Overview

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.