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HEALEY ALS Platform Trial - Master Protocol
NCT04297683 · Massachusetts General Hospital
In plain English
Click the button to translate this study into plain language — what it is, who qualifies, and what participation looks like.
Official title
HEALEY ALS Platform Trial
About this study
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 I - NUZ-001
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
Inclusion Criteria:
1. Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS defined by revised El Escorial criteria.
2. Age 18 years or older.
3. Capable of providing informed consent and complying with study procedures, in the SI's opinion.
4. Time since onset of weakness due to ALS ≤ 24 months at the time of the Master Protocol Screening Visit.
5. Vital Capacity ≥ 50% of predicted capacity at the time of the Master Protocol Screening Visit measured by Slow Vital Capacity (SVC), or, if required due to pandemic-related restrictions, Forced Vital Capacity (FVC) measured in person.
6. Participants must either not take riluzole or be on a stable dose of riluzole for ≥ 30 days prior to the Master Protocol Screening Visit.
7. Participants must either not take edaravone or have completed at least one cycle (typically 14 days) of edaravone prior to the Master Protocol Screening Visit.
8. 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.
9. Geographically accessible to the site.
Exclusion Criteria:
1. 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), or clinically significant laboratory abnormality or EKG changes. Clinically significant abnormal liver or kidney function is exclusionary. The following values \[alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> 3 times the upper limit of normal (ULN) or estimated Glomerular Filtration Rate (eGFR) \< 30 mL/min/1.73m2\] are exclusionary regardless of clinical symptoms.
2. 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.
3. 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.
4. 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.
5. Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational).
6. 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 as specified in each RSA, after discontinuing study treatment.
7. If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 3 months, or as specified in each RSA, after discontinuing study treatment.
8. 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.
9. If a participant is being re-screened, the disqualifying condition has not been resolved, or the mandatory wash-out duration has not occurred.
Study design
Enrollment target: 1500 participants
Allocation: randomized
Masking: quadruple
Age groups: adult, older_adult
Timeline
Starts: 2020-06-14
Estimated completion: 2028-08
Last updated: 2026-07-21
Interventions
Drug: ZilucoplanDrug: VerdiperstatDrug: CNM-Au8Drug: PridopidineDrug: SLS-005 TrehaloseDrug: ABBV-CLS-7262Drug: DNL343Drug: NUZ-001
Primary outcomes
- • Disease Progression (36 Weeks)
Sponsor
Merit E. Cudkowicz, MD · other
With: Massachusetts General Hospital
Contacts & investigators
InvestigatorMerit Cudkowicz, MD · principal_investigator, Massachusetts General Hospital
All locations (81)
Barrow Neurological Institute
Phoenix, Arizona, United States
Mayo Clinic Scottsdale
Scottsdale, Arizona, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
UC San Diego
La Jolla, California, United States
Loma Linda University Health
Loma Linda, California, United States
Kaiser Permanente Los Angeles Medical Center
Los Angeles, California, United States
University of Southern California
Los Angeles, California, United States
Cedars-Sinai Medical Center
Los Angeles, California, United States
University of California, Irvine
Orange, California, United States
Forbes Norris MDA/ALS Research Center, California Pacific Medical Center
San Francisco, California, United States
University of California, San Francisco
San Francisco, California, United States
University of Colorado
Aurora, Colorado, United States
Hospital for Special Care
New Britain, Connecticut, United States
Yale University
New Haven, Connecticut, United States
Georgetown University
Washington D.C., District of Columbia, United States
George Washington University
Washington D.C., District of Columbia, United States
Nova Southeastern University
Davie, Florida, United States
University of Florida
Gainesville, Florida, United States
Mayo Clinic Florida
Jacksonville, Florida, United States
University of Miami
Miami, Florida, United States
University of South Florida
Tampa, Florida, United States
Augusta University
Augusta, Georgia, United States
Saint Alphonsus Regional Medical Center
Boise, Idaho, United States
Northwestern University
Chicago, Illinois, United States
University of Chicago
Chicago, Illinois, United States
Indiana University Health
Indianapolis, Indiana, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
University of Kansas Medical Center
Fairway, Kansas, United States
University of Kentucky
Lexington, Kentucky, United States
Ochsner Health System
New Orleans, Louisiana, United States
Johns Hopkins University
Baltimore, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States
Lahey Hospital and Medical Center
Burlington, Massachusetts, United States
University of Massachusetts Medical School
North Worcester, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Henry Ford Health System
Detroit, Michigan, United States
Spectrum Health/Corewell Health
Grand Rapids, Michigan, United States
Essentia Health
Duluth, Minnesota, United States
University of Minnesota Medical School
Minneapolis, Minnesota, United States
Mayo Clinic - Rochester
Rochester, Minnesota, United States
University of Missouri Health Care
Columbia, Missouri, United States
Saint Louis University
St Louis, Missouri, United States
Washington University School of Medicine
St Louis, Missouri, United States
Neurology Associates, P.C./Somnos Clinical Research
Lincoln, Nebraska, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Dent Neurologic Institute
Amherst, New York, United States
Mount Sinai
New York, New York, United States
Columbia University
New York, New York, United States
University of Rochester
Rochester, New York, United States
Stony Brook University Hospital
Stony Brook, New York, United States
SUNY Upstate
Syracuse, New York, United States
University of North Carolina
Chapel Hill, North Carolina, United States
Atrium Health
Charlotte, North Carolina, United States
Duke University
Durham, North Carolina, United States
Wake Forest Health Science
Winston-Salem, North Carolina, United States
University of Cincinnati
Cincinnati, Ohio, United States
Cleveland Clinic
Cleveland, Ohio, United States
The Ohio State University
Columbus, Ohio, United States
OhioHealth Research Institute
Westerville, Ohio, United States
Providence Brain and Spine Institute ALS Center
Portland, Oregon, United States
Lehigh Valley Health Network
Allentown, Pennsylvania, United States
Penn State Hershey
Hershey, Pennsylvania, United States
Jefferson Weinberg ALS Center, Thomas Jefferson University
Philadelphia, Pennsylvania, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
Lewis Katz School of Medicine at Temple University
Philadelphia, Pennsylvania, United States
University of Pittsburg Medical Center
Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Austin Neuromuscular Clinic
Austin, Texas, United States
Texas Neurology
Dallas, Texas, United States
Baylor College of Medicine
Houston, Texas, United States
Houston Methodist
Houston, Texas, United States
UTHSCSA
San Antonio, Texas, United States
University of Utah
Salt Lake City, Utah, United States
University of Virginia
Charlottesville, Virginia, United States
Virginia Commonwealth University
Henrico, Virginia, United States
Swedish Medical Center
Seattle, Washington, United States
University of Washington
Seattle, Washington, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States