Gene Therapy for Developmental Encephalopathy

Recruiting at 4 trial locations

Overseen By Lori Brozena

Age: < 18

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Capsida Biotherapeutics, Inc.

Must not be taking: Antisense oligonucleotides, corticosteroids

Disqualifiers: Gene therapy history, prematurity, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 3

Duration: 2 years

12 Participants Needed

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

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

I have a developmental brain disorder caused by an STXBP1 mutation.

My current medications have been stable for 6 weeks and will remain so for 12 weeks after starting the trial.

Exclusion Criteria

I have a genetic mutation linked to epilepsy or neurodevelopmental issues, not in the STXBP1 gene.

I haven't had antisense oligonucleotide therapy in the last 6 months.

I have had gene therapy before.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Participants receive a single intravenous infusion of CAP-002 in a dose-escalation format

Varies

Multiple visits for dose escalation

Treatment

Participants receive a single intravenous infusion of CAP-002 and are monitored for safety and efficacy

2 years

Regular clinic visits for checkups and tests

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 years

Invited to participate in a 3-year safety follow-up study

Treatment Details

Interventions

• CAP-002

Trial Overview The study tests the safety and effectiveness of CAP-002 gene therapy in treating children with STXBP1 Encephalopathy. It involves one infusion of the therapy followed by regular clinic visits over two years for monitoring, including seizure tracking by caregivers.

Participant Groups

Group I: Dose Level 2 Experimental Treatment 1 Intervention

Participants will receive a single dose of STXBP1, administered IV

Group II: Dose Level 1 Experimental Treatment 1 Intervention

Participants will receive a single dose of STXBP1, administered IV

Who Is Running the Clinical Trial?

Capsida Biotherapeutics, Inc.

Lead Sponsor

Trials

1