Natural History Study in Pediatric Patients with STXBP1 Encephalopathy with Epilepsy

NCT ID: NCT05462054

Last Updated: 2024-10-18

Basic Information

• Recruitment Status: WITHDRAWN
• Study Classification: OBSERVATIONAL
• Study Start Date: 2024-10-31
• Study Completion Date: 2026-01-31

Brief Summary

This is a prospective, non-interventional, longitudinal study designed to characterize the natural history of STXBP1 related encephalopathy with epilepsy, in participants ≤ <5 years of age.

Conditions

STXBP1 Encephalopathy with Epilepsy

Study Design

• Observational Model Type: CASE_ONLY
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

Inclusion Criteria

• Male or female, > 1 day to ≤ 5 years of age at the time of informed consent.
• Diagnosed with seizure disorder
• Presence of a STXBP1 gene mutation. Historical documentation is sufficient to support eligibility for the study. Confirmatory testing will be obtained at baseline and performed by a CLIA certified laboratory
• Written informed consent provided by a parent or legal guardian

Exclusion Criteria

• History of prior gene therapy treatment
• Current enrollment in an interventional study or has received an investigational drug within 30 days or fewer than 5 half-lives prior to screening visit, whichever is longer
• Treatment with any antisense oligonucleotide therapy within 6 months prior to screening and anticipate remaining on treatment throughout the study
• The presence of a confirmed mutation in a gene other than STXPB1 that is known to contribute to a neurodevelopmental disability
• Presence of a significant non-STXBP1 related central nervous impairment/behavioral disturbance that would confound the scientific rigor or interpretation of results of the study
• History of prematurity (defined as gestational age <35 weeks), interventricular hemorrhage, structural brain deficit or congenital heart disease
• Requires mechanical ventilation or non-invasive respiratory support such as continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) at the time of informed consent

• Minimum Eligible Age: 1 Day
• Maximum Eligible Age: 5 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Capsida Biotherapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Monitor, MD

Role: STUDY_DIRECTOR

Capsida Biotherapeutics, Inc.

Other Identifiers

CAP-002-501

Identifier Type: -

Identifier Source: org_study_id