Study design and baseline characteristics for the reflect gene therapy trial ofm.11778g>A/ND4-LHON

ABSTRACT:

Objective

REFLECT is the first randomised, double-masked, placebo-controlled multicentre phase 3 clinical trial that evaluated the efficacy and safety of bilateral intravitreal (IVT) injection of lenadogene nolparvovec in subjects with Leber hereditary optic neuropathy carrying the m.11778G>A mutation.

Methods and analysis

A total of 98 subjects were enrolled with vision loss of ≤12 months. The subjects were randomised to one of two treatment arms with all subjects receiving an intravitreal (IVT) injection of lenadogene nolparvovec in their first affected eye and the second-affected eye randomised to receive IVT of either lenadogene nolparvovec or placebo.

Results

The majority of subjects were male with a mean duration of vision loss of 8.3 months. All but one su

SUBMITTER: Subramanian P 

PROVIDER: S-EPMC9723886 | biostudies-literature | 2022 Jan

REPOSITORIES: biostudies-literature