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

December 25, 2023 updated by: Innostellar Biotherapeutics Co.,Ltd

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

9

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
      • Beijing, China
        • Beijing Tongren Hospital, Capital Medical University
      • Shanghai, China
        • Shanghai General Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: LX101 Dose 1
Genetic: LX101
Subretinal Administration
Experimental: LX101 Dose 2
Genetic: LX101
Subretinal Administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
12 months
Incidence of dose-limiting toxicity (DLT)
Time Frame: 1 month
Incidence of DLT following LX101 subretinal injection at different doses
1 month

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Efficacy of LX101 in study eye
Time Frame: 12 months
Changes in BCVA from baseline
12 months
Efficacy of LX101 in study eye
Time Frame: 12 months
Changes in full-field stimulus threshold (FST) from baseline
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Innostellar Biotherapeutics Co.,Ltd

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 2, 2022

Primary Completion (Actual)

December 6, 2023

Study Completion (Estimated)

December 1, 2027

Study Registration Dates

First Submitted

December 25, 2023

First Submitted That Met QC Criteria

December 25, 2023

First Posted (Actual)

January 9, 2024

Study Record Updates

Last Update Posted (Actual)

January 9, 2024

Last Update Submitted That Met QC Criteria

December 25, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • INNOSTELLAR-LX101-1 (phase 1)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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