Phase 1/2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients With FOXG1 Syndrome (FS)
Part of paid clinical trials in Houston, Texas.
- Sponsor
- FOXG1 Research Foundation
- Study ID
- NCT07293546
- Phase
- PHASE1/PHASE2
- Status
- Enrolling By Invitation
Conditions
- FOXG1 Syndrome
Eligibility Criteria
- Sex
- ALL
- Age
- 2 Years - 20 Years
- Healthy Volunteers
- Not accepted
Interventions
- FRF-001 — GENETICAAV-9 gene therapy delivered by intracerebroventricular injection
Study Details
The goal of this clinical trial is to learn if FRF-001 is a safe, tolerable, and efficacious treatment for children and adults with FOXG1 syndrome.
Key Dates
- First listed
- Dec 19, 2025
- Start date
- Jul 20, 2026
- Status verified
- Jul 2026
- Primary completion
- Jul 31, 2029
- Completion
- Dec 31, 2029
Study Design
- Enrollment
- 12 participants (estimated)
- Allocation
- NA
- Intervention model
- SINGLE_GROUP
- Primary purpose
- TREATMENT
Arms
- Experimental: Treatment
Primary Outcome Measure
To evaluate the safety and tolerability of single-dose FRF-001 in participants with FOXG1 syndrome [ Time Frame: Through Week 104 ]
Locations (1)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| Texas Children's Hospital (Baylor College of Medicine) | Houston | Texas | 77030 | - |
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