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

June 27, 2025 updated by: Innostellar Biotherapeutics Co.,Ltd

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

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

9

Phase

  • Phase 2
  • Phase 1

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

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:

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

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

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)
Time Frame: 6 months
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
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、12 months
Changes in functional vision from baseline, determined by mobility test score
6 months、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 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

Additional Relevant MeSH Terms

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

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.

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