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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
9
Subretinal Administration
Beijing Tongren Hospital, Capital Medical University
Beijing, China
Shanghai General Hospital
Shanghai, China
Incidence of adverse events (AEs) and serious adverse events (SAEs)
Incidence of ocular and non-ocular AEs and SAEs following LX101 subretinal injection
Time frame: 12 months
Incidence of dose-limiting toxicity (DLT)
Incidence of DLT following LX101 subretinal injection at different doses
Time frame: 1 month
Efficacy of LX101 in study eye
Changes in BCVA from baseline
Time frame: 12 months
Efficacy of LX101 in study eye
Changes in full-field stimulus threshold (FST) from baseline
Time frame: 12 months
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