drugset / Trial / NCT06238908
Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
NaSingle-groupOpen-labelTreatment
Summary
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients. NGGT003 uses adeno-associated virus (AAV) as a vector, carrying a liver specific promoter and codon optimized human FVIII gene B domain deletion mutant (hFVIII BDD), and expresses human FVIII protein in the liver through intravenous injection.
Timeline
- Start
- 2024-01-17
- Primary completion
- 2026-01-31
- Completion
- 2030-01-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | NGGT003 | Gene therapy (AAV / viral vector) | 4e+11 vg/kg | Intravenous |
| Subject | NGGT003 | Gene therapy (AAV / viral vector) | 1e+12 vg/kg | Intravenous |
| Subject | NGGT003 | Gene therapy (AAV / viral vector) | 2.5e+12 vg/kg | Intravenous |