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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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University of California, San Diego - Rady Children's
La Jolla, California, 92093, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••