Experimental gene therapy hopes to quiet seizures in kids with rare STXBP1 brain disease
NCT ID NCT06983158
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 31 times
Summary
This early-stage trial tests a single dose of CAP-002 gene therapy in 12 children aged 18 months to 8 years with STXBP1 encephalopathy, a genetic condition that causes severe seizures and developmental delays. The main goal is to check safety, but researchers will also track whether the therapy reduces seizures and improves development over 2 years. The study is currently suspended.
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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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Contacts and locations
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Locations
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Buerger Center for Advanced Pediatric Care, Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19146, United States
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Colorado Child Health Research Institute
Aurora, Colorado, 80045, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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Weill Cornell Medicine
New York, New York, 10021, United States
Conditions
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