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Gene Therapy Trial Report

Summary

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
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

Therapy Information


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
Delivery System Viral transduction
Vector Type AAV
Editor Type none
Dose 1 Undisclosed dose escalation, 2 levels

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2025-05-05
Completion Date 2028-12-20
Last Update 2025-10-01

Participation Criteria


Eligible Age 18 Months - 7 Years
Standard Ages Child
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: Male or female, ≥18 months to \<8 years of age; Has diagnosis of developmental encephalopathy due to an STXBP1 mutation with confirmation of a pathogenic or likely pathogenic STXBP1 gene mutation. Has a legally authorized representative (LAR) willing and able to complete the informed consent process, willing to comply with trial procedures, and able to travel for repeat visits. Is stable on any medication regimens (if being administered to control the signs and symptoms of underlying disease) for at least 6 weeks prior to trial entry and expected to be stable for at least 12 weeks post-CAP-002 administration. Exclusion Criteria: History of prior gene therapy; Treatment with antisense oligonucleotide therapy within 6 months; Presence of a confirmed mutation in a gene other than STXBP1 that is known to contribute to a neurodevelopmental disability or epilepsy; Has presence of a significant non-STXBP1-related central nervous impairment/behavioral disturbance that would confound the scientific rigor or interpretation of results of the trial; History of prematurity (defined as gestational age \<35 weeks), history of low birth weight (\<2.5 kg) and/or intra-uterine growth restriction, significant interventricular hemorrhage, structural brain deficit, or congenital heart disease; Known contraindication to immunosuppression or other protocol-defined medications, including but not limited to corticosteroids or PPIs; Clinically significant abnormalities in safety lab tests, vital signs; Other illnesses or medications that may affect the interpretation of the study results; Positive anti-capsid antibody test result.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 4
Locations United States

Regulatory Information


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

Resources/Links