drugset / Trial / NCT06088992
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)
Phase 0
Active not recruiting
9 enrolled
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
HuidaGene Therapeutics Co., Ltd. · collab
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 mutationsin RPE65 gene.
Timeline
- Start
- 2023-01-10
- Primary completion
- 2024-10-30
- Completion
- 2028-10-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | HG004 | Gene therapy (AAV / viral vector) | — | Other |