New drug RAG-21 for ALS with FUS gene changes
Part of Brain & nervous system, Hormones & metabolism clinical trials.
This trial tests a new drug called RAG-21 in people with ALS who have a specific genetic change (FUS gene mutation). The goal is to see if it is safe and effective in slowing the disease.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 18 and 75 years old.
- You must have ALS caused by a specific genetic change in the FUS gene (a type of familial ALS).
- Your breathing capacity must be at least 50% of what is expected for your age and size.
- Your first symptoms must have started in your arms or legs (not in speech or swallowing).
- You must not have certain FUS mutation locations, prior treatment with Ulefnersen, or other major health conditions (like active hepatitis, severe heart/liver/kidney problems, or permanent ventilator use).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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