HEALEY ALS Platform Trial - Master Protocol

Running, not enrolling · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Amyotrophic Lateral Sclerosis

In brief

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.

Key facts

Study ID

NCT04297683

Run by

Merit E. Cudkowicz, MD

People needed

1500

Starts

2020-06-14

Expected to finish

2028-08-01

Last updated by the study team

2026-07-21

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 ≤ 24 months at the time of the Master Protocol Screening Visit.
• 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.
• Participants must either not take riluzole or be on a stable dose of riluzole for ≥ 30 days prior to the Master Protocol Screening Visit.
• 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.
• 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.

You may not qualify if…

• 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.
• 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 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 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.

Where it is running

• 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
• Barrow Neurological Institute — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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