drugset / Trial / NCT06432140
A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency
NaSingle-groupOpen-labelTreatment
Summary
This trial includes dose-escalating part (phase 1) and dose confirming part, to prove the safety and efficacy of VGN-R09b to treat patients with severe AADC deficiency
Timeline
- Start
- 2024-07-02
- Primary completion
- 2026-07-23
- Completion
- 2030-09-20
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | VGN-R09b | Gene therapy (AAV / viral vector) | 6e+11 vg | Other |
| Subject | VGN-R09b | Gene therapy (AAV / viral vector) | 1.28e+12 vg | Other |