The goal of this clinical trial is to learn about the safety of CAP-002 gene therapy in children with Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy. It will also provide information about whether CAP-002 demonstrates efficacy in treating children with STXBP1 with and without seizures. Participants will have a single infusion of CAP-002, visit the clinic regularly for 2 years for checkups and tests and have seizures recorded in a diary by their caregiver.
The goal of this clinical trial is to learn about the safety of CAP-002 gene therapy in children with Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy. It will also provide information about whether CAP-002 demonstrates efficacy in treating children with STXBP1 with and without seizures. Participants will have a single infusion of CAP-002, visit the clinic regularly for 2 years for checkups and tests and have seizures recorded in a diary by their caregiver.
A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy
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Colorado Child Health Research Institute, Aurora, Colorado, United States, 80045
Weill Cornell Medicine, New York, New York, United States, 10021
Buerger Center for Advanced Pediatric Care, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19146
Texas Children's Hospital, Houston, Texas, United States, 77030
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
For general information about clinical research, read Learn About Studies.
18 Months to 7 Years
ALL
No
Capsida Biotherapeutics, Inc.,
Melanie Brandabur, MD, STUDY_DIRECTOR, Capsida Biotherapeutics
2028-12-20