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

Study Purpose:

REFINE-ALS is a prospective, observational, longitudinal, multicenter study designed to identify biomarkers to serve as quantifiable biological non-clinical measures of Edaravone effects in ALS. Epigenetic and protein biomarkers will also be investigated.

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 #):

NCT04259255

Neals Affiliated?

Yes

Coordinating Center Contact Information

Massachusetts General Hospital
Matthew Davis / .(JavaScript must be enabled to view this email address) / 617-724-4246
Sofia DiStefano / .(JavaScript must be enabled to view this email address) / 617-724-3314
Neurological Clinical Research Institute
165 Cambridge Street
Boston, Massachusetts 02114 United States

Full Study Summary:

Treatment will be prescribed by HCPs in accordance with their clinical judgement and the prescribing information for Edaravone. The decision to prescribe Edaravone to the participants should be made separately from the decision to enroll then in the study. There will be no randomized assignments to treatment and no restrictions on the use of commercially available medications (but those participating in an experimental study, even if taking Edaravone, will be excluded). No experimental treatment is evaluated in this study. The intervention is limited to the collection of blood and urine samples for biomarker testing.

During the estimated study period, eligible patients who are prescribed Edaravone within the approved indication will be invited to participate in the study. An initial screening/baseline visit will be scheduled for participants who are considered for study participation.

Participants in this study will be followed from enrollment up to 24 weeks after treatment initiation (6 treatment cycles [each cycle consisting of 28 days], corresponding to a treatment period of approximately 24 weeks) or premature study discontinuation. Throughout the study period, the investigators will record participant baseline and follow-up information and perform clinical and biomarker assessments.

Study Sponsor:

Mitsubishi Tanabe Pharma America, Inc.

Participant Duration:

 24 weeks

Estimated Enrollment:

300

Estimated Study Start Date:

10/21/2019

Estimated Study Completion Date:

02/28/2022

Posting Last Modified Date:

02/13/2020

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


    Study Population
    This study will be conducted in participants who have sporadic or familial amyotrophic lateral sclerosis (ALS) as defined by revised El Escorial criteria. Participants must provide written informed consent prior to screening. At screening, eligible patient must be at least 18 years old with a decision made to prescribe Edaravone prior to consenting. Participants who are either Edaravone naïve or who did not receive any Edaravone dose within one month of consenting are eligible for inclusion given they meet all other protocol requirements.

    Inclusion Criteria:

    • Male and female aged 18 years or older at enrollment
    • 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
    • Decision made to prescribe Edaravone prior to screening
    • Participant will likely be able to obtain commercial Edaravone and likely to complete 6 cycles of treatment, per site investigator estimation
    • Participant either naïve to Edaravone or who did not receive any Edaravone does within 1 month prior to screening
    • 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:

    • Participant with a contraindication to Edaravone
    • Participant is participating in an interventional clinical trial

  • Site Contact Information

    Barrow Neurological Institute
    Nicole Turcotte / .(JavaScript must be enabled to view this email address) / Shaundra Hall / .(JavaScript must be enabled to view this email address) / 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
    Jessica Bercow / .(JavaScript must be enabled to view this email address) / 310-825-6930
    Los Angeles, California 90095
    United States

    UC Irvine
    Vivian Li / .(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) / 415-600-3758
    Simon Carty / .(JavaScript must be enabled to view this email address) / 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
    Neurology Research Group / .(JavaScript must be enabled to view this email address) / Denver, Colorado 80309
    United States

    Georgetown University Medical Center
    Alexis Ahmad / .(JavaScript must be enabled to view this email address) / Washington, District of Columbia 20007
    United States

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

    University of South Florida
    Allison Schleutker / .(JavaScript must be enabled to view this email address) / Tampa, Florida 33612
    United States

    University of Florida, Jacksonville
    Alyssa Ruckel / .(JavaScript must be enabled to view this email address) / Lisa Smith / .(JavaScript must be enabled to view this email address) / 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
    Hellen Tanui / .(JavaScript must be enabled to view this email address) / 913-945-9934
    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
    Diane McKenna-Yasek / .(JavaScript must be enabled to view this email address) / 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

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

    HealthPartners Institute
    Maria Pyle / .(JavaScript must be enabled to view this email address) / 651-495-6371
    Emily Green / .(JavaScript must be enabled to view this email address) / Twin Cities ALS Consortium
    Saint Paul, Minnesota 55130
    United States

    University of Missouri Health Care
    Raghav Govindarajan, MD, FAAN / .(JavaScript must be enabled to view this email address) / Columbia , Missouri 65212
    United States

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

    Saint Louis University
    Susan Brown / .(JavaScript must be enabled to view this email address) / 314-977-4900
    Christina Morefield / .(JavaScript must be enabled to view this email address) / 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

    Las Vegas Clinic
    Kaitlyn Fell / .(JavaScript must be enabled to view this email address) / Las Vegas, Nevada 89145
    United States

    Columbia University
    Brixhilda Dedi / .(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

    OhioHealth
    Elizabeth Ferguson / .(JavaScript must be enabled to view this email address) / Columbus, Ohio 43215
    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
    Carol VonHofen / .(JavaScript must be enabled to view this email address) / 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
    Rachel Applegate / .(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

    University of Utah
    Teresa Janecki / .(JavaScript must be enabled to view this email address) / Salt Lake City , Utah 84112
    United States

    Swedish Medical Center
    Jennifer Cardey / .(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
    Marie Mejaki / .(JavaScript must be enabled to view this email address) / 414-805-9307
    Milwaukee, Wisconsin 53226
    United States