drugset / Trial / NCT07603050
A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease
Non-randomizedSingle-groupOpen-labelTreatment
Summary
A phase I/II clinical study to evaluate the tolerance, safety and efficacy of VGN-R08b intracerebroventricular injection in patients with type III Gaucher's disease
Timeline
- Start
- 2026-05-14
- Primary completion
- 2028-05-29
- Completion
- 2032-08-16
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | VGN-R08b | Gene therapy (AAV / viral vector) | 6e+10 unknown | Other |
| Subject | VGN-R08b | Gene therapy (AAV / viral vector) | 1.2e+11 unknown | Other |
| Subject | VGN-R08b | Gene therapy (AAV / viral vector) | 1.8e+11 unknown | Other |