with the Hospital Anxiety and Depression Scale), attained mindfulness level (assessed with the Langer Mindfulness Scale) functional, cognitive and respiratory variables, assessed respectively with the Self-Administered ALS Functional Rating Scale-Revised, the Edinburgh Cognitive Assessment and Forced Vital Capacity. Caregivers outcomes, considered as secondary outcomes, will be QOL (assessed with the Short Form-36), depression and anxiety (assessed with the Hospital Anxiety and Depression Scale), care burden (assessed with the Zarit Burden Inventory) and mindfulness (assessed with the Langer Mindfulness Scale). A sub-sample of the subjects from the intervention group may be invited to a semi-structured interview about the experience with the treatment.

After the last assessment, subjects in the control group will have the option to enter the mindfulness program. All the subjects will have the option to continue to use the program after the 5-week intervention.

Study Sponsor:

Harvard University

Participant Duration:

Estimated Enrollment:

100

Estimated Study Start Date:

01/01/2015

Estimated Study Completion Date:

12/31/2017

Posting Last Modified Date:

05/10/2019

Date Study Added to alsconsortium.org:

09/01/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Inclusion Criteria for ALS Subjects:

    Definite, probable, probable laboratory-supported, or possible ALS by revised El-Escorial criteria (Brooks, Miller, Swash, & Munsat, 2000).
    Must have the physical ability, with or without adaptive devices, to use a computer and access the Internet.
    A forced vital capacity of 50% or greater than the predicted value, measured within 30 days of enrollment. If impaired bulbar function compromises accurate pulmonary function testing as determined by the study neurologist, then a forced vital capacity as low as 40% of predicted is permitted. If taken, stable doses of anti-anxiety or antidepressant medications for 30 days before study entry, and during the study from the T1 to the T2 time point.

    Inclusion Criteria for Caregivers:

    Must have the physical ability to use a computer and access the Internet.

    Exclusion Criteria:

    Exclusion Criteria for ALS Subjects:

    Scores on the Edinburgh Cognitive Assessment (ECAS) within 90 days of study entry, that meet criteria for mild frontotemporal dysfunction (either cognitive or behavioral)
    Significant cognitive impairment or significant uncontrolled psychiatric disease (for example, schizophrenia, bipolar disorder), in the opinion of the study neurologist.
    Unsuitable for the study as determined by the study neurologist.
    Regular meditation or participation in a mindfulness program in past 6 months.

    Exclusion Criteria for Caregivers:

    Unwillingness to participate in the study
    Unsuitable for the study as determined by the clinical staff. For example, the clinical staff may consider inappropriate a caregiver who showed no real interest toward the care process or showed signs of severe mental health problems.

  • Site Contact Information

    Penn State Hershey Medical Center
    Anne Morris, MPH / .(JavaScript must be enabled to view this email address) / 717-531-0003 ext 289123
    Hershey, Pennsylvania 17033
    United States

A Phase 2, Multi-Center, Double-Blind, Randomized, Dose-Ranging, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of CK-2127107 in Patients With ALS (FORTITUDE-ALS)

Study Purpose:

The purpose of this study is to assess the effect of CK-2127107 versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03160898

Neals Affiliated?

Yes

Coordinating Center Contact Information


MD Cytokinetics / .(JavaScript must be enabled to view this email address) / 650-624-2929
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Enrolled participants will be dosed with CK-2127107 150, 300, 450 mg or placebo twice daily (300, 600, 900 mg/day or placebo) for a period of 12 weeks.

Study Sponsor:

Cytokinetics

Participant Duration:

12 weeks

Estimated Enrollment:

445

Estimated Study Start Date:

07/24/2017

Estimated Study Completion Date:

07/01/2018

Posting Last Modified Date:

11/29/2018

Date Study Added to alsconsortium.org:

09/01/2017
  • More Information

    Watch this webinar from January 30, 2018 to learn about the FORTITUDE-ALS trial design and hopes for the drug: recording link.

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Min Vital Capacity (% predicted normal):

    65

    Time since Symptom Onset:

    Time since Diagnosis:

    <24 months

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:
    Diagnosis of familial or sporadic ALS ≤ 24 months prior to screening
    Upright Slow Vital Capacity (SVC) ≥ 65% of predicted for age, height and sex at screening
    Able to swallow tablets
    A caregiver (if one is needed)
    Able to perform reproducible pulmonary function tests
    Pre-study clinical laboratory findings within the normal range or, if outside the normal range, deemed not clinically significant by the Investigator
    Male patients who have not had a vasectomy and confirmed zero sperm count must agree to either use acceptable methods of contraception or abstain from sex
    Female patients must be post-menopausal or sterilized or must not be breastfeeding, have a negative pregnancy test, have no intention to become pregnant during the study and use acceptable methods of contraception
    Patients must be either on riluzole for at least 30 days prior to screening or have not taken riluzole for at least 30 days prior to screening and not planning to start riluzole during the course of the study.

    Exclusion Criteria:
    At the time of screening, any use of non-invasive ventilation (NIV), e.g. continuous positive airway pressure [CPAP], noninvasive bi-level positive airway pressure [NPPV] or noninvasive volume ventilation [NVV]for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation
    Neurological impairment due to a condition other than ALS
    Presence at screening of any medically significant cardiac, pulmonary, GI, musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety or efficacy data
    Has taken any investigational study drug within 30 days or five half-lives of the prior agent, whichever is longer, prior to dosing
    Known to have received CK-2127107 or tirasemtiv in any previous clinical trial
    Has received or is considering receiving during the course of the study any form of stem cell therapy for the treatment of ALS
    Has received or is considering receiving during the course of the study any form of gene therapy for the treatment of ALS
    Has received or is considering obtaining during the course of the study a diaphragmatic pacing system
    History of substance abuse within the past 2 years
    Use of certain medications

  • Site Contact Information

    Barrow Neurological Institute
    Jacquelyn Nicolari / .(JavaScript must be enabled to view this email address) / 602-406-6606
    St. Joseph's Hospital and Medical Center
    Phoenix, Arizona 85013
    United States

    University of California Irvine
    Ivonne Turner / .(JavaScript must be enabled to view this email address) / Orange, California 92868
    United States

    Forbes Norris MDA/ALS Research Center
    Marguerite Engel / .(JavaScript must be enabled to view this email address) / San Francisco, California 94115
    United States

    Stanford Hospital and Clinics
    Michael Nguyen / .(JavaScript must be enabled to view this email address) / 650-724-6147
    Stanford, California 94305
    United States

    Cedars-Sinai Medical Center
    Koral Wheeler / .(JavaScript must be enabled to view this email address) / Los Angeles , California 90048
    United States

    University of Colorado Hospital Anschutz Outpatient Pavilion
    Nicola Haakonsen / .(JavaScript must be enabled to view this email address) / Aurora, Colorado 80045
    United States

    Hospital for Special Care
    New Britain, Connecticut 06053
    United States

    George Washington University Medical Faculty Associates
    Washington, District of Columbia 20037
    United States

    Mayo Clinic
    Pamela Desaro / .(JavaScript must be enabled to view this email address) / Jacksonville, Florida 32224
    United States

    University of Florida
    Gainesville, Florida 32610
    United States

    University of South Florida, Carol & Frank Morsani Center for Advanced Healthcare
    Tampa, Florida 33612
    United States

    Emory ALS Clinic
    Meraida Polak / .(JavaScript must be enabled to view this email address) / Atlanta, Georgia 30322
    United States

    Duchossois Center for Advanced Medicine
    Jacquelyn Hill / .(JavaScript must be enabled to view this email address) / 773-702-4610
    Chicago, Illinois 60637
    United States

    IU Health Neuroscience Center of Excellence
    Indianapolis, Indiana 46202
    United States

    University of Iowa Hospitals and Clinics
    Jeri Sieren / .(JavaScript must be enabled to view this email address) / Iowa City, Iowa 52242
    United States

    University of Kansas Medical Center
    Ayla McCalley / .(JavaScript must be enabled to view this email address) / Kansas City , Kansas 66160
    United States

    Johns Hopkins University - Outpatient Center
    Kristin M. Riley / .(JavaScript must be enabled to view this email address) / 410-955-8511
    Baltimore, Maryland 21287
    United States

    University of Massachusetts Medical School
    Worcester, Massachusetts 01655
    United States

    Michigan Medicine
    Jayna Duell / .(JavaScript must be enabled to view this email address) / 734-763-9037
    Ann Arbor, Michigan 48109
    United States

    Henry Ford Health System
    Ashkhen Movsisyan / .(JavaScript must be enabled to view this email address) / 313-916-4123
    Detroit, Michigan 48202
    United States

    Hennepin County Medical Center
    Cindy Rohde / .(JavaScript must be enabled to view this email address) / 612-873-2607
    Minneapolis, Minnesota 55415
    United States

    Mayo Clinic
    Rochester, Minnesota 55905
    United States

    Saint Louis University, Department of Neurology
    Saint Louis, Missouri 63104
    United States

    Washington University School of Medicine
    Saint Louis, Missouri 63110
    United States

    Neurology Associates, P.C.
    Kelli Ferguson / .(JavaScript must be enabled to view this email address) / Desirae Eschiti / .(JavaScript must be enabled to view this email address) / Lincoln, Nebraska 68506
    United States

    Hospital For Special Surgery
    Shara Holzberg / .(JavaScript must be enabled to view this email address) / New York, New York 10021
    United States

    Neurological Institute, Columbia University Medical Center
    New York, New York 10032
    United States

    SUNY Upstate Medical University
    Syracuse, New York 13210
    United States

    Neurosciences Institute, Neurology
    Charlotte, North Carolina 28207
    United States

    Duke Neurological Disorders Clinic
    Karen Grace / .(JavaScript must be enabled to view this email address) / Durham, North Carolina 27705
    United States

