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HEALEY ALS Platform Trial

Study Purpose:

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.

Disease:

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

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II, Phase III

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

Merit Cudkowicz, MD Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):

NCT04297683

Neals Affiliated?

Yes

Coordinating Center Contact Information

Healey Center for ALS at Massachusetts General Hospital
Platform Trial Research Navigator / .(JavaScript must be enabled to view this email address) / 833-425-8257 (HALT ALS)
.(JavaScript must be enabled to view this email address) Boston, Massachusetts 02114 United States

Full Study Summary:

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial.

In this trial, multiple investigational products for ALS will be tested simultaneously or sequentially. Each investigational product will be tested in a regimen. Each regimen consists of a placebo-controlled trial, meaning that the active investigational product and matching placebo will be tested in each regimen.

The additional details that govern the testing of each investigational product will be summarized in separate regimen-specific appendices (RSAs). Each regimen will have a separate ClinicalTrials.gov posting, which will include specific information about the regimen. All regimen-specific outcome measures will be detailed in each regimen posting.

Participants will have an equal chance to be randomized to all regimens that are active at the time of screening. Once randomized to a regimen, participants will be randomized in a 3:1 ratio to either study drug or placebo.

The following regimens are active in the trial:

Regimen A - Zilucoplan Regimen B - Verdiperstat Regimen C - CNM-Au8 Regimen D - Priodopidine Regimen E - SLS-005 Trehalose

New regimens will be continuously added as new investigational products become available. The HEALEY ALS Platform Trial will enroll additional participants as each new regimen is available.

Study Sponsor:

Merit E. Cudkowicz, MD

Participant Duration:

Estimated Enrollment:

640

Estimated Study Start Date:

07/01/2020

Estimated Study Completion Date:

11/30/2022

Posting Last Modified Date:

08/25/2022

Date Study Added to alsconsortium.org:

03/09/2020
  • More Information

    Visit the Sean M. Healey & AMG Center for ALS at Mass General website for more information: www.massgeneral.org/neurology/als/research/platform-trial

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    <36 months

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    • Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS defined by revised El Escorial criteria.
    • Age 18 years or older.
    • Capable of providing informed consent and complying with study procedures, in the SI's opinion.
    • Time since onset of weakness due to ALS ≤ 36 months at the time of the Master Protocol Screening Visit.
    • SVC ≥ 50% of predicted capacity for age, height, and sex at the time of the Master Protocol Screening Visit.
    • Participants must either not take riluzole or be on a stable dose of riluzole for ≥ 30 days prior to the Master Protocol Screening Visit. Riluzole-naïve participants are permitted in the study.
    • Participants must either not take edaravone or have completed at least one cycle of edaravone prior to the Master Protocol Screening Visit. Edaravone-naïve participants are permitted in the study.
    • Participants must have the ability to swallow pills and liquids at the time of the Master Protocol Screening Visit and, in the SI's opinion, have the ability to swallow for the duration of the study.
    • Geographically accessible to the site.

    Exclusion Criteria:

    • Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to SI's judgment (e.g., cardiovascular instability, systemic infection, untreated thyroid dysfunction, or clinically significant laboratory abnormality or EKG changes). Lab abnormalities include, but are not limited to: Hemoglobin < 10 g/dL, White Blood Cells < 3.0 x 103/mm3, Neutrophils, Absolute ≤ 1000/mm3, Eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per microliter), low platelet counts (< 150 x 109 per liter), alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 times the upper limit of normal (ULN), eGFR < 30 mL/min/1.73m2, thyroid-stimulating hormone (TSH) levels >10 mIU/L or <0.01 mIU/L.
    • Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, in the SI's opinion.
    • Active cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.
    • Use of investigational treatments for ALS (off-label use or active participation in a clinical trial) within 5 half-lives (if known) or 30 days (whichever is longer) prior to the Master Protocol Screening Visit.
    • Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational).
    • If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception for the duration of the trial and for 3 months, or longer as specified in each RSA, after discontinuing study treatment.
    • If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 3 months, or longer as specified in each RSA, after discontinuing study treatment.
    • Anything that would place the participant at increased risk or preclude the participant's full compliance with or completion of the study, in the SI's opinion.
    • If a participant is being re-screened, the disqualifying condition has not been resolved, or the mandatory wash-out duration has not occurred.
    • For those participating in the optional CSF collection, contraindication to undergoing a lumbar puncture (LP) in the SI's opinion. Participants undergoing the LP must not be currently taking anticoagulation medications such as warfarin that would be a contraindication to LP; aspirin and non-steroidal anti-inflammatories are allowed.

