Home › Trials › NCT04297683

HEALEY ALS Platform Master Protocol for testing multiple ALS treatment regimens

HEALEY ALS Platform Trial

PHASE2; PHASE3 · Massachusetts General Hospital · NCT04297683

This platform tests several experimental drugs against placebo to see if they slow disease progression in people with ALS who are within 24 months of symptom onset and have adequate lung function.

Quick facts

PhasePHASE2; PHASE3
Study typeInterventional
Enrollment1500 (estimated)
Ages18 Years and up
SexAll
SponsorMassachusetts General Hospital (other)
Locations78 sites (Phoenix, Arizona and 77 other locations)
Trial IDNCT04297683 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Adults aged 18 or older with possible, probable, or definite ALS, symptom onset within 24 months, and slow vital capacity ≥50% who can swallow study medication and attend site visits are ideal candidates.

Not a fit: People with symptom onset more than 24 months earlier, vital capacity below 50%, inability to swallow study medication, or who cannot attend required visits are unlikely to qualify or benefit from participation.

Why it matters

Potential benefit: If successful, this platform could identify one or more therapies that slow ALS progression and speed the availability of new treatment options for patients.

How similar studies have performed: Adaptive and platform designs have accelerated testing in other fields (for example oncology and COVID-19), but using a perpetual platform to test multiple ALS treatments is relatively novel and still investigational.

Eligibility criteria

Show full inclusion / exclusion 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.

Where this trial is running

Phoenix, Arizona and 77 other locations

+28 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Amyotrophic Lateral Sclerosis, ALS, Placebo-Controlled, Double-Blind, Master Protocol, Lou Gehrig's Disease

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.