HEALEY ALS Platform Master Protocol for testing multiple ALS treatment regimens

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

The HEALEY ALS Platform is a perpetual, multi-center Master Protocol that allows multiple investigational products to be tested simultaneously or sequentially under regimen-specific appendices. Each regimen is a randomized, double-blind, placebo-controlled comparison with participants randomized 3:1 to active drug versus matching placebo. Eligible adults have possible to definite ALS by revised El Escorial criteria, symptom onset within 24 months, and slow vital capacity (or FVC if required) of ≥50%, with stable use of riluzole or edaravone permitted. New regimens and sites can be added over time so additional investigational products are continuously evaluated.

Why it matters

Who can join: the full rules

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.

Where it's running

• Barrow Neurological Institute
  Phoenix, Arizona, United States Recruiting
• Mayo Clinic Scottsdale
  Scottsdale, Arizona, United States Recruiting
• University of Arkansas for Medical Sciences
  Little Rock, Arkansas, United States Recruiting
• University of Southern California
  Los Angeles, California, United States Recruiting
• Cedars-Sinai Medical Center
  Los Angeles, California, United States Recruiting
• Forbes Norris MDA/ALS Research Center, California Pacific Medical Center
  San Francisco, California, United States Recruiting

+28 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Principal investigator: Merit Cudkowicz, MD — Massachusetts General Hospital
• Study coordinator: HEALEY Center for ALS at Massachusetts General Hospital
• Email: healeyalsplatform@mgh.harvard.edu
• Phone: 833-425-8257 (HALT ALS)

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.