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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| FRF-001 | Genetic | AAV-9 gene therapy delivered by intracerebroventricular injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the safety and tolerability of single-dose FRF-001 in participants with FOXG1 syndrome | Incidence, severity, and causality of treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), and adverse events of special interest (AESIs) | Through Week 104 |
| To evaluate the efficacy of single-dose FRF-001 in participants with FOXG1 syndrome | Attainment of motor milestones, as assessed by the Peabody Developmental Motor Scales - Third Edition (PDMS-3) | Week 52 and Week 104 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Gai Ayalon | Contact | 650-665-0310 | clinical@foxg1research.org |
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