HEALEY ALS Platform Trial

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.

• 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

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

  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
  Jillian Doherty / .(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

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

  Holy Cross Hospital
  Lindita Burba / .(JavaScript must be enabled to view this email address) / Phil Smith Neuroscience Institute
  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) / 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

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

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

  Massachusetts General Hospital
  Ella Collins / .(JavaScript must be enabled to view this email address) / 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
  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

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

  Mayo Clinic - Rochester
  Michelle Hewlett / .(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.
  Ashley Calhoun / .(JavaScript must be enabled to view this email address) / Somnos Clinical Research
  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
  Jessica Singleton / .(JavaScript must be enabled to view this email address) / New York, New York 10032
  United States

  SUNY Upstate
  Jacob Pusey / .(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

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

  University of Penn
  Sara-Claude Michon / .(JavaScript must be enabled to view this email address) / Philadelphia, Pennsylvania 19107
  United States

  Temple University
  Kathleen Hatala / .(JavaScript must be enabled to view this email address) / Lewis Katz School of Medicine
  Philadelphia, Pennsylvania 19140
  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
  Robert Henegar / .(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
  Sharon Halton / .(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
  Diana Diana / .(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