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

A Multi-center Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of rAAV2-RPE65 Gene Therapy (LX101) in Subjects With Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy

Status

Active, not recruiting

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06196827

Enrollment

Registered

2024-01-09

Start date

2022-07-02

Completion date

2027-12-01

Last updated

2026-03-06

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

Conditions

Inherited Retinal Dystrophy Associated With RPE65 Mutations

Brief summary

The purpose of the study is to evaluate the safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy.

Interventions

GENETICLX101

Subretinal Administration

Study design

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

6 Years to No maximum

Healthy volunteers

Inclusion criteria

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.

Exclusion criteria

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.

Design outcomes

Primary

Measure	Time frame	Description
Incidence of adverse events (AEs) and serious adverse events (SAEs)	12 months	Incidence of ocular and non-ocular AEs and SAEs following LX101 subretinal injection
Incidence of dose-limiting toxicity (DLT)	1 month	Incidence of DLT following LX101 subretinal injection at different doses

Secondary

Measure	Time frame	Description
Efficacy of LX101 in study eye	12 months	Changes in BCVA from baseline

Countries

China

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 7, 2026