Clinical Trials Directory

Trials / Active Not Recruiting

Active Not RecruitingNCT07054632

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

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)

Status
Active Not Recruiting
Phase
Phase 3
Study type
Interventional
Enrollment
30 (actual)
Sponsor
Innostellar Biotherapeutics Co.,Ltd · Industry
Sex
All
Age
6 Years
Healthy volunteers
Not accepted

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.

Conditions

Interventions

TypeNameDescription
GENETICLX101Subretinal administration of LX101 to the study eye

Timeline

Start date
2023-09-13
Primary completion
2025-08-13
Completion
2029-08-01
First posted
2025-07-08
Last updated
2026-03-05

Locations

4 sites across 1 country: China

Source: ClinicalTrials.gov record NCT07054632. Inclusion in this directory is not an endorsement.