A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy

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.

NCT Number	NCT06983158
Lead Sponsor	Capsida Biotherapeutics, Inc.
Conditions	Developmental and Epileptic Encephalopathy
Enrollment	1 participants
Start Date	2025-07-03
Primary Completion	2026-03-25 (estimated)
Study Completion	2026-05-26 (estimated)
Updated on ClinicalTrials.gov	2026-06-10