One-Shot gene therapy trial offers hope for kids with devastating brain disease
NCT ID NCT06983158
Summary
This study is testing a single-dose gene therapy called CAP-002 in children with STXBP1 encephalopathy, a rare genetic disorder that causes severe developmental delays and seizures. The main goals are to see if the treatment is safe and if it can improve children's development, motor skills, and reduce seizures. About 12 children, aged 18 months to under 8 years, will receive one infusion and be closely monitored for two years.
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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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