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NCT Number: NCT06196827

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Inherited Retinal Dystrophy Associated With RPE65 Mutations

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Beijing Tongren Hospital, Capital Medical University, Beijing, China

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

LX101

Genetic

Subretinal Administration

Primary outcomes

  1. Incidence of adverse events (AEs) and serious adverse events (SAEs)

    Time frame: 12 months

    Incidence of ocular and non-ocular AEs and SAEs following LX101 subretinal injection

  2. Incidence of dose-limiting toxicity (DLT)

    Time frame: 1 month

    Incidence of DLT following LX101 subretinal injection at different doses

Secondary outcomes

  1. Efficacy of LX101 in study eye

    Time frame: 12 months

    Changes in BCVA from baseline

  2. Efficacy of LX101 in study eye

    Time frame: 12 months

    Changes in full-field stimulus threshold (FST) from baseline

Sponsors and collaborators

Lead sponsor

Innostellar Biotherapeutics Co.,Ltd

Industry

Registry information

Official study title

A Multi-center Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of rAAV2-RPE65 Gene Therapy (LX101) in Subjects With Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy

Important dates

Study start
2022
Primary completion
2023
Study completion
2027
First posted
Jan 9, 2024
Registry last updated
Mar 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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