Trials / NCT05906953
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
Status unknown Phase 1 / Phase 2 Gene therapy
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
- Treatment
- HG004
- Gene
- RPE65
- Conditions
- Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
- Ages
- 6 Years to 50 Years
- Enrollment
- 20 participants
- Started
- 2023-10-31
- Main results expected
- 2025-12
- Results posted
- Not yet
- Record last updated
- 2024-09-19
What the study is testing
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.
Where it runs
China (1)
- Research Site, Shanghai, Shanghai Municipality (recruiting now)
United States (2)
- Research Site, Sacramento, California (recruiting now)
- Research Site, Houston, Texas (recruiting now)