July 2019
Volume 60, Issue 9
Free
ARVO Annual Meeting Abstract  |   July 2019
Visual Function Questionnaire Responses in the Voretigene Neparvovec Phase 3 Trial
Author Affiliations & Notes
  • Stephen R Russell
    Ophthalmology/Institute for Vision Reserch, University of Iowa, Iowa City, Iowa, United States
  • Albert M. Maguire
    Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
    Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, United States
  • Jean Bennett
    Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
    Ophthalmology, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, United States
  • Paulo Falabella
    Spark Therapeutics, Inc., Philadelphia, Pennsylvania, United States
  • Elliott H Sohn
    Ophthalmology/Institute for Vision Reserch, University of Iowa, Iowa City, Iowa, United States
  • Vinit B Mahajan
    Ophthalmology and Visual Sciences, Stanford University School of Medicine, Palo Alto, California, United States
  • Daniel C Chung
    Spark Therapeutics, Inc., Philadelphia, Pennsylvania, United States
  • Kathleen Z Reape
    Spark Therapeutics, Inc., Philadelphia, Pennsylvania, United States
  • Katherine A High
    Spark Therapeutics, Inc., Philadelphia, Pennsylvania, United States
  • Footnotes
    Commercial Relationships   Stephen Russell, IDx Technologies, Inc (I), Novartis (C), ProQR (F), Sanofi (C), Spark Therapeutics, Inc. (F), Spark Therapeutics, Inc. (C); Albert Maguire, Foundation Fighting Blindness (F), Spark Therapeutics, Inc. (F), Spark Therapeutics, Inc. (C); Jean Bennett, Foundation Fighting Blindness (F), Spark Therapeutics, Inc. (F), Spark Therapeutics, Inc. (C); Paulo Falabella, Spark Therapeutics, Inc. (E); Elliott Sohn, None; Vinit Mahajan, None; Daniel Chung, Spark Therapeutics, Inc. (E); Kathleen Reape, Spark Therapeutics, Inc. (E); Katherine High, Spark Therapeutics, Inc. (E)
  • Footnotes
    Support  grant to University of Iowa, Children's Hospital of Philadelphia subsequently Spark Therapeutics
Investigative Ophthalmology & Visual Science July 2019, Vol.60, 4968. doi:
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      Stephen R Russell, Albert M. Maguire, Jean Bennett, Paulo Falabella, Elliott H Sohn, Vinit B Mahajan, Daniel C Chung, Kathleen Z Reape, Katherine A High; Visual Function Questionnaire Responses in the Voretigene Neparvovec Phase 3 Trial. Invest. Ophthalmol. Vis. Sci. 2019;60(9):4968.

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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.

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