    Wake Forest School of Medicine
    Mozhdeh Marandi / .(JavaScript must be enabled to view this email address) / Winston-Salem, North Carolina 27157
    United States

    Cleveland Clinic
    Cleveland, Ohio 44195
    United States

    The Ohio State University Wexner Medical Center
    Ifeanyi Okoh / .(JavaScript must be enabled to view this email address) / Columbus, Ohio 43210
    United States

    Providence Brain and Spine Institute ALS Center
    Arlena Cummings / .(JavaScript must be enabled to view this email address) / 503-962-1171
    Portland, Oregon 97213
    United States

    Penn State Milton S. Hershey Medical Center
    Hershey, Pennsylvania 17033
    United States

    Temple University School of Medicine
    Kathleen Hatala / .(JavaScript must be enabled to view this email address) / 215-707-4171
    Philadelphia, Pennsylvania 19140
    United States

    Vanderbilt University Medical Center - Clinical Research Center
    Diana Davis / .(JavaScript must be enabled to view this email address) / 615-322-8957
    Nashville, Tennessee 37232
    United States

    Texas Neurology
    Todd Morgan / .(JavaScript must be enabled to view this email address) / 214-827-3610 ext 228
    Dallas, Texas 75214
    United States

    UTHSCSA Medical Arts and Research Center
    Pamela Kittrell / .(JavaScript must be enabled to view this email address) / 210-450-0524
    San Antonio, Texas 78229
    United States

    Houston Methodist Hospital
    Luis F Lay, Jr. / .(JavaScript must be enabled to view this email address) / 713-441-3057
    Houston, Texas 77030
    United States

    University of Vermont Medical Center
    Burlington , Vermont 05405
    United States

    University of Virginia Health System
    Mary Wagoner / .(JavaScript must be enabled to view this email address) / Charlottesville, Virginia 22908
    United States

    VCU Health - Ambulatory Care Center (ACC)
    Tamika Walthour / .(JavaScript must be enabled to view this email address) / Richmond, Virginia 23298
    United States

    University of Washington Medical Center
    Laura Sissons-Ross / .(JavaScript must be enabled to view this email address) / Seattle , Washington 98195
    United States

    West Virginia University, Dept. of Neurology
    Morgantown, West Virginia 26506-9180
    United States

    Froedtert Memorial Lutheran Hospital
    Lynn Wheeler / .(JavaScript must be enabled to view this email address) / Milwaukee , Wisconsin 53226
    United States

    University of Calgary, Heritage Medical Research Center
    Jose Antonio Martinez / .(JavaScript must be enabled to view this email address) / Calgary, Alberta T2N 4Z6
    Canada

    McMaster University Medical Centre
    Daniela Trapsa / .(JavaScript must be enabled to view this email address) / Hamilton, Ontario L8N 3Z5
    Canada

    Sunnybrook Health Science Centre
    Toronto, Ontario M4N 3M5
    Canada

    Montreal Neurological Institute and Hospital
    Montreal, Quebec H3A 2B4
    Canada

    Centre de recherche du Centre Hospitalier de l'Universite de Montreal
    Stefanie Houle / .(JavaScript must be enabled to view this email address) / Montréal, Quebec H2X 0A9
    Canada

    CHU de Quebec-Universite Laval, Hopital de l'Enfant Jesus
    Quebec G1J 1Z4
    Canada

    Edmonton Kaye Clinic
    Shelley Wrona / .(JavaScript must be enabled to view this email address) / Edmonton, Alberta T6GT 1Z1
    Canada

A Multicenter, Double Blind, Placebo Controlled Study to Assess the Efficacy and Safety of H.P. Acthar® Gel in the Treatment of Subjects With Amyotrophic Lateral Sclerosis

Study Purpose:

This is a multicenter, multiple dose study to examine the effect of H.P. Acthar® (Acthar) on functional decline in adult subjects with amyotrophic lateral sclerosis (ALS).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Susan VanMeter, Mallinckrodt

Clinicaltrials.gov ID (11 digit #):

NCT03068754

Neals Affiliated?

No

Coordinating Center Contact Information


Michael Zhang / .(JavaScript must be enabled to view this email address) / 800-556-3314
.(JavaScript must be enabled to view this email address)

Full Study Summary:

This is a multicenter, multiple dose study to examine the effect of Acthar on functional decline in adult subjects with amyotrophic lateral sclerosis (ALS). Approximately 195 subjects will be enrolled.

Following a screening period of up to 28 days, subjects with ALS and symptom onset (defined as first muscle weakness or dysarthria) ≤ 2 years prior to the Screening Visit will be randomized on a 2:1 basis to receive subcutaneous (SC) Acthar 0.2 mL (16 Units ) daily (QD) or SC matching placebo 0.2 mL QD for 36 weeks, followed by a 3 week taper.

Study Sponsor:

Mallinckrodt

Participant Duration:

Estimated Enrollment:

213

Estimated Study Start Date:

06/14/2017

Estimated Study Completion Date:

03/31/2020

Posting Last Modified Date:

10/11/2017

Date Study Added to alsconsortium.org:

08/25/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    75

    Min Vital Capacity (% predicted normal):

    60

    Time since Symptom Onset:

    <24 months

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Diagnosis of clinically definite ALS, clinically probable-laboratory supported ALS, or clinically probable ALS based on revised El Escorial criteria.
    ALS symptom onset ≤ 2 years prior to the Screening Visit.
    On riluzole 50 mg twice a day for ≥ 4 weeks prior to the Screening Visit.
    Forced vital capacity (FVC) ≥ 60% at the Screening Visit.
    Systolic blood pressure ≤ 140 mm Hg and a mean diastolic blood pressure of ≤ 90 mm Hg at the Screening and Baseline Visits.

    Exclusion Criteria:

    History of use of adrenocorticotropic hormone (ACTH) preparations for treatment of ALS.
    Any medical condition known to have an association with motor neuron dysfunction (other than ALS) which might confound or obscure the diagnosis of ALS.
    Requires nocturnal continuous positive airway pressure or bilevel positive airway pressure.
    Tracheostomy, diaphragm pacing, or ongoing need for assisted ventilation of any type at Screening Visit.
    Known contraindication(s) to Acthar.
    History of chronic active hepatitis including active or chronic hepatitis B, or acute or chronic hepatitis C.
    History of tuberculosis (TB) infection, any signs/symptoms of TB, or any close contact with an individual with an active TB infection.
    Clinically significant infection requiring intravenous administration of antibiotics and hospitalization in the 4 weeks prior to the Screening Visit.

  • Site Contact Information

    Neuromuscular Research Center
    Phoenix, Arizona 85208
    United States

    Loma Linda University Health System, Department of Neurology
    Loma Linda, California 92354
    United States

    Colorado Springs Neurological Associates
    Colorado Springs, Colorado 80907
    United States

    GW Medical Faculty Associates
    Washington, D.C., District of Columbia 20037
    United States

    University of South Florida
    Tampa, Florida 33609
    United States

    Indiana University-Neuroscience Center of Excellence
    Indianapolis, Indiana 46202
    United States

    University of Kansas Medical Center
    Kansas City, Kansas 66160
    United States

    John Hopkins Outpatient Center
    Baltimore, Maryland 21287
    United States

    Wesley Neurology Clinic
    Cordova, Tennessee 38018
    United States

    Texas Neurology, P.A.
    Dallas, Texas 75215
    United States

    Austin Neuromuscular Center
    Austin, Texas 78756
    United States

    Medical College of Wisconsin/Froedtert Hospital
    Milwaukee, Wisconsin 53226
    United States

Physiological Flow of Liquids Used in Dysphagia Management (Previously Tongue-Pressure Timing for Liquid Flow Detection and Control in Swallowing)

Study Purpose:

For individuals with neurodegenerative conditions, such as Amyotrophic Lateral Sclerosis and Parkinson disease, swallowing impairment (i.e., dysphagia) is a common and serious symptom. Dysphagia places the affected individual at risk for secondary health consequences, including malnutrition and aspiration pneumonia, and negatively affects quality of life.

Thickened liquids are commonly recommended for individuals with dysphagia, as they flow more slowly and reduce the risk of entry into the airway. However, there is limited understanding about how changes in liquid thickness modulate swallowing physiology in individuals with neurodegenerative conditions, and previous reports have shown that increased liquid thickness may contribute to the accumulation of residue in the throat.

The purpose of this study is to explore swallowing physiology and function in individuals with neurodegenerative conditions, across five levels of liquid thickness (thin, slightly-thick, mildly-thick, moderately-thick, and extremely-thick), and to identify boundaries of "optimal liquid thickness", which maintain airway safety, without contributing to the accumulation of significant residue. Results from this study will help guide the clinical recommendations for thickened liquids in dysphagia management.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Observational Study

Study Category:

Diagnostic Test: Videofluoroscopic Swallowing Examination and Tongue Strength Measurement

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Catriona M Steele, PhD, University Health Network - Toronto Rehabilitation Institute
Emily K Plowman, PhD, University of Florida

Clinicaltrials.gov ID (11 digit #):

NCT03192358

Neals Affiliated?

No

Coordinating Center Contact Information

University Health Network - Toronto Rehabilitation Institute
Ashley A Waito, MSc / .(JavaScript must be enabled to view this email address) / 416-597-3422 ext 7812
Kelby Magennis, MPH / .(JavaScript must be enabled to view this email address) / 352-273-8632
Toronto, Canada

Full Study Summary:

This research study will measure swallowing function in individuals with Amyotrophic Lateral Sclerosis or Parkinson's disease. The aims of this study are to (1) identify parameters of swallowing physiology that are associated with impaired swallowing safety and efficiency, and (2) explore how liquid thickness influences swallowing function.

