Inherited Retinal Dystrophy Associated With RPE65 Mutations
Conditions
Brief summary
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.
Interventions
GENETICLX101
Subretinal administration of LX101 to the study eye
Sponsors
Innostellar Biotherapeutics Co.,Ltd
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subject and/or their guardian signing a written informed consent. Diagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy. Subjects are 6 years of age or older. Visual acuity of ≤ 20/63 or visual field less than 20 degrees in the eye to be injected.
Exclusion criteria
* Prior gene therapy for IRD and other hereditary eye diseases. Pre-existing eye conditions that would interfere with interpretation of study endpoints. Active intraocular or periocular infections in the study eye. Lacking of sufficient surviving retinal cells. Prior ocular surgery within six months. Complicating systemic diseases or clinically significant abnormal baseline laboratory values. Pre-existing systemic diseases that should not discontinue the use of any retinal toxic compounds. Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mobility Test | 12 months | Changes in functional vision from baseline, determined by mobility test score |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Full-field Light Sensitivity Threshold (FST) Test | 6 months、12 months | Changes in light sensitivity from baseline, assessed by FST in log cd.s/m2 |
| Visual Acuity | 6 months、12 months | Changes in visual acuity from baseline, based on the ability to read letters using the Early Treatment Diabetic Retionpathy Study (ETDRS) chart |
| Mobility Test | 6 months | Changes in functional vision from baseline, determined by mobility test score |
| Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs) | 12 months | Incidence of ocular and non-ocular AEs and SAEs following LX102 subretinal injection |
Countries
China
Outcome results
None listed