Gene therapy RJK002 injected into spine for ALS – first human test begins
NCT ID NCT06493279
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a single injection of RJK002, a gene therapy, into the spinal fluid of 9 people with ALS. The main goal is to see if it is safe and to find the best dose for future studies. Researchers will also track changes in daily function, but the trial is too small to prove whether it works.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- RJK002 (gene therapy given as a single injection into the spinal fluid)
- What this could lead to
- If safe, this could point toward a treatment that slows or stabilizes ALS progression.
- What could go wrong
- This is a very early, small trial (9 people) focused only on safety, not effectiveness. Gene therapies can have serious side effects, and the dose may not be strong enough to help.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Peking University Third Hospital
Beijing, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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