Plain-English translation of NCT07294144 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.
This trial is testing whether , a medicine that's already approved for one type of ALS, might also help people with other forms of the disease. The study will measure whether the medication can lower certain markers of nerve damage in the blood and spinal fluid, and whether it's safe and tolerable for participants. Researchers also want to see if the treatment affects muscle strength, breathing, and quality of life.
is currently only approved for people with a specific genetic form of ALS caused by SOD1 mutations. This trial exists to find out if this medication might also help the many people with ALS who don't have that particular mutation, potentially offering them a new treatment option.
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You will receive 8 doses of the medication through a lumbar puncture (spinal injection) over 24 weeks, with injections at weeks 0, 2, 4, 8, 12, 16, 20, and 24. Between and after your doses, you'll complete follow-up visits where doctors will test your muscle strength, measure your breathing, and ask you questions about how you're doing. The entire study lasts about 32 weeks from start to finish.
AI-generated summary from trial data · Jun 28, 2026 · Not medical advice
United States