drugset / Trial / NCT06224907
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
NaSingle-groupOpen-labelTreatment
Summary
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.
Timeline
- Start
- 2023-12-25
- Primary completion
- 2025-04-16
- Completion
- 2029-03
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | valoctocogene roxaparvovec | Gene therapy (AAV / viral vector) | 6e+13 vg/kg | Intravenous |