Experimental gene therapy aims to halt rare childhood brain disease
NCT ID NCT07582484
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests a gene therapy for children with CLN6 Batten disease, a rare and fatal brain disorder. Twelve children will receive a single injection of the therapy into the fluid around the spinal cord. Researchers will check if it is safe and if it can slow the disease's progression over two years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- scAAV9.CB.CLN6 gene therapy
- What this could lead to
- If it works, this could slow or stop the progression of CLN6 Batten disease, giving children more time and better quality of life.
- What could go wrong
- This is an early-phase trial with only 12 children. Gene therapy is complex and may not work for everyone; there are also risks of side effects from the injection or the virus.
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
-
University of California, San Diego - Rady Children's
La Jolla, California, 92093, United States
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