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 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.

• 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

  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

  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

  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
  Danny Hevert / .(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
  Stephanie Hansen / .(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

  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)
  Charlotte Rhodes / .(JavaScript must be enabled to view this email address) / 210-450-8454
  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

  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