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

Phase 1

Active, not recruiting

• Conditions: Inherited Retinal Dystrophy Associated With RPE65 Mutations
• Interventions: Genetic: LX101

• Registration Number: NCT06196827
• Lead Sponsor: Innostellar Biotherapeutics Co.,Ltd

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2022-07-02
• Status Verified: 2023-12
• Primary Completion: 2023-12-06
• Study First Submitted: 2023-12-25
• Study First Submitted QC: 2023-12-25
• Last Update Submitted: 2023-12-25
• Study First Posted: 2024-01-09
• Last Update Posted: 2024-01-09
• Study Completion: 2027-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 9

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
LX101 Dose 1	LX101	-
LX101 Dose 2	LX101	-

Primary Outcome Measures

Name	Time	Method
Incidence of dose-limiting toxicity (DLT)	1 month	Incidence of DLT following LX101 subretinal injection at different doses
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

Secondary Outcome Measures

Name	Time	Method
Efficacy of LX101 in study eye	12 months	Changes in full-field stimulus threshold (FST) from baseline

Trial Locations

Locations (2)

Beijing Tongren Hospital, Capital Medical University; 🇨🇳 Beijing, China

Shanghai General Hospital; 🇨🇳 Shanghai, China