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

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06212297

Enrollment

Registered

2024-01-18

Start date

2023-09-12

Completion date

2029-03-31

Last updated

2025-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Inherited Retinal Dystrophy

Keywords

RPE65 Mutations

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.

Interventions

GENETICLX101

Subretinal administration of LX101 to the contralateral, previously uninjected eye

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

6 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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
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
Mobility Test	6 months、12 months	Changes in functional vision from baseline, determined by mobility test score

Countries

China

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026