Participation in this research study involves a single appointment at the University of Florida Swallowing Systems Core laboratory located at Shands Hospital, Gainesville. The appointment will last approximately 1 hour, and will involve tasks to measure tongue-strength, and a dynamic swallowing x-ray (known as videofluoroscopy) to evaluate swallowing function. A selection of demographic information (e.g., age, onset of symptoms) will also be recorded.

To measure tongue strength, participants will be given a disposable air-filled bulb and asked to perform a series of tongue presses, and swallow their saliva. Next, during the videofluoroscopy, participants will take sips of various liquids ranging in thickness from thin (like water), to extremely-thick (similar to the consistency of pudding or yogurt). The liquids will be mixed with a safe substance called barium, to make them visible on x-ray images. After the videofluoroscopy has been completed, each participant will have their tongue strength measured again, which will conclude their participation in the study.

Swallowing physiology will be measured from the videofluoroscopy images, post-hoc, by an experienced team of blinded raters.

Study Sponsor:

University Health Network, Toronto

Participant Duration:

Estimated Enrollment:

40

Estimated Study Start Date:

11/01/2017

Estimated Study Completion Date:

02/28/2021

Posting Last Modified Date:

05/15/2019

Date Study Added to alsconsortium.org:

08/07/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    90

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:
    Adults (18+) with a confirmed diagnosis of Amyotrophic Lateral Sclerosis (ALS) or Parkinson's disease (PD)
    Reported speech or swallowing problems

    Exclusion Criteria:
    People with a prior medical history of stroke
    People with a prior medical history of acquired brain injury
    People with a prior medical history of spinal or spinal cord injury
    People with a prior medical history of cancer or surgery in the head and neck region
    People who have had radiation to the head and neck for cancer
    People who have a prior history of swallowing problems (e.g., from childhood, medical complication)
    People with significant breathing difficulties (e.g., rely on mechanical ventilation)
    People who rely solely on tube-feeding for all meals and nutrition
    People who have Type I (insulin-dependent) Diabetes
    Women who are pregnant
    People who have allergies to barium, potato starch, corn starch, xanthan gum, milk products, latex or dental glue

  • Site Contact Information

    University of Florida
    Emily K Plowman, PhD / .(JavaScript must be enabled to view this email address) / 352-273-9215
    Kelby Magennis, MPH / .(JavaScript must be enabled to view this email address) / 352-273-8632
    Gainesville, Florida 32611
    United States

A Pilot Study of Lung Volume Recruitment Combined With Expiratory Muscle Strength Training in ALS

Study Purpose:

The purpose of this study is to investigate the effects of two treatment techniques called Expiratory Muscle Strength Training (EMST) and Lung Volume Recruitment (LVR) on breathing, swallowing, speech, and cough function in persons with mild to moderate ALS. Half of the participants will do EMST alone, and the other half of the participants will do EMST and LVR.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Procedures: Expiratory Muscle Strength Training (EMST) and EMST + Lung Volume Recruitment (LVR)

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

David Walk, MD, University of Minnesota - Clinical and Translational Science Institute

Clinicaltrials.gov ID (11 digit #):

NCT03202017

Neals Affiliated?

No

Coordinating Center Contact Information

University of Minnesota
Valerie Ferment / .(JavaScript must be enabled to view this email address) / 612-301-1535
.(JavaScript must be enabled to view this email address) Minneapolis, Minnesota 55455 United States

Full Study Summary:

Study Sponsor:

University of Minnesota - Clinical and Translational Science Institute

Participant Duration:

12 weeks

Estimated Enrollment:

24

Estimated Study Start Date:

03/01/2018

Estimated Study Completion Date:

12/31/2021

Posting Last Modified Date:

05/14/2019

Date Study Added to alsconsortium.org:

08/07/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    65

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:
    ALS defined as possible, laboratory-supported probable, probable, or definite by El Escorial criteria
    Reduced Maximal Expiratory Pressure (MEP) compared to norms for age and sex
    Forced Vital Capacity (FVC) > 65% predicted

    Exclusion Criteria:
    Inability to provide informed consent
    Relative contraindications to LVR, including known pneumothorax, active internal bleeding, unstable hypertension, unstable angina, emphysema, recent barotrauma, or FEV1 (Forced Expiratory Volume, trial 1)/FVC ratio < 0.7.
    Use of EMST or breath stacking > 3 days/week within 12 weeks of screening
    Amyotrophic Lateral Sclerosis-Cognitive Behavioral Scale (ALS-CBS) score predictive of dementia (≤ 10)
    Participation in another clinical trial of an intervention in ALS within 30 days of study enrollment or during study enrollment

  • Site Contact Information

    University of Florida
    Kelby Magennis / .(JavaScript must be enabled to view this email address) / 352-273-8632
    Gainesville, Florida 32611
    United States

    University of Minnesota
    Valerie Ferment / .(JavaScript must be enabled to view this email address) / 612-301-1535
    Minneapolis, Minnesota 55455
    United States

ALS Study Determining Various Biomarkers and Strength Comparison After Exercise (ADVANCE)

Study Purpose:

The purpose of this study is to determine the muscle strength of a muscle in the thigh after 12 weeks of home exercise.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Resistance Exercise Program

Study Status:

Not enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Omar Jawdat, MD, University of Kansas Medical Center

Clinicaltrials.gov ID (11 digit #):

NCT03201991

Neals Affiliated?

No

Coordinating Center Contact Information

University of Kansas Medical Center
Laura Herbelin / .(JavaScript must be enabled to view this email address) / 913-588-5095
.(JavaScript must be enabled to view this email address) Kansas City, Kansas 66160 United States

Full Study Summary:

Study Sponsor:

University of Kansas Medical Center

Participant Duration:

12 weeks

Estimated Enrollment:

7

Estimated Study Start Date:

05/01/2017

Estimated Study Completion Date:

05/01/2018

Posting Last Modified Date:

08/02/2017

Date Study Added to alsconsortium.org:

08/02/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Diagnosis of possible, probable or definite amyotrophic lateral sclerosis (ALS) based on the revised El-Escorial criteria
    Presence in ipsilateral leg of either weakness in any muscle group or of active denervation by needle electromyography (EMG) which is a surrogate marker of early denervation
    Ipsilateral quadriceps femoris strength: >=4
    Ambulatory with or without assistance
    Revised ALS Functional Rating Scale (ALSFRS-R) Score > 30
    Forced expiratory vital capacity (FVC) >50% of predicted

    Exclusion Criteria:

    ALSFRS-R ≤ 30
    Quadriceps femoris strength <4
    Unable to walk or uses wheelchair as primary means of mobility
    More than mild atrophy of quadriceps
    Bleeding disorder or uptake of anticoagulants
    Unwilling to comply with exercise and needle muscle biopsy
    Not a good research candidate according to the medical opinion of investigator

  • Site Contact Information

    University of Kansas Medical Center
    Laura Herbelin / .(JavaScript must be enabled to view this email address) / 913-588-5095
    Kansas City, Kansas 66160
    United States

A Randomized, Double-Blind, Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of FLX-787-ODT for Treatment of Muscle Cramps in Adult Subjects With Motor Neuron Disease

Study Purpose:

The COMMEND Study will assess the safety and effectiveness of FLX-787 in men and women with Motor Neuron Disease [including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy (PMA)] experiencing muscle cramps. Participants will be asked to take two study products during the course of the study. One of these study products will be a placebo.

Approximately 120 participants in approximately 30 study centers across the United States are expected to take part. Participants will be in the study for approximately 3 months and visit the study clinic 3 times.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Primary Lateral Sclerosis (PLS)

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Bjorn Oskarsson, MD, Mayo Clinic Jacksonville Florida

Clinicaltrials.gov ID (11 digit #):

NCT03196375

Neals Affiliated?

No

Coordinating Center Contact Information


Jennifer Szegda / .(JavaScript must be enabled to view this email address) / 617-893-9898
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Flex Pharma, Inc.

Participant Duration:

3 months

Estimated Enrollment:

120

Estimated Study Start Date:

07/28/2017

Estimated Study Completion Date:

06/30/2018

Posting Last Modified Date:

08/06/2018

Date Study Added to alsconsortium.org:

08/02/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:
    Documented diagnosis of Motor Neuron Disease (MND) [including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy (PMA)]
    Expected survival > 6 months
    Weekly muscle cramping (defined as: a sustained muscle contraction that's most often painful and lasts seconds to minutes)

    Exclusion Criteria:
    Presence of major gastrointestinal disorders, such as inflammatory bowel disease, diverticulitis, active peptic ulcer disease, or significant gastroesophageal reflux disease (i.e., not well-controlled on antacids or proton pump inhibitors), or oral or esophageal lesions/ulcers
    Presence of laryngospasm or significant swallowing problems
    Presence of percutaneous endoscopic gastrostomy, esophagogastroduodenoscopy, or G-tube
    Unable or unwilling to discontinue medications for cramps and/or opiates
    Inability to tolerate a spicy sensation in the mouth or stomach
    Actively using illicit drugs or history of chronic substance abuse within the past year prior to screening, including abuse of alcohol
    Intention to change the current level of tobacco use or use of nicotine-containing products (i.e., new smokers or those actively trying to quit may not enrolled)
    Participated in a clinical study (except natural history studies without administration of an investigational product) within 30 days prior to screening

  • Site Contact Information

    Honor Health Research Institute
    Angelina Cooper / .(JavaScript must be enabled to view this email address) / 480-882-5564
    Scottsdale, Arizona 85251
    United States

    Mayo Clinic
    Mark Ross / .(JavaScript must be enabled to view this email address) / 480-301-6711
    Scottsdale, Arizona 85259
    United States

    California Pacific Medical Center
    Dallas Forshew / .(JavaScript must be enabled to view this email address) / 415-309-5178
    Sacramento, California 94115
    United States

    University of Colorado
    Nicola Haakonsen / .(JavaScript must be enabled to view this email address) / 303-724-4644
    Aurora, Colorado 80045
    United States

