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

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:

480

Estimated Study Start Date:

07/01/2020

Estimated Study Completion Date:

09/30/2021

Posting Last Modified Date:

08/28/2020

Date Study Added to alsconsortium.org:

03/09/2020
  • More Information
  • 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
    Jessie Duncan / .(JavaScript must be enabled to view this email address) / Phoenix, Arizona 85013
    United States

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

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

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

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

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

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

    Phil Smith Neuroscience Institute at Holy Cross Hospital
    Donovan Mott / .(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
    Lisa Thuro / .(JavaScript must be enabled to view this email address) / Jacksonville, Florida 32224
    United States

    University of Miami
    Wendy Levy / .(JavaScript must be enabled to view this email address) / 888-413-9315
    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
    Meraida Polak / .(JavaScript must be enabled to view this email address) / Atlanta, Georgia 30322
    United States

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

    University of Chicago
    Shail Bhatnagar / .(JavaScript must be enabled to view this email address) / Chicago, Illinois 60637
    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
    Andrew Heim / .(JavaScript must be enabled to view this email address) / Fairway, Kansas 66205
    United States

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

    Ochsner Health System
    Kristina Brown / .(JavaScript must be enabled to view this email address) / 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
    Danny Hevert / .(JavaScript must be enabled to view this email address) / 617-643-3902
    Boston, Massachusetts 02114
    United States

    University of Massachusetts Medical School
    Diane McKenna-Yasek / .(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
    Anne Vallis / .(JavaScript must be enabled to view this email address) / Detroit, Michigan 48202
    United States

    Spectrum Health
    Nichole Roderique / .(JavaScript must be enabled to view this email address) / Grand Rapids, Michigan 49525
    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
    Carol Denny / .(JavaScript must be enabled to view this email address) / 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
    Elizabeth Karanja / .(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
    Renee Hogue / .(JavaScript must be enabled to view this email address) / Omaha, Nebraska 68198
    United States

    Dartmouth-Hitchcock Medical Center
    Caren Saunders / .(JavaScript must be enabled to view this email address) / Lebanon, New Hampshire 03756
    United States

    Columbia University
    Brixhilda Dedi / .(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
    Lisa Harrison / .(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

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

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

    Penn State Hershey
    Anne Haulman / .(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
    Laura Oakley / .(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

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

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

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

    Texas Neurology
    Todd Morgan / .(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

    UTHSCSA
    Pamela Kittrell / .(JavaScript must be enabled to view this email address) / San Antonio, Texas 78229
    United States

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