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Invited Commentary
August 1, 2019

Toward a Treatment Trial for Stargardt Disease: Putting Out the Fire

Author Affiliations
  • 1Grassi Retina, Naperville, Illinois
  • 2Department of Ophthalmology and Visual Sciences, University of Illinois at Chicago
JAMA Ophthalmol. Published online August 1, 2019. doi:10.1001/jamaophthalmol.2019.2930

Determining the best outcome measure for a randomized clinical trial of Stargardt disease is complex. Vision, the most commonly used ophthalmic outcome, does not correlate well with the extent of atrophy, changes very little over a 1-year or 2-year period, and, consequently, lacks sensitivity.1

The purpose of the Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) consortium is to identify a more reliable measure to be used for a therapeutic trial of Stargardt disease. In the present report (ProgStar report No. 11) by Strauss et al,2 the investigators specifically seek to characterize and delineate the natural history and progression of atrophy in Stargardt disease. Importantly, the US Food and Drug Administration has deemed progression of atrophy a suitable primary outcome measure for randomized clinical trials.