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.
Grassi MA. Toward a Treatment Trial for Stargardt Disease: Putting Out the Fire. JAMA Ophthalmol. Published online August 01, 2019. doi:10.1001/jamaophthalmol.2019.2930
Browse and subscribe to JAMA Network podcasts!
Customize your JAMA Network experience by selecting one or more topics from the list below.
Create a personal account or sign in to: