Plain-English translation of NCT07287397 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 is an early-stage trial testing a new gene therapy called VTx-002 for amyotrophic lateral sclerosis (ALS). The treatment is given as a single injection into the fluid around the spinal cord. Researchers want to see if this medication is safe and how well people tolerate it over a 5-year period.
ALS is a serious condition that progressively weakens muscles, and current treatments have limited effectiveness. This trial is exploring whether this new gene therapy approach might slow disease progression or improve symptoms in people with early-stage ALS.
You likely qualify if…
You likely don't qualify if…
You will receive a single injection of the medication under general anesthesia, followed by an overnight hospital stay for monitoring. In the first year, you will have 12 in-person visits to the study center plus 4 remote visits (by phone or video) to check on your health and perform tests. From years 2 to 5, you will have visits every 6 months—either in-person or remote—for a total of up to 20 study visits over the entire 5-year period. Throughout, researchers will monitor how you are doing and check for any side effects.
AI-generated summary from trial data · Jun 1, 2026 · Not medical advice
United States