    Hospital for Special Care
    Agnes Koczon-Jaremko / .(JavaScript must be enabled to view this email address) / 860-612-6356
    New Britain, Connecticut 06053
    United States

    GW Medical Faculty Associates Inc.
    Lindsey Covington / .(JavaScript must be enabled to view this email address) / 202-741-2745
    Washington , District of Columbia 20037
    United States

    University of South Florida Health
    Brittany Harvey / 813-974-9413
    Tampa, Florida 33612
    United States

    Mayo Clinic
    Arijana Draganovic / .(JavaScript must be enabled to view this email address) / 904-953-6912
    Jacksonville, Florida 32224
    United States

    Indiana University Neuroscience Center
    Sandra Guingrich / .(JavaScript must be enabled to view this email address) / 317-963-7382
    Indianapolis, Indiana 46202
    United States

    University of Kansas Medical Center
    Jennifer Tuttle / .(JavaScript must be enabled to view this email address) / 913-588-5703
    Kansas City, Kansas 66160
    United States

    Johns Hopkins Hospital
    Kristen Riley / .(JavaScript must be enabled to view this email address) / 410-955-8511
    Baltimore, Maryland 21205
    United States

    Saint Louis University
    Susan Eller / .(JavaScript must be enabled to view this email address) / 314-977-4900
    Saint Louis, Missouri 63104
    United States

    Hospital for Special Surgery
    Shara Holzberg / .(JavaScript must be enabled to view this email address) / 646-797-8592
    New York, New York 10021
    United States

    Guilford Neurologic Associates
    Marsela Ferko / .(JavaScript must be enabled to view this email address) / 336-516-9106
    Greensboro, North Carolina 27405
    United States

    Wake Forest University
    Mozhdeh Marandi / .(JavaScript must be enabled to view this email address) / 336-713-8577
    Winston-Salem, North Carolina 27157
    United States

    Providence Brain and Spine Institute
    Kimberly Goslin / .(JavaScript must be enabled to view this email address) / 503-215-8580
    Portland, Oregon 97213
    United States

    Penn State Milton S. Hershey Medical Center
    Ruth Stewart / .(JavaScript must be enabled to view this email address) / 717-531-0003 ext 287666
    Hershey, Pennsylvania 17033
    United States

    Temple University
    Kathleen Hatala / .(JavaScript must be enabled to view this email address) / 215-707-4171
    Philadelphia, Pennsylvania 19140
    United States

    University of Pittsburgh
    David Lacomis / .(JavaScript must be enabled to view this email address) / 412-647-1706
    Pittsburgh , Pennsylvania 15213
    United States

    UT Health San Antonio
    Pamela Kittrell / .(JavaScript must be enabled to view this email address) / 210-450-0524
    San Antonio, Texas 78229
    United States

    The University of Utah
    Teresa Janecki / .(JavaScript must be enabled to view this email address) / 801-581-3724
    Salt Lake City, Utah 84112
    United States

    University of Vermont Medical Center
    Shannon Lucy / .(JavaScript must be enabled to view this email address) / 802-656-4582
    Burlington, Vermont 05405
    United States

    Saint Luke's Rehabilitation Institute
    Douglas Weeks / .(JavaScript must be enabled to view this email address) / 509-473-6234
    Spokane, Washington 99202
    United States

    Medical College of Wisconsin
    Lynn Wheeler / .(JavaScript must be enabled to view this email address) / 414-805-9307
    Milwaukee, Wisconsin 53226
    United States

Safety of Urate Elevation in Amyotrophic Lateral Sclerosis (SURE-ALS2)

Study Purpose:

This is a multi-center, 20-week study of inosine treatment.

Study Objectives and Endpoints The primary objective of the study is to determine the safety and tolerability of oral administration of inosine (administered daily) dosed to moderately elevate serum urate over 20 weeks.

The primary outcome measures will be

  • Safety, as measured by adverse events
  • Tolerability, defined as the ability of subjects to complete the entire 20-week study.

As an exploratory objective, we will test the feasibility and utility of a smartphone application for monitoring symptoms and disease progression in patients with amyotrophic lateral sclerosis (ALS).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Sabrina Paganoni, MD, PhD, Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):

NCT03168711

Neals Affiliated?

Yes

Coordinating Center Contact Information

Massachusetts General Hospital
Lindsay Pothier / .(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address) Boston, Massachusetts 02114 United States

Full Study Summary:

Amyotrophic lateral sclerosis (ALS) is a fatal, neurodegenerative disease for which there is no cure. Multiple lines of evidence have implicated oxidative stress in the pathophysiology of ALS. Urate (uric acid) is an endogenous antioxidant system, and urate may serve as a major defense against oxidative stress. Urate has emerged as a promising neuro-protectant and therapeutic target based on convergent epidemiological, laboratory, and clinical data in multiple neurodegenerative diseases, most notably Parkinson's disease (PD). In PD, urate elevation has been pursued as a potential therapy by administration of inosine, a urate precursor that is available as an over-the-counter supplement. Administration of inosine results in a predictable elevation of urate levels and has been shown to be safe and well tolerated in PD.

Analysis of ALS databases revealed that higher urate levels are an independent predictor of slower progression and prolonged survival in ALS. However, whether elevating urate in people with ALS would result in better outcomes is unknown.

The Principal Investigator has recently concluded a Pilot Study of Inosine in ALS, which was a short, open label, single center study involving 25 subjects [NCT02288091]. The study showed safety and feasibility of urate elevation in patients with ALS. The Principal Investigator is now pursuing a multi-center Phase II trial to confirm these findings with longer exposure time.

Study Sponsor:

Massachusetts General Hospital, The Salah Foundation, MGH cure ALS Fund

Participant Duration:

20 weeks

Estimated Enrollment:

30

Estimated Study Start Date:

10/01/2017

Estimated Study Completion Date:

01/01/2020

Posting Last Modified Date:

08/28/2019

Date Study Added to alsconsortium.org:

06/02/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    85

    Min Vital Capacity (% predicted normal):

    60

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:
    Age 18-85.
    Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria (Appendix 1).
    Slow vital capacity (SVC) ≥ 60% of predicted for age, height, and gender at the Screening Visit.
    Capable of providing informed consent and following trial procedures.
    Serum urate < 5.5 mg/dL at screening (i.e. below the population median serum urate levels).
    Women must not be able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and 3 months after study completion. Adequate contraception includes: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal (patch or contraceptive ring, for example) contraception), intrauterine device (IUD) in place for ≥ 3 months, barrier method in conjunction with spermicide, or another adequate method.
    Is able and willing to participate in the Mobile app study procedures.

    Exclusion Criteria:
    History of urolithiasis.
    Urine pH < 5.5 at screening (as acidic urine is a major determinant of uric acid urolithiasis).
    History of gout.
    History of stroke or myocardial infarction.
    History of symptomatic coronary artery disease (e.g. angina pectoris) or symptomatic peripheral arterial disease within 1 year prior to Screening.
    Symptomatic congestive heart failure with a documented ejection fraction below 45%.
    Poorly controlled arterial hypertension (SBP>160mmHg or DBP>100mmHg at Screening).
    Women who are pregnant or lactating.
    The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the subject to provide informed consent, according to Site Investigator judgment, or a history of active substance abuse within the prior year.
    Anything that, in the opinion of the Site Investigator, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.
    Use of the following within 30 days prior to Screening: inosine, allopurinol, probenecid, more than 300mg vitamin C daily (note that a subject may take a standard multivitamin up to one tablet or capsule daily). Use of thiazides is permissible as long as the subject is on a stable dose from 1 week prior to Screening.
    Known hypersensitivity or intolerability to inosine.

  • Site Contact Information

    Holy Cross Hospital
    Franco Salas / .(JavaScript must be enabled to view this email address) / 954-229-7965
    Lindita Burba / .(JavaScript must be enabled to view this email address) / 954-489-4314
    Fort Lauderdale, Florida 33308
    United States

    Massachusetts General Hospital
    Jianing (Ning) Liu / .(JavaScript must be enabled to view this email address) / 617-726-1880
    Boston, Massachusetts 02114
    United States

    University of Minnesota
    Valerie Ferment / .(JavaScript must be enabled to view this email address) / 612-301-1535
    Minneapolis, Minnesota 55455
    United States

An Open-Label Pilot Study Comparing the Efficacy of Selective Serotonin Re-Uptake Inhibitors (SSRIs) Versus Tricyclic Antidepressants (TCAs) for Treating Depression in Amyotrophic Lateral Sclerosis

Study Purpose:

Depression is seen in 9-11% of ALS patients and adequate and proper treatment is needed. In this study, ALS patients will be screened for depression using self-reported multiple choice questionnaire. Patients who fulfill the criteria for depression based on this screening tool will be evaluated by psychiatrist before inclusion in the study. The investigators will also measure quality of life and functional status by simple questionnaires. The patients will be allocated into two treatment groups to receive either TCA or SSRI for 12 weeks. Patients will be evaluated every 4 weeks and phone calls will be made in between the visits if needed to assess about efficacy and any side effects. If any patient reports having suicidal thoughts on any of these phone calls or clinic visits, he/she will be immediately sent to the ER for appropriate management. The investigators will repeat the questionnaires in the clinic visits, and use them in the data analysis to look for any improvement and to compare the two medication classes used in this study. This data may be used later on to do larger studies and help to make standard recommendations in treating depression in ALS patients.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Ghazala Hayat, St. Louis University

Clinicaltrials.gov ID (11 digit #):

NCT02851914

Neals Affiliated?

