Plain-English translation of NCT07674667 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.
This trial is testing a new medication called , which is designed to restore the function of a protein called UNC13A that stops working properly in most people with ALS. The medication is given as a single injection into the fluid surrounding your spine, and it may stay in your nervous system long enough that you'd only need it once every 24 weeks. Researchers want to see if this treatment is safe, well-tolerated, and whether it can help slow ALS progression.
Most people with ALS have a breakdown in how their nerve cells process genetic information, which damages the UNC13A protein. This medication is being tested because it may be able to restore how that protein works, potentially slowing nerve damage and helping people maintain function longer.
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You would receive either the new medication or a placebo as a single injection into the spinal fluid. The trial will include multiple visits to the study site for safety monitoring, breathing tests, brain scans, and spinal taps to measure how the medication works. You'll also wear sensors at home and complete speech assessments to help researchers track how the treatment affects your physical function and quality of life over time.
AI-generated summary from trial data · Aug 21, 2026 · Not medical advice
Germany
Netherlands
United Kingdom