Inherited Retinal Dystrophy Associated With RPE65 Mutations
Investigational drug(s) / intervention(s)
LX101
LX101: Subretinal Administration
Study summary
The purpose of the study is to evaluate the safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy.
Eligibility
Sex
ALL
Min age
6 Years
Max age
—
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.
Primary outcome measure(s)
Incidence of adverse events (AEs) and serious adverse events (SAEs) — 12 months Incidence of ocular and non-ocular AEs and SAEs following LX101 subretinal injection
Incidence of dose-limiting toxicity (DLT) — 1 month Incidence of DLT following LX101 subretinal injection at different doses
Trial sites (2)
Facility
City
Region
Status
Beijing Tongren Hospital, Capital Medical University
Beijing
China
Shanghai General Hospital
Shanghai
China
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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