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

Phase 1

Recruiting

• Conditions: Inherited Retinal Dystrophy
• Interventions: Genetic: LX101

• Registration Number: NCT06212297
• Lead Sponsor: Innostellar Biotherapeutics Co.,Ltd

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-09-12
• Study First Submitted: 2023-12-25
• Status Verified: 2024-01
• Study First Submitted QC: 2024-01-08
• Last Update Submitted: 2024-01-08
• Study First Posted: 2024-01-18
• Last Update Posted: 2024-01-18
• Primary Completion: 2024-09
• Study Completion: 2029-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 9

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
LX101 Subretinal Administration	LX101	-

Primary Outcome Measures

Name	Time	Method
Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs)	6 months	Incidence of ocular and non-ocular AEs and SAEs following LX102 subretinal injection

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Shanghai General Hospital; 🇨🇳 Shanghai, Shanghai, China