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

NCT06983158 PHASE1, PHASE2 SUSPENDED

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 sponsorCapsida Biotherapeutics, Inc.
PhasePHASE1, PHASE2
StatusSUSPENDED
Enrolment12
Start dateThu Jul 03 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
CompletionWed Dec 20 2028 00:00:00 GMT+0000 (Coordinated Universal Time)

Conditions

Interventions

Countries

United States