No

Coordinating Center Contact Information

Saint Louis University
Ghazala s Hayat, MD / .(JavaScript must be enabled to view this email address) / 314-977-4800
Gayatriben Gadani, MD / .(JavaScript must be enabled to view this email address) / 314-977-4800
1438 South Grand Blvd.
Saint Louis, Missouri 63104 United States

Full Study Summary:

This study is a 12-week, open-label, non-randomized, pilot clinical intervention trial. This is investigator initiated study.This trial will be done at St Louis University ALS clinic. ALS patients will be screened for depression using Beck depression inventory (BDI-II) scale. A mental healthcare provider will evaluate the patients scoring 19 or above, before inclusion in the study. Quality of Life (QOL) assessment by questionnaire (McGill) and ALS functional rating scale (ALS-FRS) measurement will be done at the baseline. Then these patients will be allocated into two treatment groups to receive either TCA or SSRI medication for 12 weeks based on the clinical judgment (non-randomized). Patients will require clinical encounters every 4 weeks and telephone encounters in between the visits to assess the effectiveness of medication and tolerability of the side effects if any. If any patient endorses active suicidal ideation on any of these assessments, he/she will be immediately sent to the ER for appropriate management. At 4, 8 and 12-week clinic visits, repeat BDI, QOL and ALS-FRS measurement will be done on each patient from both groups and used in the data analysis.

Study Sponsor:

St. Louis University

Participant Duration:

12 weeks

Estimated Enrollment:

40

Estimated Study Start Date:

07/01/2015

Estimated Study Completion Date:

12/31/2018

Posting Last Modified Date:

01/11/2018

Date Study Added to alsconsortium.org:

05/17/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    25

    Maximum Age:

    80

    Min Vital Capacity (% predicted normal):

    26

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?


    Inclusion Criteria:

    Documented diagnosis of definite or probable ALS
    Informed and written consent for enrollment in study
    Gender: both male and female
    Age: 25-80 years
    BDI score 19 or above
    Depression diagnosis by mental health provider

    Exclusion Criteria:

    History of psychotic disorder, premorbid bipolar depression
    ALS-FRS score < 26
    Cognitive impairment
    Currently on SSRIs or TCAs. However if for some reason they are off their treatment, then they can be enrolled in the study after a washout period of 30 days.
    Currently on other antidepressants such as monoamine oxidase inhibitors (MAOIs), selective norepinephrine re-uptake inhibitors (SNRIs) etc.

  • Site Contact Information

    Saint Louis University
    Cindy M Roseman, LPN / .(JavaScript must be enabled to view this email address) / 314-977-4829
    Peggy A Suzor / .(JavaScript must be enabled to view this email address) / 314-977-4822
    1438 South Grand Blvd.
    St. Louis, Missouri 63104
    United States

A Treatment Continuation Study for Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease Who Have Successfully Completed Study CMD-2016-001

Study Purpose:

Treatment extension study for ALS/MND patients who participated in phase 1 study CMD-2016-001, completed assessments following six 28-day cycles of treatment, and whom the Investigator considers would benefit from continued CuATSM treatment.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase I ,Phase II

Study Chair(s)/Principal Investigator(s):

Dominic Rowe, MD Macquarie University

Clinicaltrials.gov ID (11 digit #):

NCT03136809

Neals Affiliated?

No

Coordinating Center Contact Information


Kay Noel, PhD / .(JavaScript must be enabled to view this email address) / 415-444-9602
Craig Rosenfeld, MD / .(JavaScript must be enabled to view this email address) / 415-444-9602

Full Study Summary:

Treatment extension study for ALS/MND patients who participated in phase 1 study CMD-2016-001, completed assessments following six 28-day cycles of treatment, and whom the Investigator considers would benefit from continued CuATSM treatment. The same safety assessments as in the CMD-2016-001 study will be conducted after each cycle of treatment and the same efficacy assessments as in the CMD-2016-001 study will be conducted after every 3 cycles of treatment. Treatment will continue until the first to occur of (1) Investigator considers the patient is no longer deriving benefit from CuATSM treatment, (2) patient develops dependence on mechanical ventilation where dependence is defined as being unable to lie flat (supine) without it, unable to sleep without it, or daytime use, or (3) the patient withdraws consent.

Study Sponsor:

Collaborative Medicinal Development Pty Limited

Participant Duration:

24 months

Estimated Enrollment:

50

Estimated Study Start Date:

05/08/2017

Estimated Study Completion Date:

05/31/2019

Posting Last Modified Date:

05/17/2017

Date Study Added to alsconsortium.org:

05/17/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    75

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Signed informed consent prior to initiation of any study-specific procedures and treatment
    Documented completion of protocol-specific assessments following completion of six 28-day treatment cycles in study CMD-2016-001
    Principal Investigator considers the patient would benefit from continued treatment with Cu(II)ATSM
    Not taking riluzole or on the same (or lower) dose used during the CMD-2016-001 study
    Adequate bone marrow reserve, renal and liver function
    Women and men with partners of childbearing potential must take effective contraception while on study treatment

    Exclusion Criteria:

    Inability to swallow oral medications or presence of a gastrointestinal disorder (e.g.,malabsorption) deemed to jeopardize intestinal absorption of study drug
    Dependence on mechanical ventilation (invasive or non-invasive) for any part of day or night, where dependence is defined as being unable to lie flat (supine) without it, unable to sleep without it, or daytime use
    Dementia that may affect either outcome measures or patient understanding and/or compliance with study requirements and procedures
    Current use of strong inducers or inhibitors of CYPs 2C19 and 2D6

  • Site Contact Information

    Macquarie University
    Dominic Rowe, MD / .(JavaScript must be enabled to view this email address) / Sydney, Australia 2109
    Australia

Evaluation of the Safety, Tolerability, Efficacy and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for the Treatment of ALS (CENTAUR)

Study Purpose:

The CENTAUR trial will be a 2:1 (active:placebo) randomized, double-blind, placebo-controlled Phase II trial to evaluate the safety and efficacy of AMX0035 for the treatment of ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Study Director: Patrick Yeramian, MD, Amylyx Pharmaceuticals Inc.
Principal Investigator: Sabrina Paganoni, MD, PhD, Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):

NCT03127514

Neals Affiliated?

Yes

Coordinating Center Contact Information

Massachusetts General Hospital, NCRI
Andrea Ariza / .(JavaScript must be enabled to view this email address) / 617-724-3871
.(JavaScript must be enabled to view this email address) Boston, Massachusetts 02114 United States

Full Study Summary:

AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and able to slow decline in function as measured by the ALSFRS-R. The trial will also assess the effects of AMX0035 on muscle strength, vital capacity, and biomarkers of ALS including markers of neuronal death and neuroinflammation.

Study Sponsor:

Amylyx Pharmaceuticals Inc.

Participant Duration:

6 months

Estimated Enrollment:

132

Estimated Study Start Date:

05/31/2017

Estimated Study Completion Date:

05/31/2019

Posting Last Modified Date:

09/20/2019

Date Study Added to alsconsortium.org:

05/02/2017
  • More Information

    The lead Principal Investigator for the AMX0035 trial, Sabrina Paganoni, MD, PhD, Massachusetts General Hospital, has led three webinars on the CENTAUR trial. Click the webinar recording links below for more information about the study and how to enroll.

    Click here to watch the January 10, 2018 webinar recording.

    Click here to watch the May 7, 2018 webinar recording.

    Click here to watch the September 5, 2018 webinar recording.

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Min Vital Capacity (% predicted normal):

    60

    Time since Symptom Onset:

    <18 months

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Key Inclusion Criteria:
    Male or female, aged 18-80 years of age
    Sporadic or familial ALS diagnosed as definite as defined by the World Federation of Neurology revised El Escorial criteria
    Less than or equal to 18 months since ALS symptom onset
    Capable of providing informed consent and following trial procedures
    Slow Vital Capacity (SVC) >60% of predicted value for gender, height, and age at the Screening Visit
    Subjects must either not take riluzole or be on a stable dose of riluzole for at least 30 days prior to the Screening Visit. Riluzole-naïve subjects are permitted in the study.
    Women of child bearing potential (e.g. not post-menopausal for at least one year or surgically sterile) must agree to use adequate birth control for the duration of the study and 3 months after last dose of study drug. Women must not be planning to become pregnant for the duration of the study and 3 months after last dose of study drug
    Men must agree to practice contraception for the duration of the study and 3 months after last dose of study drug. Men must not plan to father a child or provide for sperm donation for the duration of the study and 3 months after last dose of study drug

    Key Exclusion Criteria:
    Presence of tracheostomy
    Exposure to PB, TUDCA or UDCA within 3 months prior to the Screening Visit or planning to use these medications during the course of the study
    History of known allergy to PB or bile salts
    Abnormal liver function defined as AST and/or ALT > 3 times the upper limit of the normal
    Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of normal
    Poorly controlled arterial hypertension (SBP>160mmHg or DBP>100mmHg) at the Screening Visit
    Pregnant women or women currently breastfeeding
    History of cholecystectomy
    Biliary disease which impedes biliary flow including active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gallbladder polyps, gangrene of the gallbladder, abscess of the gallbladder.
    History of Class III/IV heart failure (per New York Heart Association - NYHA)
    Severe pancreatic or intestinal disorders that may alter the enterohepatic circulation and absorption of TUDCA including biliary infections, pancreatitis and ileal resection
    The presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the subject to provide informed consent, according to Site Investigator judgment
    Clinically significant unstable medical condition (other than ALS) that would pose a risk to the subject if they were to participate in the study
    Active participation in an ALS clinical trial evaluating a small molecule within 30 days of the Screening Visit
    Exposure at any time to any biologic under investigation for the treatment of subjects with ALS (off-label use or investigational) including cell therapies, gene therapies, and monoclonal antibodies.
    Implantation of Diaphragm Pacing System (DPS)

  • Site Contact Information

    Barrow Neurological Institute
    Nicole Turcotte / .(JavaScript must be enabled to view this email address) / 602-406-4775
    Phoenix, Arizona 85013
    United States

