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

NCT07054632 | Active Not Recruiting Phase 3

• 1 other identifier: INNOSTELLAR-LX101-1 (stage 2)
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 4

Brief 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

Inherited Retinal Dystrophy Associated With RPE65 Mutations

Outcome Measures

Primary Outcomes (1)

• Mobility Test

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

  12 months

Secondary Outcomes (4)

• Full-field Light Sensitivity Threshold (FST) Test

  6 months、12 months

• Visual Acuity

  6 months、12 months

• Mobility Test

  6 months

• Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs)

  12 months

Study Arms (2)

Intervention group

EXPERIMENTAL

Genetic: LX101

Control group

NO INTERVENTION

Interventions

LX101 GENETIC

Subretinal administration of LX101 to the study eye

Intervention group

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Innostellar Biotherapeutics Co.,Ltd: lead

Study Sites (4)

Zhongshan Ophthalmic Center, Sun Yat sen University

Guangzhou, Guangdong, China

Location

Southwest Hospital of AMU

Chongqing, China

Location

Shanghai General Hospital

Shanghai, China

Location

Tianjin Medical University Ophthalmology Hospital

Tianjin, China

Location

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 27, 2025
• First Posted: July 8, 2025
• Study Start: September 13, 2023
• Primary Completion: August 13, 2025
• Study Completion (Estimated): August 1, 2029
• Last Updated: March 5, 2026

Record last verified: 2026-03

Locations

CN (4)