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

NCT ID: NCT07054632

Last Updated: 2025-07-10

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

PHASE3

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-09-13

Study Completion Date

2029-08-31

Brief Summary

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The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. This is an open-label, multicenter, randomized controlled Phase III clinical trial. Subjects were randomly assigned in a 1:1 ratio to either the intervention group or the control group. Subjects in the intervention group received subretinal injection of LX101, while those in the control group received no treatment.

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

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Intervention group

Group Type EXPERIMENTAL

LX101

Intervention Type GENETIC

Subretinal administration of LX101 to the study eye

Control group

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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LX101

Subretinal administration of LX101 to the study eye

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.

Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
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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Zhongshan Ophthalmic Center, Sun Yat sen University

Guangzhou, Guangdong, China

Site Status

Southwest Hospital of AMU

Chongqing, , China

Site Status

Shanghai General Hospital

Shanghai, , China

Site Status

Tianjin Medical University Ophthalmology Hospital

Tianjin, , China

Site Status

Countries

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China

Other Identifiers

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INNOSTELLAR-LX101-1 (phase 2)

Identifier Type: -

Identifier Source: org_study_id

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