Gene therapy trial hopes to quiet seizures in kids with rare STXBP1 brain disease
NCT ID NCT06983158
Summary
This early-stage trial tests a single dose of CAP-002 gene therapy in 12 children (ages 1.5 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 watch for improvements in seizures and development over 2 years. Because this is a gene therapy that aims to correct the underlying genetic problem, it may reduce disease burden, but children may still need ongoing care.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for DEVELOPMENTAL AND EPILEPTIC ENCEPHALOPATHY are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Buerger Center for Advanced Pediatric Care, Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19146, United States
-
Colorado Child Health Research Institute
Aurora, Colorado, 80045, United States
-
Texas Children's Hospital
Houston, Texas, 77030, United States
-
Weill Cornell Medicine
New York, New York, 10021, United States
Conditions
Explore the condition pages connected to this study.