  • Site Contact Information

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

    Loma Linda University Health
    David Borg / .(JavaScript must be enabled to view this email address) / Loma Linda, California 92354
    United States

    Cedars-Sinai Medical Center
    Sophia Mostowy / .(JavaScript must be enabled to view this email address) / Los Angeles, California 90048
    United States

    University of Southern California
    Salma Akhter / .(JavaScript must be enabled to view this email address) / 323-442-6221
    Los Angeles, California 90033
    United States

    University of California, Irvine
    Jeanette Overton / .(JavaScript must be enabled to view this email address) / Orange, California 92868
    United States

    Forbes Norris MDA/ALS Research Center, California Pacific Medical Cente
    Henry Chen / .(JavaScript must be enabled to view this email address) / 415-600-5764
    San Francisco, California 94115
    United States

    University of California San Francisco
    Neha Madugala / .(JavaScript must be enabled to view this email address) / 916-236-7172
    San Francisco, California
    United States

    University of Colorado
    Brenna Baines / .(JavaScript must be enabled to view this email address) / Aurora, Colorado 80045
    United States

    Hospital for Special Care
    Natalie Cartwright / .(JavaScript must be enabled to view this email address) / New Britain, Connecticut 06053
    United States

    Georgetown University
    Sarai Bartlett / .(JavaScript must be enabled to view this email address) / 202-630-8445
    Washington, District of Columbia 20007
    United States

    Nova Southeastern University
    Donovan Mott / .(JavaScript must be enabled to view this email address) / 954-203-2829
    Davie, Florida 33314
    United States

    Phil Smith Neuroscience Institute at Holy Cross Hospital
    Ashley Stepler / .(JavaScript must be enabled to view this email address) / Fort Lauderdale, Florida 33308
    United States

    University of Florida
    Jennifer Steshyn / .(JavaScript must be enabled to view this email address) / Gainesville, Florida 32610
    United States

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

    University of Miami
    Jessica Hernandez / .(JavaScript must be enabled to view this email address) / Miami, Florida 33136
    United States

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

    Emory University
    Atlanta, Georgia 30322
    United States

    Augusta University
    Brandy Quarles / .(JavaScript must be enabled to view this email address) / 706-721-0390
    Augusta, Georgia 30912
    United States

    Saint Alphonsus Regional
    Joshua Harrison / .(JavaScript must be enabled to view this email address) / Boise, Idaho
    United States

    Northwestern University
    Emma Schmidt / .(JavaScript must be enabled to view this email address) / Chicago, Illinois 60611
    United States

    University of Chicago
    Sravya Aluri / .(JavaScript must be enabled to view this email address) / Chicago, Illinois 60637
    United States

    Indiana University
    Sandy Guingrich / .(JavaScript must be enabled to view this email address) / 317-963-7382
    Indianapolis, Indiana 46202
    United States

    University of Iowa Hospitals and Clinics
    Emily McDonnell / .(JavaScript must be enabled to view this email address) / Iowa City, Iowa 52242
    United States

    University of Kansas Medical Center
    Katheryn Jennens / .(JavaScript must be enabled to view this email address) / Fairway, Kansas 66205
    United States

    University of Kentucky
    Emma Hanley / .(JavaScript must be enabled to view this email address) / Lexington, Kentucky 40536
    United States

    Ochsner Health System
    Lisa Edmond / .(JavaScript must be enabled to view this email address) / 985-989-7182
    New Orleans, Louisiana 70115
    United States

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

    University of Maryland
    Vikram Nambiar / .(JavaScript must be enabled to view this email address) / Baltimore, Maryland 21201
    United States

    Beth Israel Deaconess Medical Center
    Hilda Guitierrez / .(JavaScript must be enabled to view this email address) / Boston, Massachusetts 02215
    United States

    Massachusetts General Hospital
    Mia Resendes / .(JavaScript must be enabled to view this email address) / 617-643-3902
    Boston, Massachusetts 02114
    United States

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

    University of Michigan
    Jayna Duell / .(JavaScript must be enabled to view this email address) / Ann Arbor, Michigan 48109
    United States

    Henry Ford Health System
    Beverley Duthie / .(JavaScript must be enabled to view this email address) / Detroit, Michigan 48202
    United States

