RecruitingEarly Phase 1NCT07063030

A Study of LX107 Gene Therapy in AIPL1-IRD Patients

An Exploratory Clinical Study to Evaluate LX107 Gene Therapy in Patients With AIPL1 Biallelic Mutation-related Inherited Retinal Dystrophy (AIPL1-IRD)


Sponsor

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Enrollment

13 participants

Start Date

Jul 15, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

Administering subretinal injection of LX107 injection (a gene therapy drug) to patients with retinal dystrophy caused by AIPL1 gene mutation to evaluate its efficacy and safety.


Eligibility

Min Age: 4 Years

Plain Language Summary

Simplified for easier understanding

This study tests a gene therapy (LX107) for people with a rare inherited eye condition called AIPL1-associated inherited retinal dystrophy, which causes progressive vision loss due to a faulty gene. The therapy aims to deliver a working copy of the gene directly into the eye. **You may be eligible if...** - You are 4 years old or older - You have been diagnosed with inherited retinal dystrophy caused by mutations in both copies of the AIPL1 gene, confirmed by genetic testing - The vision in your study eye is significantly reduced (visual acuity of 0.3 or less on standard testing) - You or your guardian can consent to long-term follow-up **You may NOT be eligible if...** - You do not have confirmed AIPL1 gene mutations - Your eye has certain structural conditions that could affect the safety or success of the injection - You are unable to comply with long-term follow-up requirements Talk to your doctor to see if this trial is right for you.

This is a simplified summary. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICLX107 Injection

Subjects will receive subretinal injection of LX107 on Day 0.


Locations(1)

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, China

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NCT07063030


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