Testing new medicines for muscle weakness disease

Plain-English translation of NCT04297683 on ClinicalTrials.gov ↗ · Source last updated June 2026 · Translation generated June 2026 · How we translate trials

Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.

What is this trial?

The HEALEY ALS Platform Trial is testing several different investigational medications to see if they can slow or improve ALS (amyotrophic lateral sclerosis), a disease that causes progressive muscle weakness. You would be randomly assigned to receive either the active medication or a placebo (inactive treatment), and researchers will monitor your health and muscle function over time to see if the treatment helps.

ALS is a serious disease with limited treatment options, and doctors need to find new medicines that work better. This trial is designed to test multiple promising medications quickly and efficiently so that patients have access to potentially helpful treatments sooner.

Do you qualify?

You likely qualify if…

you have been diagnosed with ALS (clinically possible, probable, or definite) within the past 24 months
you are 18 years of age or older
you have a vital capacity (lung function measure) of at least 50% of predicted normal
you can swallow pills and liquids and are expected to keep this ability throughout the study
you are able to understand the study and follow its procedures

You likely don't qualify if…

you have serious unstable medical conditions (other than ALS) such as heart problems or active infections, or abnormal liver or kidney function
you have untreated psychiatric disease, dementia, or substance abuse that would prevent you from giving informed consent
you have active cancer or a history of cancer (except successfully treated skin cancer) without at least 3 years of remission
you have received other experimental ALS treatments within the last 30 days or are currently in another ALS drug trial
you are pregnant, breastfeeding, or unwilling to use effective contraception during the study

What participation looks like

You will have screening visits to confirm you qualify, then be randomly assigned to receive either the active medication or placebo. Most participants will have a three-in-four chance of receiving the actual medication. You'll visit the research site regularly for check-ups, blood tests, and measurements of muscle strength and breathing function. The exact duration and number of visits depend on which medication regimen you are assigned to, but the trial is designed to monitor you closely to ensure your safety and to measure whether the treatment is helping.

AI-generated summary from trial data · Jun 13, 2026 · Not medical advice

Trial locations(81 sites)

United States

Barrow Neurological Institute, Phoenix, Arizona

Mayo Clinic Scottsdale, Scottsdale, Arizona

University of Arkansas for Medical Sciences, Little Rock, Arkansas

UC San Diego, La Jolla, California

Loma Linda University Health, Loma Linda, California