@article{26b3c1ae9e6a4050bf3acf508a90181c,
title = "Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease (SMART) Study: Design and Baseline Characteristics (Report No. 1)",
abstract = " Purpose: To describe the study design and characteristics at first visit of participants in the longitudinal Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease (SMART) study. Methods: Scotopic microperimetry (sMP) was performed in one designated study eye in a subset of participants with molecularly proven ABCA4-associated Stargardt disease (STGD1) enrolled in a multicenter natural history study (ProgStar). Study visits were every 6 months over a period ranging from 6 to 24 months, and also included fundus autofluorescence (FAF). Results: SMART enrolled 118 participants (118 eyes). At the first visit of SMART, the mean sensitivity in mesopic microperimetry was 11.48 (±5.05; range 0.00-19.88) dB and in sMP 11.25 (±5.26; 0-19.25) dB. For FAF, all eyes had a lesion of decreased autofluorescence (mean lesion size 3.62 [±3.48; 0.10-21.46] mm 2 ), and a total of 76 eyes (65.5%) had a lesion of definitely decreased autofluorescence with a mean lesion size of 3.46 (±3.60; 0.21-21.46) mm 2 . Conclusions: Rod function is impaired in STGD1 and can be assessed by sMP. Testing rod function may serve as a potential outcome measure for future clinical treatment trials. This is evaluated in the SMART study.",
keywords = "Clinical trials, Endpoints, Mesopic microperimetry, Scotopic microperimetry, Stargardt disease",
author = "Strauss, {Rupert W.} and Xiangrong Kong and Bittencourt, {Millena G.} and Alexander Ho and Anamika Jha and Sch{\"o}nbach, {Etienne M.} and Ahmed, {Mohamed I.} and Beatriz Mu{\~n}oz and Ervin, {Ann Margret} and Michel Michaelides and Birch, {David G.} and Sahel, {Jos{\'e} Alain} and Sunness, {Janet S.} and Eberhart Zrenner and Saghar Bagheri and Michael Ip and Srinivas Sadda and Sheila West and Scholl, {Hendrik P.N.}",
note = "Funding Information: Dr. Hendrik Scholl is supported by the Foundation Fighting Blindness Clinical Research Institute (FFB CRI); Shulsky Foundation, New York, NY; National Centre of Competence in Research (NCCR) Molecular Systems Engineering (University of Basel and ETH Z{\"u}rich), Swiss National Science Foundation. Dr. Scholl is a paid consultant of the following entities: Boehringer Ingelheim Pharma GmbH & Co. KG; Gerson Lehrman Group; and Guide-point. Dr. Scholl is member of the Scientific Advisory Board of the Astellas Institute for Regenerative Medicine; ReNeuron Group Plc/Ora Inc.; and Vision Medicines, Inc. Dr. Scholl is member of the Data Monitoring and Safety Board/Committee of the following entities: Genentech Inc./F. Hoffmann-La Roche Ltd; and ReNeu-ron Group Plc/Ora Inc. These arrangements have been reviewed and approved by the Johns Hopkins University in accordance with its conflict of interest policies. Johns Hopkins University and Bayer Pharma AG have an active research collaboration and option agreement. These arrangements have also been reviewed and approved by the University of Basel (Universit{\"a}tsspital Basel, USB) in accordance with its conflict of interest policies. Dr. Hendrik Scholl is principal investigator of grants at the USB sponsored by the following entity: Acucela Inc.; NightstaRx Ltd.; Ophthotech Corporation; Grants at USB are negotiated and administered by the institution (USB) which receives them on its proper accounts. Individual investigators who participate in the sponsored project(s) are not directly compensated by the sponsor but may receive salary or other support from the institution to support their effort on the project(s). Funding Information: This study was supported by the Foundation Fighting Blindness Clinical Research Institute. Rupert W. Strauss is supported by the Austrian Science Fund (FWF; Project number: J 3383-B23) and Foundation Fighting Blindness Clinical Research Institute. Etienne M. Schonbach is supported by the German National Academy of Sciences Leopoldina, Grant Number LPDS 2015-14. Michel Michaelides is supported by an FFB Career Development Award, the Macular Society, Fight for Sight, Retinitis Pigmentosa Fighting Blindness, and the National Institute for Health Research Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology Funding Information: This study was supported by the Foundation Fighting Blindness Clinical Research Institute. Rupert W. Strauss is supported by the Austrian Science Fund (FWF; Project number: J 3383-B23) and Foundation Fighting Blindness Clinical Research Institute. Eti-enne M. Sch{\"o}nbach is supported by the German National Academy of Sciences Leopoldina, Grant Number LPDS 2015-14. Michel Michaelides is supported by an FFB Career Development Award, the Macular Society, Fight for Sight, Retinitis Pigmentosa Fighting Blindness, and the National Institute for Health Research Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology. Publisher Copyright: {\textcopyright} 2018 S. Karger AG, Basel. All rights reserved.",
year = "2019",
month = jan,
day = "1",
doi = "10.1159/000488711",
language = "English (US)",
volume = "61",
pages = "36--43",
journal = "Ophthalmic Research",
issn = "0030-3747",
publisher = "S. Karger AG",
number = "1",
}