NCT06196827

Active Not RecruitingPhase 1

Safety and Tolerability of LX101 for Inherited Retinal Dystr...

Innostellar Biotherapeutics Co.,Ltd Source

ClinicalTrials.gov

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Other study identifiers

Sponsor study identifier

INNOSTELLAR-LX101-1 (stage 1)

Study Type

interventional

Actual enrollment (reported)

9

Locations

1 country

Sites

2

Timeline

Study Start

July 2, 2022

Actual (reported)

Primary Completion

December 6, 2023

Actual (reported)

Study Completion

December 2027

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 safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy.

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): 9 participants
Study completion date: December 2027
Estimated
Planned date reported by the registry; this milestone is not confirmed as achieved.
Listed locations: 1 countries · 2 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
July 2, 2022
Actual (reported)
Primary Completion
December 6, 2023
Actual (reported)
First Submitted
December 25, 2023
Actual (reported)
First Posted
January 9, 2024
Actual (reported)
Study Completion
December 2027
Estimated
Planned date reported by the registry; this milestone is not confirmed as achieved.

Last Updated: March 6, 2026
Status Verified: March 2026
Last Update Submitted: March 4, 2026

Conditions

Inherited Retinal Dystrophy Associated With RPE65 Mutations

Outcome Measures

Primary Outcomes (2)

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

Secondary Outcomes (2)

Efficacy of LX101 in study eye
12 months
Efficacy of LX101 in study eye
12 months

Study Arms (2)

LX101 Dose 1

EXPERIMENTAL

Genetic: LX101

LX101 Dose 2

EXPERIMENTAL

Genetic: LX101

Interventions

LX101GENETIC

Subretinal Administration

LX101 Dose 1LX101 Dose 2

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 (2)

Beijing Tongren Hospital, Capital Medical University

Beijing, China

Shanghai General Hospital

Shanghai, China

Enrollment

9 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 1
Allocation: NON RANDOMIZED
Masking: NONE
Purpose: TREATMENT
Intervention Model: PARALLEL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

December 25, 2023

First Posted

January 9, 2024

Study Start

July 2, 2022

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.

Contact the study team to confirm eligibility.

Primary Completion

December 6, 2023

Actual (reported)

Study Completion

December 2027

Estimated

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

Last Updated

March 6, 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 22:20:52 UTC

Locations

CN(2)

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

Primary Completion

First Submitted

First Posted

Study Completion

Conditions

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (2)

Study Arms (2)

LX101 Dose 1

LX101 Dose 2

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (2)

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Locations