Clinical Trial

Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations

Active, Not Recruiting Phase 3
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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.
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2025-06-27; most recent amendment 2026-03-04.
Trial Details
NCT Number NCT07054632
Lead Sponsor Innostellar Biotherapeutics Co.,Ltd
Conditions Inherited Retinal Dystrophy Associated With RPE65 Mutations
Enrollment 30 participants
Start Date 2023-09-13
Primary Completion 2025-08-13 (estimated)
Study Completion 2029-08 (estimated)
Updated on ClinicalTrials.gov 2026-03-05