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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.
Details
| Lead sponsor | Capsida Biotherapeutics, Inc. |
|---|---|
| Phase | PHASE1, PHASE2 |
| Status | SUSPENDED |
| Enrolment | 12 |
| Start date | Thu Jul 03 2025 00:00:00 GMT+0000 (Coordinated Universal Time) |
| Completion | Wed Dec 20 2028 00:00:00 GMT+0000 (Coordinated Universal Time) |
Conditions
- Developmental and Epileptic Encephalopathy
Interventions
- gene therapy
Countries
United States