    UC Irvine Medical Center
    Veena Mathew / .(JavaScript must be enabled to view this email address) / 714-456-2864
    Orange, California 92868
    United States

    Forbes Norris MDA/ALS Research Center - California Pacific Medical Center
    San Francisco, California 94114
    United States

    University of Florida Medical Center
    Jennifer Steshyn / .(JavaScript must be enabled to view this email address) / 352-273-9022
    Gainesville, Florida 32610
    United States

    Carol and Frank Morsini Center for Advanced Health Care - University of South Florida
    Brittany Harvey / .(JavaScript must be enabled to view this email address) / 813-974-9413
    Tampa, Florida 33612
    United States

    Emory University
    Janet Brantley / .(JavaScript must be enabled to view this email address) / 404-727-1673
    Atlanta, Georgia 30322
    United States

    University of Iowa Hospitals and Clinics
    Jen Sieren / .(JavaScript must be enabled to view this email address) / 319-356-8744
    Iowa City, Iowa 52242
    United States

    University of Kentucky Medical Center
    Meha Joshi / .(JavaScript must be enabled to view this email address) / 859-218-5046
    Lexington, Kentucky 40536
    United States

    Ochsner Neuroscience Institute
    Ashley Laroche / .(JavaScript must be enabled to view this email address) / 504-703-0755
    New Orleans, Louisiana 70121
    United States

    Johns Hopkins Hospital
    Kristen Riley / .(JavaScript must be enabled to view this email address) / 410-955-9036
    Baltimore, Maryland 21287
    United States

    Massachusetts General Hospital
    Aileen Shaughnessy / .(JavaScript must be enabled to view this email address) / 617-643-0801
    Boston, Massachusetts 02114
    United States

    University of Massachusetts Memorial Medical Center
    Diane McKenna-Yasek, RN, BSN / .(JavaScript must be enabled to view this email address) / 508-856-4697
    Worcester, Massachusetts 01655
    United States

    University of Michigan Medical Center
    Jayna Duell / .(JavaScript must be enabled to view this email address) / 734-763-9037
    Ann Arbor, Michigan 48109
    United States

    Hennepin County Medical Center
    Cindy Rohde / .(JavaScript must be enabled to view this email address) / 612-873-2607
    Twin Cities
    Minneapolis, Minnesota 55415
    United States

    Washington University Medical Center
    Jennifer Jockel-Balsarotti / .(JavaScript must be enabled to view this email address) / 314-362-8624
    Saint Louis, Missouri 63110
    United States

    Neurology Associates P.C.
    Desi Eschiti / .(JavaScript must be enabled to view this email address) / 402-486-3430
    Lincoln, Nebraska 68506
    United States

    Mount Sinai Beth Israel
    Emily Ripps / .(JavaScript must be enabled to view this email address) / 212-844-6188
    New York, New York 10003
    United States

    Wake Forest Baptist Medical Center
    Mozhdeh Marandi / .(JavaScript must be enabled to view this email address) / 336-713-8577
    Winston-Salem, North Carolina 27157
    United States

    The Ohio State University Wexner Medical Center
    Ifeanyi Okoh / .(JavaScript must be enabled to view this email address) / 614-688-7837
    Columbus, Ohio 43221
    United States

    Oregon Health & Science University
    Diana Dimitrova / .(JavaScript must be enabled to view this email address) / 503-494-7269
    Portland, Oregon 97239
    United States

    The Penn Comprehensive ALS Center
    Kelly Almasy / .(JavaScript must be enabled to view this email address) / 215-829-5041
    Philadelphia, Pennsylvania 19107
    United States

    Temple University Hospital
    Kathleen Hatala / .(JavaScript must be enabled to view this email address) / 215-707-4171
    Philadelphia, Pennsylvania 19140
    United States

    Texas Neurology, P.A.
    Todd Morgan / .(JavaScript must be enabled to view this email address) / 214-827-3610, ext. 228
    Dallas, Texas 75214
    United States

    University of Texas Health Science Center at San Antonio
    Pamela Kittrell, RN, MSN, CCRC / .(JavaScript must be enabled to view this email address) / 210-450-0524
    San Antonio, Texas 78229
    United States

    ALS Center at the Swedish Neuroscience Institute
    Lindsey Maassel / .(JavaScript must be enabled to view this email address) / 206-320-7121
    Seattle, Washington 98122
    United States

Longitudinal Study of Innate Lymphoid Cells in Peripheral Blood in ALS

Study Purpose:

Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disorder characterized by progressive muscle weakness and eventual death. Studies demonstrate that the immune system plays a key role in ALS progression; however, the role of the immune system is unclear, as various aspects can play both a beneficial and detrimental role in the disease course. Attempts to universally suppress the immune system in ALS patients have at best had negligible effects on progression or at worst accelerated the disease. Thus, there is a critical need to identify immune cell populations to serve as biomarkers and therapeutic targets.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Healthy Volunteer

Study Type:

Observational Study

Study Category:

Blood draw

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Ben Murdock, PhD, University of Michigan

Clinicaltrials.gov ID (11 digit #):

NCT03090932

Neals Affiliated?

No

Coordinating Center Contact Information

University of Michigan
Blake Swihart / .(JavaScript must be enabled to view this email address) / 734-763-8284
.(JavaScript must be enabled to view this email address) Ann Arbor, Michigan 48109 United States

Full Study Summary:

Study Sponsor:

University of Michigan

Participant Duration:

Estimated Enrollment:

25

Estimated Study Start Date:

01/18/2016

Estimated Study Completion Date:

07/01/2018

Posting Last Modified Date:

05/02/2017

Date Study Added to alsconsortium.org:

05/02/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Age 18 years or older.
    Fluency in English at the 6th grade level or higher.
    Able to communicate sufficiently well by speaking
    Able to communicate over the phone.
    Capable of providing informed consent.
    Lives within 1 hour of the University of Michigan

    Exclusion Criteria:

    Definite, probable, or possible ALS diagnosis
    Unable to provide informed consent.
    Clinically significant dementia, as judged by the site investigator.
    Other neurological or psychiatric disorders which are expected to impair cognitive function.
    Other serious and uncontrolled medical disorders.
    History of autoimmune disease.
    Use of prednisone, IVIG, or immunosuppression within the last 12 months.
    Lives more than 1 hour from the University of Michigan

  • Site Contact Information

    University of Michigan
    Blake Swihart / 734-763-8284
    Ann Arbor, Michigan 48109
    United States

ALS Testing through Home-Based Outcome Measures (ALS AT HOME)

Study Purpose:

The purpose of this study is two-fold: to assess the abililty of ALS patients to take their own meaningful outcome measures at home, and to determine whether frequent sampling can increase the reliability of the outcome measures tests.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Healthy Volunteer

Study Type:

Observational Study

Study Category:

Biomarkers/Imaging

Study Status:

Not enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Dr. Jeremy Shefner, MD, PhD, Barrow Neurological Institute

Clinicaltrials.gov ID (11 digit #):

NCT03016897

Neals Affiliated?

Yes

Coordinating Center Contact Information

Barrow Neurological Institute
Kerisa Shelton, PhD / .(JavaScript must be enabled to view this email address) / 602-406-6598
.(JavaScript must be enabled to view this email address) 240 W. Thomas Rd.
Suite 400
Phoenix, Arizona 85013 United States

Full Study Summary:

Barrow Neurological Institute is seeking individuals to participate in a remote research study called ALS AT HOME, enrolling under the direction of Dr. Jeremy Shefner.

In the ALS AT HOME study we hope to show: 1) that ALS patients can evaluate their own function at home, and 2) that taking frequent measures of function will improve the consistency of the tests we use.  If we can demonstrate these things, future research studies may become faster and easier to complete.

This study will involve up to 250 participants; 220 of the participants will be ALS patients, and 30 participants will be healthy individuals. To be eligible for participation, you must be between the ages of 18 and 85, have a Smart Device with Bluetooth capabilities, and have continuous internet access at home.

All of the activities for this study will take place at your home. If you are found to be eligible and are selected for the study, we will ship you the supplies you will need to take your own measurements at home. You will be trained in using the equipment via an online webinar, and you will be instructed to enter data into the study website. Total participation time for this study is nine months. For the first three months you will be asked to enter data daily (this will take approximately 45 minutes per day), and for the last six months you will enter data twice a week. After the study is over, the equipment you were given for the study will be yours to keep.

Study Sponsor:

Barrow Neurological Institute

Participant Duration:

9 Months

Estimated Enrollment:

250

Estimated Study Start Date:

01/01/2017

Estimated Study Completion Date:

12/31/2018

Posting Last Modified Date:

08/15/2018

Date Study Added to alsconsortium.org:

02/27/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    85

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    >36 months

    Can participants use Riluzole?

    Yes


    Own a smartphone with Bluetooth capabilities, continuous internet access at home, willingness to comply with study protocol

  • Site Contact Information

    Barrow Neurological Institute
    Kerisa Shelton, PhD / .(JavaScript must be enabled to view this email address) / 602-406-6598
    Phoenix, Arizona 85013
    United States

Delineating Physiologic Mechanisms of Swallowing Impairment and Decline in ALS

Study Purpose:

Individuals with Amyotrophic Lateral Sclerosis are at high risk for swallowing impairment (dysphagia) which leads to malnutrition, decreased pulmonary health, aspiration and aspiration pneumonia. These sequelae necessitate timely identification of at risk individuals to ensure optimal management of oral intake and pulmonary function. The purpose of this study is to evaluate the discriminant ability of several non-invasive screening tools at detecting swallowing impairment in individuals with ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Observational Study

Study Category:

Device , 

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Emily Plowman, PhD, University of Florida

Clinicaltrials.gov ID (11 digit #):

NCT02962050

Neals Affiliated?

