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

NCT ID: NCT06196827

Last Updated: 2025-07-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-07-02

Study Completion Date

2027-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Inherited Retinal Dystrophy Associated With RPE65 Mutations

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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LX101 Dose 1

Group Type EXPERIMENTAL

LX101

Intervention Type GENETIC

Subretinal Administration

LX101 Dose 2

Group Type EXPERIMENTAL

LX101

Intervention Type GENETIC

Subretinal Administration

Interventions

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LX101

Subretinal Administration

Intervention Type GENETIC

Eligibility Criteria

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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.
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Innostellar Biotherapeutics Co.,Ltd

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Beijing Tongren Hospital, Capital Medical University

Beijing, , China

Site Status

Shanghai General Hospital

Shanghai, , China

Site Status

Countries

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China

Other Identifiers

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INNOSTELLAR-LX101-1 (phase 1)

Identifier Type: -

Identifier Source: org_study_id

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