One-Shot gene therapy trial aims to help kids with rare brain disease
NCT ID NCT06983158
Summary
This early-stage trial is testing a single dose of an experimental gene therapy called CAP-002 in children aged 18 months to 8 years with a rare genetic brain condition called STXBP1 encephalopathy. The main goal is to check if the treatment is safe and tolerable, while also looking for early signs it might help with development, seizures, and daily skills. Participants receive one infusion and are followed closely with checkups and caregiver diaries 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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