Gene Therapy for Developmental Encephalopathy
Trial Summary
What is the purpose of this trial?
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.
Research Team
Melanie Brandabur, MD
Principal Investigator
Capsida Biotherapeutics
Eligibility Criteria
This trial is for pediatric patients with STXBP1 Encephalopathy, a developmental brain disorder. Participants must have a confirmed diagnosis and may or may not experience seizures. Specific inclusion and exclusion criteria details are not provided but typically involve health status and other medical conditions.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Dose Escalation
Participants receive a single intravenous infusion of CAP-002 in a dose-escalation format
Treatment
Participants receive a single intravenous infusion of CAP-002 and are monitored for safety and efficacy
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- CAP-002
Find a Clinic Near You
Who Is Running the Clinical Trial?
Capsida Biotherapeutics, Inc.
Lead Sponsor