Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT07054632

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Inherited Retinal Dystrophy Associated With RPE65 Mutations

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Zhongshan Ophthalmic Center, Sun Yat sen University, Guangzhou, Guangdong, China

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

LX101

Genetic

Subretinal administration of LX101 to the study eye

Primary outcomes

  1. Mobility Test

    Time frame: 12 months

    Changes in functional vision from baseline, determined by mobility test score

Secondary outcomes

  1. 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

  2. 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

  3. Mobility Test

    Time frame: 6 months

    Changes in functional vision from baseline, determined by mobility test score

  4. 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

Sponsors and collaborators

Lead sponsor

Innostellar Biotherapeutics Co.,Ltd

Industry

Registry information

Official study title

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)

Important dates

Study start
2023
Primary completion
2025
Study completion
2029
First posted
Jul 8, 2025
Registry last updated
Mar 5, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.