Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy Associated With ND4 Mutations
Status:
Recruiting
Trial end date:
2027-04-30
Target enrollment:
Participant gender:
Summary
The objective of this clinical study is to select the optimal dose and evaluate the safety
and efficacy of the treatment. Stage 1 is a dose-finding study, which will enroll subjects
aged ≥ 18 years old and ≤ 75 years old to receive a single unilateral intravitreal (IVT)
injection of NR082 to observe its safety and efficacy. Stage 2 of the clinical trial will be
conducted after the dose is determined to further evaluate the safety and efficacy of the
study drug. In Stage 2 of the study, the first 6 subjects are aged ≥ 18 years and ≤ 75 years.
After monitoring for at least 6 weeks, if there are no new safety signals and the efficacy
data is similar to Stage 1, subjects aged 12-17 years can be enrolled upon approval by the
Independent Data Monitoring Committee (IDMC). The clinical manifestation of all subjects is
reduced visual acuity caused by Leber hereditary optic neuropathy (LHON) associated with ND4
mutation, and laboratory test showed G11778A mutation (a CLIA-certified laboratory), while
the reduced visual acuity lasted for >6 months and <10 years.