Trials / Not Yet Recruiting
Not Yet RecruitingNCT07293546
Phase 1/2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients With FOXG1 Syndrome (FS)
An Open-Label First-in-Human Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of FRF-001, an AAV-9 Gene Therapy Administered by Intracerebroventricular Injection in Participants With FOXG1 Syndrome
- Status
- Not Yet Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 12 (estimated)
- Sponsor
- FOXG1 Research Foundation · Academic / Other
- Sex
- All
- Age
- 2 Years – 20 Years
- Healthy volunteers
- Not accepted
Summary
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | FRF-001 | AAV-9 gene therapy delivered by intracerebroventricular injection |
Timeline
- Start date
- 2026-04-01
- Primary completion
- 2029-03-01
- Completion
- 2029-07-01
- First posted
- 2025-12-19
- Last updated
- 2026-01-22
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT07293546. Inclusion in this directory is not an endorsement.