Trials / NCT06088992
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)
Underway, not enrolling Early Phase 1 Gene therapy
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
- Treatment
- HG004
- Gene
- RPE65
- Conditions
- Leber Congenital Amaurosis
- Ages
- 8 Years to 50 Years
- Enrollment
- 9 participants
- Started
- 2023-01-10
- Main results expected
- 2024-10-30
- 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 mutationsin RPE65 gene.
Where it runs
China (1)
- Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine, Shanghai, Shanghai Municipality