drugset / Trial / NCT05906953
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
NaSingle-groupOpen-labelTreatment
Summary
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.
Timeline
- Start
- 2023-10-31
- Primary completion
- 2025-12
- Completion
- 2025-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | HG004 | Gene therapy (AAV / viral vector) | — | — |