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

NCT06983158 · Status: SUSPENDED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2025-10-01

No results posted yet for this study

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 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.

Conditions

  • Developmental and Epileptic Encephalopathy

Interventions

DRUG

gene therapy

Intra-venous gene therapy

Sponsors & Collaborators

  • Capsida Biotherapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Melanie Brandabur, MD · Capsida Biotherapeutics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Months
Max Age
7 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-07-03
Primary Completion
2028-12-20
Completion
2028-12-20
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06983158 on ClinicalTrials.gov