Fellow-eye Study (FE) of LX101 in Subjects With Inherited Retinal Dystrophy

A Clinical Trial to Evaluate the Safety and Efficacy of Subretinal Re-Administration of LX101 to the Contralateral Eye in Subjects With Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy

Up to nine subjects who have participated in the earlier LX101 clinical study, and who meet all study eligibility criteria, will receive LX101 administration in the previously uninjected, contralateral eye to evaluate the safety of bilateral, sequential subretinal administration of LX101.

Study Overview

• Status: Active, not recruiting
• Conditions: Inherited Retinal Dystrophy
• Intervention / Treatment: Genetic: LX101

• Study Type: Interventional
• Enrollment (Estimated): 9
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China, 200080
      • Shanghai General Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Prior subretinal administration of LX101 (unilateral)

≥ 6 years old Signed written informed consent

Exclusion Criteria:

Prior gene therapy except LX101 Active intraocular or periocular infections Lacking of sufficient surviving retinal cells Prior ocular surgery within six months Retinoid like compounds or precursors were taken within three months Complicating systemic diseases Clinically significant abnormal baseline laboratory values Using of any retinal toxic compounds

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: LX101 Subretinal Administration	Genetic: LX101; Subretinal administration of LX101 to the contralateral, previously uninjected eye

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs)	Incidence of ocular and non-ocular AEs and SAEs following LX102 subretinal injection	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Full-field Light Sensitivity Threshold (FST) Test	Changes in light sensitivity from baseline, assessed by FST in log cd.s/m2	6 months、12 months
Visual Acuity	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	Changes in functional vision from baseline, determined by mobility test score	6 months、12 months

Collaborators and Investigators

• Sponsor: Innostellar Biotherapeutics Co.,Ltd

Study record dates

Study Major Dates

• Study Start (Actual): September 12, 2023
• Primary Completion (Actual): February 6, 2025
• Study Completion (Estimated): March 1, 2029

Study Registration Dates

• First Submitted: December 25, 2023
• First Submitted That Met QC Criteria: January 8, 2024
• First Posted (Actual): January 18, 2024

Study Record Updates

• Last Update Posted (Actual): June 29, 2025
• Last Update Submitted That Met QC Criteria: June 27, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Keywords: RPE65 Mutations
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Diseases; Retinal Degeneration; Retinal Dystrophies
• Other Study ID Numbers: INNOSTELLAR-LX101-2

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No