RPE65 Gene Therapy （LX101）for Leber's Congenital Amaurosis

• Status: Not yet recruiting
• Trial end date: 2023-06-01
• Target enrollment: 9
• Participant gender: All

Summary

The purpose of this study is to determine whether gene transfer(LX101) will be safe and effective in the treatment of Leber Congenital Amaurosis (LCA).

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Criteria

Inclusion Criteria:

Age 4 years old or older at the time of administration； Diagnosed with LCA; Molecular diagnosis of LCA due to RPE65 mutations (homozygotes or compound heterozygotes) by a qualified laboratory; Visual acuity ≤ 20/200 or visual field less than 20 degrees in the eye to be injected； Must be willing to adhere to protocol and companion protocol for long-term follow-up as evidenced by written informed consent or parental permission and subject assent.

Exclusion Criteria:

Unable or unwilling to meet requirements of the study； Participation in a clinical study with an investigational drug in the past six months； Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints (for example, glaucoma, corneal or lenticular opacities)； Lack of sufficient viable retinal cells as determined by non-invasive means, such as optical coherence tomography (OCT) and/or ophthalmoscopy. Specifically, if indirect ophthalmoscopy reveals less than 1 disc area of retina which is not involved by complete retinal degeneration (indicated by geographic atrophy, thinning with tapetal sheen, or confluent intraretinal pigment migration), these eyes will be excluded. In addition, in eyes where optical coherence tomography (OCT) scans of sufficient quality can be obtained, areas of retina with thickness measurements less than 100 um, or absence of neural retina, will not be targeted for delivery of LX101（AAV2-RPE65）； Complicating systemic diseases or clinically significant abnormal baseline laboratory values.

Prior ocular surgery within six months. Known sensitivity to medications planned for use in the peri-operative period. Individuals of childbearing potential who are pregnant or unwilling to use effective contraception for the duration of the study.

Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study.