← Back to searchRecruitingRecruiting
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: 2026-04-16
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
ContactHEALEY Center for ALS at Massachusetts General Hospital · contact · healeyalsplatform@mgh.harvard.edu · 833-425-8257 (HALT ALS)
InvestigatorMerit Cudkowicz, MD · principal_investigator, Massachusetts General Hospital
All locations (75)
Barrow Neurological InstituteRecruiting
Phoenix, Arizona, United States
Mayo Clinic ScottsdaleRecruiting
Scottsdale, Arizona, United States
University of Arkansas for Medical SciencesActive Not Recruiting
Little Rock, Arkansas, United States
Loma Linda University HealthActive Not Recruiting
Loma Linda, California, United States
Kaiser Permanente Los Angeles Medical CenterActive Not Recruiting
Los Angeles, California, United States
University of Southern CaliforniaRecruiting
Los Angeles, California, United States
Cedars-Sinai Medical CenterActive Not Recruiting
Los Angeles, California, United States
University of California, IrvineActive Not Recruiting
Orange, California, United States
Forbes Norris MDA/ALS Research Center, California Pacific Medical CenterRecruiting
San Francisco, California, United States
University of California, San FranciscoRecruiting
San Francisco, California, United States
University of ColoradoRecruiting
Aurora, Colorado, United States
Hospital for Special CareRecruiting
New Britain, Connecticut, United States
Yale UniversityRecruiting
New Haven, Connecticut, United States
Georgetown UniversityRecruiting
Washington D.C., District of Columbia, United States
George Washington UniversityActive Not Recruiting
Washington D.C., District of Columbia, United States
Nova Southeastern UniversityRecruiting
Davie, Florida, United States
University of FloridaActive Not Recruiting
Gainesville, Florida, United States
Mayo Clinic FloridaActive Not Recruiting
Jacksonville, Florida, United States
University of MiamiActive Not Recruiting
Miami, Florida, United States
University of South FloridaActive Not Recruiting
Tampa, Florida, United States
Augusta UniversityActive Not Recruiting
Augusta, Georgia, United States
Saint Alphonsus Regional Medical CenterActive Not Recruiting
Boise, Idaho, United States
Northwestern UniversityRecruiting
Chicago, Illinois, United States
University of ChicagoActive Not Recruiting
Chicago, Illinois, United States
Indiana University HealthActive Not Recruiting
Indianapolis, Indiana, United States
University of Iowa Hospitals and ClinicsRecruiting
Iowa City, Iowa, United States
University of Kansas Medical CenterRecruiting
Fairway, Kansas, United States
University of KentuckyActive Not Recruiting
Lexington, Kentucky, United States
Ochsner Health SystemActive Not Recruiting
New Orleans, Louisiana, United States
Johns Hopkins UniversityActive Not Recruiting
Baltimore, Maryland, United States
Massachusetts General HospitalRecruiting
Boston, Massachusetts, United States
Beth Israel Deaconess Medical CenterRecruiting
Boston, Massachusetts, United States
University of Massachusetts Medical SchoolRecruiting
North Worcester, Massachusetts, United States
University of MichiganRecruiting
Ann Arbor, Michigan, United States
Henry Ford Health SystemRecruiting
Detroit, Michigan, United States
Spectrum Health/Corewell HealthRecruiting
Grand Rapids, Michigan, United States
Essentia HealthRecruiting
Duluth, Minnesota, United States
University of Minnesota Medical SchoolRecruiting
Minneapolis, Minnesota, United States
Mayo Clinic - RochesterRecruiting
Rochester, Minnesota, United States
University of Missouri Health CareRecruiting
Columbia, Missouri, United States
Saint Louis UniversityRecruiting
St Louis, Missouri, United States
Washington University School of MedicineRecruiting
St Louis, Missouri, United States
Neurology Associates, P.C./Somnos Clinical ResearchActive Not Recruiting
Lincoln, Nebraska, United States
University of Nebraska Medical CenterRecruiting
Omaha, Nebraska, United States
Dartmouth-Hitchcock Medical CenterRecruiting
Lebanon, New Hampshire, United States
Hackensack University Medical CenterActive Not Recruiting
Hackensack, New Jersey, United States
Dent Neurologic InstituteRecruiting
Amherst, New York, United States
Mount SinaiRecruiting
New York, New York, United States
Columbia UniversityActive Not Recruiting
New York, New York, United States
Stony Brook University HospitalActive Not Recruiting
Stony Brook, New York, United States
SUNY UpstateRecruiting
Syracuse, New York, United States
University of North CarolinaActive Not Recruiting
Chapel Hill, North Carolina, United States
Atrium HealthRecruiting
Charlotte, North Carolina, United States
Duke UniversityActive Not Recruiting
Durham, North Carolina, United States
Wake Forest Health ScienceRecruiting
Winston-Salem, North Carolina, United States
University of CincinnatiRecruiting
Cincinnati, Ohio, United States
Cleveland ClinicRecruiting
Cleveland, Ohio, United States
The Ohio State UniversityRecruiting
Columbus, Ohio, United States
Providence Brain and Spine Institute ALS CenterActive Not Recruiting
Portland, Oregon, United States
Lehigh Valley Health NetworkRecruiting
Allentown, Pennsylvania, United States
Penn State HersheyRecruiting
Hershey, Pennsylvania, United States
Jefferson Weinberg ALS Center, Thomas Jefferson UniversityRecruiting
Philadelphia, Pennsylvania, United States
University of PennsylvaniaRecruiting
Philadelphia, Pennsylvania, United States
Lewis Katz School of Medicine at Temple UniversityRecruiting
Philadelphia, Pennsylvania, United States
University of Pittsburg Medical CenterRecruiting
Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical CenterRecruiting
Nashville, Tennessee, United States
Texas NeurologyRecruiting
Dallas, Texas, United States
Houston MethodistActive Not Recruiting
Houston, Texas, United States
UTHSCSAActive Not Recruiting
San Antonio, Texas, United States
University of UtahRecruiting
Salt Lake City, Utah, United States
University of VirginiaRecruiting
Charlottesville, Virginia, United States
Virginia Commonwealth UniversityActive Not Recruiting
Henrico, Virginia, United States
Swedish Medical CenterRecruiting
Seattle, Washington, United States
University of WashingtonRecruiting
Seattle, Washington, United States
Medical College of WisconsinActive Not Recruiting
Milwaukee, Wisconsin, United States