drugset / Trial / NCT07054632
Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
RandomizedParallel-groupOpen-labelTreatment
Summary
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.
Timeline
- Start
- 2023-09-13
- Primary completion
- 2025-08-13
- Completion
- 2029-08
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | LX101 | Gene therapy (AAV / viral vector) | — | Other |