Trials / NCT07054632
Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
Underway, not enrolling Phase 3 Gene therapy
- Sponsor
- Innostellar Biotherapeutics Co.,Ltd
- Treatment
- LX101
- Gene
- RPE65
- Conditions
- Inherited Retinal Dystrophy Associated With RPE65 Mutations
- Ages
- 6 Years to no upper limit
- Enrollment
- 30 participants
- Started
- 2023-09-13
- Main results expected
- 2025-08-13
- Results posted
- Not yet
- Record last updated
- 2026-03-05
What the study is testing
The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. This is an open-label, multicenter, randomized controlled Phase III clinical trial. Subjects were randomly assigned in a 1:1 ratio to either the intervention group or the control group. Subjects in the intervention group received subretinal injection of LX101, while those in the control group received no treatment.
Where it runs
China (4)
- Zhongshan Ophthalmic Center, Sun Yat sen University, Guangzhou, Guangdong
- Southwest Hospital of AMU, Chongqing
- Shanghai General Hospital, Shanghai
- Tianjin Medical University Ophthalmology Hospital, Tianjin