    Spectrum Health
    Sehrish Khan / .(JavaScript must be enabled to view this email address) / Grand Rapids, Michigan 49525
    United States

    Essentia Health
    Brent Gavin / .(JavaScript must be enabled to view this email address) / 218-786-1220
    Duluth, Minnesota
    United States

    University of Minnesota/Twin Cities ALS Research Consortium
    Valerie Ferment / .(JavaScript must be enabled to view this email address) / Minneapolis, Minnesota 55455
    United States

    Mayo Clinic - Rochester
    Megan Means / .(JavaScript must be enabled to view this email address) / 507-293-0545
    Rochester, Minnesota 55902
    United States

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

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

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

    Neurology Associates, P.C./Somnos Clinical Research
    Desirae Eschiti / .(JavaScript must be enabled to view this email address) / Lincoln, Nebraska 68506
    United States

    University of Nebraska Medical Center
    Rachel Harper / .(JavaScript must be enabled to view this email address) / 402-552-6233
    Omaha, Nebraska 68198
    United States

    Dartmouth-Hitchcock Medical Center
    Lebanon, New Hampshire 03756
    United States

    Columbia University
    Oneika Blackman / .(JavaScript must be enabled to view this email address) / New York, New York 10032
    United States

    SUNY Upstate
    Lena Deb / .(JavaScript must be enabled to view this email address) / Syracuse, New York 13202
    United States

    Duke University
    Michelle Ward / .(JavaScript must be enabled to view this email address) / Durham, North Carolina 27702
    United States

    Wake Forest Health Science
    Mozhdeh Marandi / .(JavaScript must be enabled to view this email address) / Winston-Salem, North Carolina 27157
    United States

    University of Cincinnati
    Ashley Smith / .(JavaScript must be enabled to view this email address) / Cincinnati, Ohio
    United States

    The Ohio State University
    MacKenzie Kaschalk / .(JavaScript must be enabled to view this email address) / Columbus, Ohio 43221
    United States

    Providence Brain and Spine Institute ALS Center
    Ashley Adamo / .(JavaScript must be enabled to view this email address) / Portland, Oregon 97213
    United States

    Lehigh Valley Health Network
    Andrew Orzel / .(JavaScript must be enabled to view this email address) / 610-402-9543
    Allentown, Pennsylvania 18103
    United States

    Penn State Hershey
    Heidi Runk / .(JavaScript must be enabled to view this email address) / Hershey, Pennsylvania 17033
    United States

    Temple University, Lewis Katz School of Medicine
    Kathleen Hatala / .(JavaScript must be enabled to view this email address) / Philadelphia, Pennsylvania 19140
    United States

    Thomas Jefferson University, Jefferson Weinberg ALS Center
    Mattison Worthy / .(JavaScript must be enabled to view this email address) / Philadelphia, Pennsylvania 19107
    United States

    University of Penn
    Adreeja Guharay / .(JavaScript must be enabled to view this email address) / Philadelphia, Pennsylvania 19107
    United States

    University of Pittsburgh Medical Center
    Melissa Ilnicki / .(JavaScript must be enabled to view this email address) / 412-864-0865
    Pittsburgh, Pennsylvania 15232
    United States

    Vanderbilt University Medical Center
    Diana Davis / .(JavaScript must be enabled to view this email address) / Nashville, Tennessee 37232
    United States

    Texas Neurology
    Mohamad Asaad Nasri / .(JavaScript must be enabled to view this email address) / Dallas, Texas 75214
    United States

    Houston Methodist
    Rachel Applegate / .(JavaScript must be enabled to view this email address) / Patricia Mendoza / .(JavaScript must be enabled to view this email address) / Houston, Texas 77030
    United States

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

    University of Utah
    Crystal Neate / .(JavaScript must be enabled to view this email address) / Salt Lake City, Utah 84132
    United States

    University of Virginia
    Mary Wagoner / .(JavaScript must be enabled to view this email address) / Charlottesville, Virginia 22908
    United States

    Virginia Commonwealth University
    LaVon Smith / .(JavaScript must be enabled to view this email address) / Henrico, Virginia
    United States

    Swedish Medical Center
    Janae Rahiman / .(JavaScript must be enabled to view this email address) / Seattle, Washington
    United States

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

    Medical College of Wisconsin
    Marie Mejaki / .(JavaScript must be enabled to view this email address) / Milwaukee, Wisconsin 53226
    United States