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Early Phase 1 Study of LX107 Gene Therapy for Patients Aged 4 and Older with AIPL1-Related Inherited Retinal Dystrophy
Led by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine · Updated on 2026-03-10
13
Participants Needed
1
Research Sites
208 weeks
Total Duration
AI-Summary
What this Trial Is About
Researchers are evaluating the use of LX107, a gene therapy drug, in patients with retinal dystrophy caused by mutations in the AIPL1 gene. This early phase 1 clinical trial aims to assess the safety and effectiveness of this treatment for individuals aged 4 years and older who have a confirmed diagnosis of this inherited retinal disease. The study is sponsored by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine. Participants will receive a single subretinal injection of LX107 in one eye, designated as the study eye. The study includes three dose groups low dose 3x109 viral genomes per eye, medium dose 1x1010 viral genomes per eye, and high dose 3x1010 viral genomes per eye. Treatment will be administered on Day 0, and patients will be monitored closely throughout the study. During the study, participants will undergo evaluations such as functional MRI scans, mobility tests, full-field sensitivity threshold tests, visual acuity assessments, and visual field measurements at 6 and 12 months after treatment. Safety will be closely monitored by tracking treatment-emergent adverse events over 6 months. Participants will also be followed long-term according to the study protocol to assess treatment effects and safety over time.
CONDITIONS
Brief Title
A Study of LX107 Gene Therapy in AIPL1-IRD Patients
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