No

Coordinating Center Contact Information

University of Florida
Raele Robison, MS / .(JavaScript must be enabled to view this email address) / 610-504-4605
Lauren Tabor, MS / .(JavaScript must be enabled to view this email address) / 443-536-1234
Gainesville, Florida 32605 United States

Full Study Summary:

This research study is being performed to determine screening tools or tests that are able to identify and track swallowing problems associated with Amyotrophic Lateral Sclerosis (ALS) over time. Also, this study will provide insight into the natural progression of swallowing impairment in persons with ALS over time.

Participants enrolled in this study will complete one evaluation at the University of Florida Swallowing Systems Core laboratory located at Shands Hospital, Gainesville every three months. Each evaluation will take approximately 90 minutes. During these evaluations, a videofluoroscopy (X-ray of swallowing) examination, cough tests, tongue function test and questionnaires will be completed.

Study Sponsor:

University of Florida

Participant Duration:

Estimated Enrollment:

100

Estimated Study Start Date:

06/01/2017

Estimated Study Completion Date:

06/30/2024

Posting Last Modified Date:

09/01/2017

Date Study Added to alsconsortium.org:

02/24/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    90

    Time since Symptom Onset:

    Time since Diagnosis:

    <12 months

    Can participants use Riluzole?


    Inclusion Criteria:

    diagnosis of probable or definite ALS
    diagnosis within 120 days

    Exclusion Criteria:

    allergies to barium or capsaicin
    History of stroke
    Head and Neck Cancer
    Other disorder that might contribute to swallowing impairment
    Not enrolled in other research investigations that might impact swallowing
    Not pregnant

  • Site Contact Information

    University of Florida
    Raele Robison, MS / .(JavaScript must be enabled to view this email address) / 610-504-4605
    Lauren Tabor, MS / .(JavaScript must be enabled to view this email address) / 443-536-1234
    Gainesville, Florida 32605
    United States

    University of Florida College of Medicine
    Jacksonville , Florida 32209
    United States

    University of South Florida
    Tampa, Florida 33620
    United States

CC100: Phase 1 Multiple-Dose Safety and Tolerability in Subjects With ALS (CC100B)

Study Purpose:

Approximately 21 subjects with amyotrophic lateral sclerosis (ALS) will be randomized (6 to 1) to receive by mouth seven morning doses of CC100 or placebo for 7 days. Subjects are required to stay in the Clinic for approximately 9 hours following the first and last dose. Subjects will also have a mid-week clinic visit and will be contacted by phone within 3 to 5 days after the last dose.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Robert Pascuzzi, MD, Indiana University

Clinicaltrials.gov ID (11 digit #):

NCT03049046

Neals Affiliated?

No

Coordinating Center Contact Information

Indiana University, IU Health Physicians Neurology
Sandra Guingrich, LPN, CCRC / .(JavaScript must be enabled to view this email address) / 317-963-7382
.(JavaScript must be enabled to view this email address) Indianapolis, Indiana 46202 United States

Full Study Summary:

Primary objective: to assess the safety and tolerability of multiple doses of orally administered CC100 in subjects with amyotrophic lateral sclerosis (ALS). Secondary objectives: to determine pharmacokinetics and pharmacodynamics of CC100 in plasma after single and after multiple doses; and to determine short-term effects of CC100 on potential blood-cell ALS biomarkers.

Study Design: Phase 1 double-blind, randomized, placebo-controlled multiple-dose of three CC100-dose cohorts. Approximately 18 subjects will receive CC100. Approximately 3 subjects will be randomized to placebo (across 3 cohorts). Periodic Assessment Committee safety reviews. Note: Participation will not exclude subjects from future CC100 studies Criteria for Evaluation: Safety Endpoints: Adverse events, blood chemistry, hematology, urinalysis, vital signs, 12-lead ECGs. Pharmacokinetic (PK)/Pharmacodynamic (PD): Plasma for CC100 concentrations (PK). Blood collected at baseline and after each subject's last dose will be assayed for potential biomarker(s). Stored specimens will be de-identified or combined for validating diagnostic tools/assays related to ALS. Statistical Methods: A minimum of 6 subjects per CC100 dose group and 3 placebo-dosed subjects (total across cohorts) are considered sufficient to evaluate initial safety and tolerability for the cohorts. Pharmacokinetic parameter estimates will be calculated by standard noncompartmental methods of analysis. Absolute bioavailability of administration will be estimated based on the total area under the time- concentration curve (AUC0-∞).

Study Sponsor:

Chemigen, LLC

Participant Duration:

Subjects will receive by mouth seven morning doses of CC100 or placebo for 7 days. Subjects are required to stay in the Clinic for approximately 9 hours following the first and last dose. Subjects will also have a mid-week clinic visit and will be contacted by phone within 3 to 5 days after the last dose.

Estimated Enrollment:

21

Estimated Study Start Date:

02/01/2017

Estimated Study Completion Date:

09/30/2017

Posting Last Modified Date:

09/17/2019

Date Study Added to alsconsortium.org:

02/24/2017
  • Eligibility Criteria

    Gender:

    Female

    Minimum Age:

    18

    Maximum Age:

    64

    Min Vital Capacity (% predicted normal):

    60

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Have definite or probable ALS with a forced vital capacity of >60% predicted.
    Men must practice a reliable method of birth control during study and for 2 weeks following study. Women must be non-fertile or post-menopausal.
    Riluzole is allowed if dose has been stable for at least 30 days. Other allowed medications: lipid-lowering drugs, anti-hypertensives, anti-depressants, oral medications for type II diabetes, estrogen replacement therapy, thyroid replacement therapy, antihistamines, antacids, nonsteroidal anti-inflammatory drugs (except indomethacin), histamine H2-receptor antagonists, proton-pump inhibitors, calcium supplements, topical eye medications, and topical antibiotics.

    Exclusion Criteria:

    Greater than 250 pounds
    Have serious or unstable illnesses as determine by the investigator.
    Have current or a history of asthma or severe drug allergies or pollen allergy.
    Have had serious infectious disease affecting the brain within the preceding 5 years; or have existing evidence of serious infection.
    Have laboratory test values that are considered clinically significant as determined by the investigators.
    Have ECG abnormalities that are clinically significant.
    Have donated blood (a pint or more) or received an experimental drug within 30 days prior to dosing.
    Have a history of chronic alcohol or drug abuse within the past 2 years.

  • Site Contact Information

    Indiana University
    Sandra Guingrich, LPN, CCRC / .(JavaScript must be enabled to view this email address) / 317-963-7382
    Indianapolis, Indiana 46202
    United States

A Clinical Trial to Evaluate the Safety and Efficacy of Fycompa in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

This is a pilot trial to test perampanel (Fycompa; Eisai, Inc.) in ALS patients. The investigators will focus on safety and preliminary signs of efficacy. Perampanel is approved by the FDA for treatment of seizures in patients with epilepsy. In this study, perampanel will be used off-label for adults with ALS at an oral medication dose on the low end of the recommended dose range for epilepsy. This study will consist of two treatments arms: perampanel and matching placebo randomized at a 1:1 ratio. Subjects will receive medication for 9 months.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Nurcan Gursoy, MD, Stony Brook University Medical Center

Clinicaltrials.gov ID (11 digit #):

NCT03020797

Neals Affiliated?

No

Coordinating Center Contact Information

Stony Brook University Medical Center
Diana Kaell, BA / .(JavaScript must be enabled to view this email address) / 631-444-7832
Nurcan Gursoy, MD / .(JavaScript must be enabled to view this email address) / 631-444-2599
Stony Brook
New York, New York 11794 United States

Full Study Summary:

Study Sponsor:

Stony Brook University

Participant Duration:

Subjects will receive medication for 9 months.

Estimated Enrollment:

60

Estimated Study Start Date:

12/01/2016

Estimated Study Completion Date:

12/31/2018

Posting Last Modified Date:

Date Study Added to alsconsortium.org:

02/24/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Min Vital Capacity (% predicted normal):

    60

    Time since Symptom Onset:

    <36 months

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    diagnosis of ALS
    first clinical weakness within past 3 years
    slow vital capacity >= 60% of predicted within 1 month of treatment
    may be on stable dose of riluzole for at least 30 days, or otherwise agree to not initiate riluzole for duration of the trial
    can travel to Stony Brook to receive medical care
    must have a monitor who can be contacted at regular intervals to report on subject's clinical/psychiatric status

    Exclusion Criteria:

    use of tracheostomy or mechanical ventilation within last 3 months
    hepatic insufficiency or abnormal liver function
    renal insufficiency
    clinically significant psychiatric disorder
    history of malignancy < 5 years prior to entry
    history of HIV, clinically significant chronic hepatitis, or other active infection
    history of stomach or intestinal surgery or condition that could interfere with absorption, distribution, metabolism or secretion of study drug
    history of alcohol or substance abuse within 3 months prior to entry (subjects will be instructed to refrain from alcohol during the study)
    use of strong cytochrome P4503A inhibitors or inducers, anticonvulsants or other drugs known to interact strongly with perampanel.
    pregnancy or lactation
    clinically significant medical condition (other than ALS) that would pose a risk to the subject if they were to participate
    know hypersensitivity to perampanel
    currently participating, or has participated in a study with an investigation or marketed compound within 3 months of entry

  • Site Contact Information

    Stony Brook
    Diana Kaell, BA / .(JavaScript must be enabled to view this email address) / 631-444-7832
    New York , New York 11794
    United States

A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients With Amyotrophic Lateral Sclerosis (ALS) Who Completed VITALITY-ALS (CY 4031) (VIGOR-ALS)

Study Purpose:

The purpose of this study is to assess the long-term safety and tolerability of tirasemtiv in patients with ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase III

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT02936635

Neals Affiliated?

