FOXG1 syndrome is a rare neurodevelopmental disorder caused by alterations in FOXG1 and characterized by severe developmental impairment and structural brain abnormalities. Common manifestations include microcephaly, profound intellectual disability, movement abnormalities, seizures, and major communication deficits. The disorder affects both males and females.
The FDA has approved nivolumab with chemotherapy for pediatric and adult Hodgkin lymphoma patients aged 12+, based on clinical trial data showing improved survival. Separately, the agency cleared investigational new drug applications for FG001 for brain cancer surgery visualization and FRF-001 gene therapy for FOXG1 syndrome, allowing both to proceed to clinical trials.
The U.S. Food and Drug Administration has approved an Investigational New Drug application for FRF-001, a viral gene therapy for FOXG1 syndrome developed at the University at Buffalo. The first-in-human trial will be independently sponsored by the FOXG1 Research Foundation.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07293546 |
Phase 1/2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients With FOXG1 Syndrome (FS) |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT06938542 |
Palliative Care Needs of Children With Rare Diseases and Their Families |
ENROLLING_BY_INVITATION | NA |
| NCT02738281 |
Natural History of Rett Syndrome & Related Disorders |
COMPLETED |
