Visual Function Questionnaire Responses in the Voretigene Neparvovec Phase 3 Trial

Abstract

Purpose : To report the pre- and post-intervention changes in visual function questionnaire (VFQ) responses for voretigene neparvovec (VN) within the Phase 3 trial.

Methods : Subjects with biallelic RPE65 mutations were enrolled and randomized 2:1 to receive bilateral, subretinal injections of VN (original intervention group) versus observation (controls). After 1 year, controls were allowed to receive VN. At each visit, subjects and, separately, parents of subjects < age 18 completed a 25-question VFQ adapted for adult and pediatric subjects with inherited retinal disorders and developed to assess vision-dependent activities of daily living (ADL). Perceived difficulty of each ADL was reported utilizing a numerical scale from 0 (worst performance/vision) to 10 (best performance/vision), and an average test score was calculated for each time point and for each subject (+/- parent).

Results : Mean (SD) VFQ Baseline average scores were 4.6 (1.5) for subjects (n=29) and 3.5 (1.4) for parents (n=20). For the Original Intervention group, mean VFQ change from baseline at Year 1 was +2.6 (1.8) for subjects (n=20) and +3.9 (1.9) for parents (n=15), while Original Control mean change from baseline was +0.1 (1.4) for subjects (n=9) [comparison of Intervention versus Control groups at year 1, nominal p = 0.001] and -0.2 (1.3) for parents (n=5) [nominal p = 0.002].

Conclusions : Subject- and parent-completed VFQ showed significant, durable improvement after VN treatment.

This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.