Plain-English translation of NCT04220190 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · 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.
This trial is testing RAPA-501, a new treatment made from your own immune cells that have been reprogrammed in a laboratory to reduce inflammation in the nervous system. The medication is designed to help calm the immune system's attack on nerve cells in ALS. Researchers want to see if this treatment is safe and whether it can help slow the disease's progression.
ALS damages nerve cells over time, and the immune system appears to play a role in this damage. This trial exists because doctors need new treatment options beyond the current medications available, and this approach of using the body's own immune cells to fight inflammation is a promising new direction.
You likely qualify if…
You likely don't qualify if…
First, doctors will collect some of your blood cells through a procedure similar to blood donation. Your cells will then be sent to a laboratory to be reprogrammed. Over the course of about 5 months, you will receive up to four infusions of the reprogrammed cells, spaced 6 weeks apart. After each infusion, you'll have follow-up visits to check on your health and how you're doing. After the first 30 weeks, you'll continue in the study remotely, checking in using surveys for two years so researchers can track how you're doing long-term.
AI-generated summary from trial data · Jun 1, 2026 · Not medical advice
United States
Phase
Large-scale testing
Sponsor
Rapa Therapeutics LLC
Collaborators
Massachusetts General Hospital
Enrollment target
~41 participants
Started
January 2025
Primary completion
July 2026
Age range
18 Years and older
Last updated on clinicaltrials.gov in June 2025.
Reach out to the team running this trial. Response times vary — some teams are faster than others.
Central contact
Daniel Fowler, M.D. Chief Medical Officer, RAPA Therapeutics, LLC
Rapa Therapeutics LLC
Tell us you're interested and we'll help connect you with the research team. We'll walk you through what to expect first — no email needed to get started.