Efficacy and Safety of Gene Therapy rAAV-RPE65 (LX101) in Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy: a Phase III, Multicenter, Randomized Controlled Trial (STAR)

Study 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.

Interventions

Study Locations

Facility	City	State	Country
Zhongshan Ophthalmic Center, Sun Yat sen University	Guangzhou	Guangdong	China
Southwest Hospital of AMU	Chongqing		China
Shanghai General Hospital	Shanghai		China
Tianjin Medical University Ophthalmology Hospital	Tianjin		China

Official Trial Information

View on ClinicalTrials.gov