The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. This is an open-label, multicenter, randomized controlled Phase III clinical trial. Subjects were randomly assigned in a 1:1 ratio to either the intervention group or the control group. Subjects in the intervention group received subretinal injection of LX101, while those in the control group received no treatment.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Subretinal administration of LX101 to the study eye
Zhongshan Ophthalmic Center, Sun Yat sen University
Guangzhou, Guangdong, China
Southwest Hospital of AMU
Chongqing, China
Shanghai General Hospital
Shanghai, China
Tianjin Medical University Ophthalmology Hospital
Tianjin, China
Mobility Test
Changes in functional vision from baseline, determined by mobility test score
Time frame: 12 months
Full-field Light Sensitivity Threshold (FST) Test
Changes in light sensitivity from baseline, assessed by FST in log cd.s/m2
Time frame: 6 months、12 months
Visual Acuity
Changes in visual acuity from baseline, based on the ability to read letters using the Early Treatment Diabetic Retionpathy Study (ETDRS) chart
Time frame: 6 months、12 months
Mobility Test
Changes in functional vision from baseline, determined by mobility test score
Time frame: 6 months
Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs)
Incidence of ocular and non-ocular AEs and SAEs following LX102 subretinal injection
Time frame: 12 months
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