First gene therapy for rare brain disorder begins testing in kids
NCT ID NCT07270549
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a gene therapy called Urbagen in 12 children aged 2-12 with CTNNB1 neurodevelopmental syndrome, a rare genetic condition causing motor and cognitive delays. The therapy is given as a single infusion into the brain fluid, along with immunosuppressant drugs to prevent rejection. The study primarily checks safety and whether it can improve movement, thinking, and quality of life over three years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Urbagen gene therapy (AAV9-based) plus sirolimus immunosuppressant
- What this could lead to
- If successful, this could point toward a treatment that improves motor and cognitive function in children with CTNNB1 syndrome.
- What could go wrong
- This is a very early, first-in-human trial with only 12 participants. Gene therapy carries risks like immune reactions, and the long-term effects are unknown. Lifelong immunosuppression may be needed.
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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: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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University Medical Centre Ljubljana
RECRUITINGLjubljana, 1000, Slovenia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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