NCT07054632

Active Not RecruitingPhase 3

Efficacy and Safety of LX101 for Inherited Retinal Dystrophy...

Innostellar Biotherapeutics Co.,Ltd Source

ClinicalTrials.gov

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Other study identifiers

Sponsor study identifier

INNOSTELLAR-LX101-1 (stage 2)

Study Type

interventional

Actual enrollment (reported)

30

Locations

1 country

Sites

4

Timeline

Study Start

September 13, 2023

Actual (reported)

Primary Completion

August 13, 2025

Actual (reported)

Study Completion

August 2029

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

Brief Summary

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.

Registry Overview

Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.

Recruitment status: active not recruiting
Actual enrollment (reported): 30 participants
Study completion date: August 2029
Estimated
Planned date reported by the registry; this milestone is not confirmed as achieved.
Listed locations: 1 countries · 4 sites

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.

Study Start
September 13, 2023
Actual (reported)
First Submitted
June 27, 2025
Actual (reported)
First Posted
July 8, 2025
Actual (reported)
Primary Completion
August 13, 2025
Actual (reported)
Study Completion
August 2029
Estimated
Planned date reported by the registry; this milestone is not confirmed as achieved.

Last Updated: March 5, 2026
Status Verified: March 2026
Last Update Submitted: March 4, 2026

Conditions

Inherited Retinal Dystrophy Associated With RPE65 Mutations

Outcome Measures

Primary Outcomes (1)

Mobility Test
Changes in functional vision from baseline, determined by mobility test score
12 months

Secondary Outcomes (4)

Full-field Light Sensitivity Threshold (FST) Test
6 months、12 months
Visual Acuity
6 months、12 months
Mobility Test
6 months
Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs)
12 months

Study Arms (2)

Intervention group

EXPERIMENTAL

Genetic: LX101

Control group

NO INTERVENTION

Interventions

LX101GENETIC

Subretinal administration of LX101 to the study eye

Intervention group

Eligibility Criteria

Age6 Years+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

Subject and/or their guardian signing a written informed consent.
Diagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy.

Sponsors & Collaborators

Innostellar Biotherapeutics Co.,Ltdlead

Study Sites (4)

Zhongshan Ophthalmic Center, Sun Yat sen University

Guangzhou, Guangdong, China

Southwest Hospital of AMU

Chongqing, China

Shanghai General Hospital

Shanghai, China

Tianjin Medical University Ophthalmology Hospital

Tianjin, China

Enrollment

30 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 3
Allocation: RANDOMIZED
Masking: NONE
Purpose: TREATMENT
Intervention Model: PARALLEL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

June 27, 2025

First Posted

July 8, 2025

Study Start

September 13, 2023

Actual (reported)

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.

You may not qualify if:

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.

Contact the study team to confirm eligibility.

Primary Completion

August 13, 2025

Actual (reported)

Study Completion

August 2029

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

Last Updated

March 5, 2026

Record last verified: 2026-03

Source record

Download the complete ClinicalTrials.gov record imported by Syfrah, including fields not shown in this page. The registry may contain a newer revision.

Open registry record

Source retrieved: 2026-10-25 22:28:12 UTC

Locations

CN(4)

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (4)

Study Arms (2)

Intervention group

Control group

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (4)

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Locations