No

Coordinating Center Contact Information


.(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Enrolled participants will begin dosing of tirasemtiv 125 mg twice daily (250 mg/day) for a period of 4 weeks and will titrate to their tolerated dose, the maximum dose being 250 mg twice daily (500 mg/day). This study will also compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 with those who completed treatment with placebo in CY 4031 during continued treatment of both groups with tirasemtiv during CY 4033, compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 during that study with their clinical course during continued treatment with tirasemtiv during CY 4033, and compare the clinical course of patients who completed treatment with placebo in CY 4031 during that study with their clinical course during treatment with tirasemtiv during CY 4033.

Study Sponsor:

Cytokinetics

Participant Duration:

Estimated Enrollment:

350

Estimated Study Start Date:

10/01/2016

Estimated Study Completion Date:

10/01/2019

Posting Last Modified Date:

06/13/2017

Date Study Added to alsconsortium.org:

12/16/2016
  • More Information

    Primary Outcome Measures:

    Incidence of adverse events (AEs) in patient population [ Time Frame: Until end of study, up to 36 months ] [ Designated as safety issue: No ]


    Secondary Outcome Measures:

    Time to first use of assisted ventilation or death [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ] [ Designated as safety issue: No ]
    Time to the first occurrence of respiratory insufficiency (defined as tracheostomy or the use of non-invasive ventilation (NIV) for ≥22 hours per day for ≥10 consecutive days) or death [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ] [ Designated as safety issue: No ]
    Time to death [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ] [ Designated as safety issue: No ]
    Decline in percent predicted Slow Vital Capacity (SVC) from baseline [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ] [ Designated as safety issue: No ]
    Decline in ALS Functional Rating Scale - Revised (ALSFRS-R) score from baseline [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ] [ Designated as safety issue: No ]
    Slope of the change from baseline in percent predicted SVC [ Time Frame: First 24 weeks and first 48 weeks of either CY 4031 or CY 4033 ] [ Designated as safety issue: No ]
    Slope of the change from baseline in ALSFRS-R [ Time Frame: First 24 weeks and first 48 weeks of either CY 4031 or CY 4033 ] [ Designated as safety issue: No ]

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Able to comprehend and willing to sign an Informed Consent Form (ICF). If verbal consent is given, a Legal Designee of the patient must sign the ICF form
    Completed participation on study drug and the Follow-Up Visit in the CY 4031 study

    Male patients, who have not had a vasectomy AND confirmed zero sperm count, must agree for the duration of their participation in the study to either:

    Use a condom during sexual intercourse with female partners who are of childbearing potential AND to have female partners use a highly effective means of contraception OR
    Abstain from sexual intercourse during participation in the study

    Female patients who are not post-menopausal (≥ 1 year) or sterilized, must:

    Not be breastfeeding
    Have a negative pregnancy test
    Have no intention to become pregnant during participation in the study AND
    Practice sexual abstinence, defined as refraining from intercourse during the duration of the study OR if male partners are not vasectomized with a confirmed zero sperm count, require use of a condom AND use of a highly effective contraceptive measure

    Exclusion Criteria:

    Has a diaphragm pacing system (DPS) at study entry or anticipate DPS placement during the course of the study
    Has taken an investigational study drug (other than tirasemtiv) prior to dosing, within 30 days or five half-lives of the prior agent, whichever is greater
    Use of tizanidine and theophylline-containing medications during study participation
    Participation or planning to participate in any form of stem cell therapy for the treatment of ALS or another investigational drug

  • Site Contact Information

    Barrow
    Phoenix , Arizona 85013
    United States

    University of California, Irvine
    Orange, California 92868
    United States

    UC Davis Medical Center
    Sacramento, California 95817
    United States

    Forbes Norris MDA/ALS Research Center
    San Francisco, California 94115
    United States

    University of Colorado Hospital Anschutz Outpatient Pavilion
    Aurora, Colorado 80045
    United States

    Hospital for Special Care
    New Britain, Connecticut 06053
    United States

    George Washington University Medical Center
    Washington, D.C., District of Columbia 20037
    United States

    University of South Florida, Carol & Frank Morsani Center for Advanced Health Care
    Tampa, Florida 33512
    United States

    Mayo Clinic
    Jacksonville, Florida 32224
    United States

    University of Miami, Miller School of Medicine
    Miami, Florida 33136
    United States

    Indiana University
    Indianapolis, Indiana 46202
    United States

    University of Iowa Hospitals and Clinics
    Iowa City, Iowa 52242
    United States

    University of Kansas Medical Center
    Kansas City, Kansas 66160
    United States

    Johns Hopkins University
    Baltimore, Maryland 21287
    United States

    University of Massachusetts
    Worcester, Massachusetts 01655
    United States

    Henry Ford Health System
    Detroit, Michigan 48202
    United States

    Hennepin County Medical Center
    Minneapolis, Minnesota 55415
    United States

    St. Louis University
    Saint Louis, Missouri 63104
    United States

    Barnes-Jewish Hospital
    Saint Louis, Missouri 63110
    United States

    Dartmouth Hitchcock Medical Center
    Lebanon, New Hampshire 03756
    United States

    Hospital for Special Surgery
    New York, New York 10021
    United States

    Neurological Institute
    New York, New York 10032
    United States

    SUNY Upstate Medical University
    Syracuse, New York 13210
    United States

    Neurosciences Institute, Neurology - Charlotte
    Charlotte, North Carolina 28207
    United States

    Duke Neurological Disorders Clinic
    Durham, North Carolina 27705
    United States

    Department of Neurology, Wake Forest School of Medicine
    Winston-Salem, North Carolina 27157
    United States

    The Ohio State University Wexner Medical Center
    Columbus, Ohio 43210
    United States

    Providence Brain and Spine Inst. ALS Center
    Portland, Oregon 97213
    United States

    Penn State Milton S. Hershey Medical Center
    Hershey, Pennsylvania 17033
    United States

    Temple University School of Medicine
    Philadelphia, Pennsylvania 19140
    United States

    Vanderbilt University Medical Center - Clinical Research Center
    Nashville, Tennessee 37232
    United States

    Texas Neurology, PA
    Dallas, Texas 75214
    United States

    UTHSCSA - First Outpatient Research Unit
    San Antonio, Texas 78229
    United States

    University of Virginia Health System
    Charlottesville, Virginia 22908
    United States

    West Virginia University Hospitals
    Morgantown, West Virginia 26506
    United States

    Froedtert Memorial Lutheran Hospital
    Milwaukee, Wisconsin 53226
    United States

    Montreal Neurological Institute and Hospital
    Montreal, Quebec
    Canada

    McMaster University Medical Centre
    Hamilton, Ontario
    Canada

    University of Calgary
    Calgary, Alberta
    Canada

    University of Alberta
    Edmonton, Alberta
    Canada

    London Health Sciences Centre
    London, Ontario
    Canada

    Sunnybrook Health Sciences Center
    Toronto, Ontario
    Canada

    Hopital Notre-Dame/CHUM
    Montreal, Quebec
    Canada

    CHU de Quebec - Univerite' Laval
    Quebec
    Canada

    Clinical Research Centre Beaumont Hospital
    Dublin, Ireland
    Ireland

Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

Study Purpose:

The purpose of this study is to determine whether nebulized RNS60 is effective in the treatment of amyotrophic lateral sclerosis (ALS).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not yet enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT02988297

Neals Affiliated?

No

Coordinating Center Contact Information


.(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Revalesio Corporation

Participant Duration:

Estimated Enrollment:

140

Estimated Study Start Date:

01/01/2018

Estimated Study Completion Date:

01/01/2021

Posting Last Modified Date:

12/16/2016

Date Study Added to alsconsortium.org:

12/16/2016
  • More Information

    Primary Outcome Measures:

    The mean change of the ALS functional rating scale-revised (ALSFRS-R) total score [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]

    Secondary Outcome Measures:

    The cumulative proportion of deaths or tracheostomies [ Time Frame: 28 weeks ] [ Designated as safety issue: No ]
    The mean change in the proportion of regulatory T cells (Treg) [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
    The mean change of the slow vital capacity (SVC) score [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
    The mean change of the ALS assessment questionnaire (ALSAQ-40) score [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
    The mean number of adverse events (AEs) [ Time Frame: 28 weeks ] [ Designated as safety issue: Yes ]

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Time since Symptom Onset:

    Time since Diagnosis:

    <36 months

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Probable, probable-laboratory supported, or definite ALS, either sporadic or familial according to modified El Escorial criteria
    Disease duration < 3 years
    Age 18 to 80
    Able to provide informed consent and to comply with study procedures
    Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to screening (riluzole-naïve participants are permitted in the study)
    Women must not be lactating or able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control) for the duration of the study, and for 3 months after study completion
    Men should practice contraception for the duration of the study and for 3 months after completion

    Exclusion Criteria:

    Diagnosis of a motor neuron disease other than ALS (e.g., primary lateral sclerosis, progressive muscular atrophy, progressive bulbar palsy)
    Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant if they were to participate in the study or that would impact survival or functional disability in the next 12 months
    Abnormal liver function defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 3 times the upper limit of the normal
    Renal insufficiency (Glomerular Filtration Rate < 60)
    Active pulmonary disease
    Prior poor compliance with an inhalation device
    The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the participant to provide informed consent, according to judgment by the principal investigator.
    History of human immunodeficiency virus (HIV) infection, clinically significant chronic hepatitis, or other active infection.
    Active participation in another ALS clinical trial within 30 days of the Screening Visit
    Other experimental treatments or anti-inflammatory/immunomodulatory medications used in the preceding 3 months

  • Site Contact Information

Feasibility and Sensitivity of a Symptom Monitoring Application in Real Time (SMART) for ALS

Study Purpose:

The purpose of this study is to determine if a smartphone application would be helpful in collecting research data to better inform us about ALS disease progression. This study asks each participant to use a smartphone application for a few minutes each week to answer a questionnaire and complete a voice recording. This is a pilot study in which a smartphone application was designed and customized for use in ALS clinical trials.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Observational Study

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable