The objective of this clinical study is to evaluate the safety and efficacy of NR082 in the treatment of LHON caused by mitochondrial ND4 gene mutation. This study will enroll subjects aged ≥ 12 years old and ≤ 75 years old to receive a single bilateral intravitreal (IVT) injection of NR082 to evaluate safety and efficacy. The clinical manifestations of all subjects are to be reduced visual acuity caused by LHON associated with ND4 mutation, with laboratory test showing G11778A mutation and reduced visual acuity lasted for \>6 months and \<10 years.
Safety run-in phase: The safety run-in phase will enroll 6 evaluable subjects aged ≥ 12 years and ≤ 75 years , namely 4.5 x 109 vg, 0.05 mL eye/dose(bilaterally) and monitor the safety for at least 6 weeks. If there is no new safety concern evaluated by the SRC, the randomized, double-blind, sham-injection control study can be initiated. Second Stage: randomized, double-blind, sham-injection control study: The randomized, double-blind, sham-injection control study is to verify the efficacy and safety of NR082 in LHON caused by mitochondrial gene ND4 mutation . This part is divided into the NR082 treatment group and the control group (sham-injection group).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
95
a single bilateral intravitreal (IVT) injection of NR082
sham-injection
Beijing Tongren Hospital, Capital Medical University
Beijing, Beijing Municipality, China
Efficacy of NR082 in study eye
Proportion of ≥ 0.3 LogMAR from baseline in BCVA in the study eye in the NR082 treatment and the sham-injection at Week 52 after treatment
Time frame: 52 weeks
Efficacy of NR082 in study eye and non-study eye
Mean change from baseline in BCVA;
Time frame: 52 weeks
Mean change from baseline in visual field, contrast sensitivity
Efficacy of NR082 in study eye and non-study eye
Time frame: 52 weeks
Safety and tolerability of NR082
Incidence rates of AEs, SAEs within 52 weeks after injection of NR082
Time frame: 52 weeks
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