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

NCTID	NCT06983158 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Active
Indication	Developmental and Epileptic Encephalopathy
Disease Ontology Term	DOID:0080436
Compound Name	CAP-002
Sponsor	Capsida Biotherapeutics, Inc.
Funder Type	Industry
Recruitment Status	Suspended
Enrollment Count	12 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	STXBP1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV
Editor Type	none
Dose 1	Undisclosed dose escalation, 2 levels
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation
Recent Updates	Trial stoppage rules were triggered upon the death of the first dosed patient in September 2025