Status:
SUSPENDED
A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy
Lead Sponsor:
Capsida Biotherapeutics, Inc.
Conditions:
Developmental and Epileptic Encephalopathy
Eligibility:
All Genders
18-7 years
Phase:
PHASE1
PHASE2
Brief Summary
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...
Detailed Description
This is a Phase 1/2a, FIH, open-label, multi-center, dose-escalation trial to assess the safety, tolerability, and efficacy of a single intravenous (IV) dose of CAP-002 administered to participants wi...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
July 3 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 20 2028
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT06983158
Start Date
July 3 2025
End Date
December 20 2028
Last Update
October 1 2025
Active Locations (4)
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1
Colorado Child Health Research Institute
Aurora, Colorado, United States, 80045
2
Weill Cornell Medicine
New York, New York, United States, 10021
3
Buerger Center for Advanced Pediatric Care, Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19146
4
Texas Children's Hospital
Houston, Texas, United States, 77030