Plain-English translation of NCT06849609 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.
This is an early-stage study testing , a gene therapy designed to treat amyotrophic lateral sclerosis (ALS). The medication is injected directly into the spinal fluid to reach nerve cells affected by ALS. Researchers want to find out whether this treatment is safe and whether it can help slow or improve symptoms in people with newly diagnosed ALS.
ALS is a serious disease that currently has no cure, and existing medications have not been able to stop or reverse its progression. This trial exists to explore whether this new gene therapy approach might offer hope where other treatments have failed.
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You will receive one injection of the medication directly into your spinal fluid. Before the injection, you will start taking immunosuppressive medications to help your body accept the treatment. After the injection, you will return for follow-up visits and tests over the next year (52 weeks) to check your safety and whether the treatment is helping. The study team will monitor your lung function, muscle strength, and other markers of ALS progression throughout this period.
AI-generated summary from trial data · Jun 4, 2026 · Not medical advice
China
Phase
Safety & dosing
Sponsor
Hong Chen
Enrollment target
~6 participants
Started
January 2025
Primary completion
May 2026
This trial's estimated completion date has passed — the record may not be fully up to date.
Age range
18 Years and older
Last updated on clinicaltrials.gov in March 2025.
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Central contact
Hong Chen
Hong Chen
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