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

March 4, 2026 updated by: Innostellar Biotherapeutics Co.,Ltd

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)

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

30

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Subject and/or their guardian signing a written informed consent.

Diagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy.

Subjects are 6 years of age or older.

Visual acuity of ≤ 20/63 or visual field less than 20 degrees in the eye to be injected.

Exclusion Criteria:

  • Prior gene therapy for IRD and other hereditary eye diseases.

Pre-existing eye conditions that would interfere with interpretation of study endpoints.

Active intraocular or periocular infections in the study eye.

Lacking of sufficient surviving retinal cells.

Prior ocular surgery within six months.

Complicating systemic diseases or clinically significant abnormal baseline laboratory values.

Pre-existing systemic diseases that should not discontinue the use of any retinal toxic compounds.

Complicating systemic diseases or clinically significant abnormal baseline laboratory values.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
No Intervention: Control group
Experimental: Intervention group
Genetic: LX101
Subretinal administration of LX101 to the study eye

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Mobility Test
Time Frame: 12 months
Changes in functional vision from baseline, determined by mobility test score
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Full-field Light Sensitivity Threshold (FST) Test
Time Frame: 6 months、12 months
Changes in light sensitivity from baseline, assessed by FST in log cd.s/m2
6 months、12 months
Visual Acuity
Time Frame: 6 months、12 months
Changes in visual acuity from baseline, based on the ability to read letters using the Early Treatment Diabetic Retionpathy Study (ETDRS) chart
6 months、12 months
Mobility Test
Time Frame: 6 months
Changes in functional vision from baseline, determined by mobility test score
6 months
Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs)
Time Frame: 12 months
Incidence of ocular and non-ocular AEs and SAEs following LX102 subretinal injection
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Innostellar Biotherapeutics Co.,Ltd

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 13, 2023

Primary Completion (Actual)

August 13, 2025

Study Completion (Estimated)

August 1, 2029

Study Registration Dates

First Submitted

June 27, 2025

First Submitted That Met QC Criteria

June 27, 2025

First Posted (Actual)

July 8, 2025

Study Record Updates

Last Update Posted (Actual)

March 5, 2026

Last Update Submitted That Met QC Criteria

March 4, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • INNOSTELLAR-LX101-1 (stage 2)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Inherited Retinal Dystrophy Associated With RPE65 Mutations

Clinical Trials on LX101

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Saudi Arabia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe