Stargardt disease is currently an incurable and untreatable macular dystrophy that causes severe visual loss in children and young adults, thereby causing enormous morbidity with economic, psychological, emotional, and social implications. There are no FDA approved therapeutic treatments for this disease. Therefore, the objective of this study is to collect natural history data from a large population of children and adults in order to evaluate possible efficacy measures for planned clinical trials. Participants will be recruited from each Investigator's own patient population as the study requires the availability of both multiyear retrospective data, as well as ongoing prospectively collected data. A concurrent ancillary study (SMART study) is also being conducted with a subset of the prospective study patients during their regular ProgSTAR study visits to expand the collection of retinal images to include microperimetry measurements gathered under scotopic (low light) conditions.
Study Type
OBSERVATIONAL
Enrollment
259
Greater Baltimore Medical Center
Baltimore, Maryland, United States
Wilmer Eye Institute, Johns Hopkins University
Baltimore, Maryland, United States
Cole Eye Institute, Cleveland Clinic
Cleveland, Ohio, United States
Scheie Eye Institute, University of Pennsylvania
Philadelphia, Pennsylvania, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
Moran Eye Center, University of Utah
Salt Lake City, Utah, United States
Institut de la Vision
Paris, France
Center for Ophthalmic Research, University of Teubingen
Tübingen, Germany
Moorfields Eye Hospital
London, United Kingdom
Yearly Progression Rate of Atrophic Lesions Using Fundus Autofluorescence (FAF) Images
Yearly increase in area of decreased auto-fluorescence (DAF) which is defined as the sum of definite and questionable decreased auto-fluorescence
Time frame: 2-12 years
Yearly Rate of Loss of Retinal Sensitivity as Measured by Scotopic Microperimetry (MP)
The yearly rate of change in retinal sensitivity. Sensitivity tested with a Nidek MP-1 machine using a modified Humphrey 10-2 grid. The sensitivity was the average sensitivity from a 68-points test pattern (Prospective cohort only)
Time frame: 2 years
Yearly Rate of Visual Acuity Loss
Yearly change of visual acuity. Visual acuity measures of best-corrected or presenting VA extracted from medical record charts. Prospective cohort is best-corrected visual acuity using Early-Treatment Diabetic Retinopathy study methods
Time frame: 2-12 years
Difference in the Rate of Retinal Sensitivity Change Per Year Between Photopic and Scotopic Micro-perimetry Testing
Difference in the yearly rate of change in retinal sensitivity under photopic and scotopic conditions. Sensitivity tested with a Nidek MP-1. Scotopic sensitivity was obtained using a 40 points test pattern, and photopic sensitivity was obtained using a 68 points test pattern in a subset of Prospective cohort patients
Time frame: 2 years
Yearly Rate of Loss of Overall Retinal Thickness
Yearly decrease of overall retinal thickness using spectral domain optical coherence tomography (SD-OCT) scans from a 20° x 20° scan area centered on the fovea. Data are only available for the Prospective cohort.
Time frame: Participants followed at Baseline, 6 months, 12 months and 24 months
Yearly Rate of Loss of Outer Ring Retinal Thickness
Yearly decrease of outer ring retinal thickness using SD-OCT scans from a 20° x 20° scan area centered on the fovea. Outer ring defined as ETDRS fields 1-4.
Time frame: Participants followed at Baseline, 6 months, 12 months and 24 months
Yearly Rate of Loss of the Inner Ring Retinal Thickness
Yearly decrease of the inner ring retinal thickness using SD-OCT scans from a 20° x 20° scan area centered on the fovea. Inner ring defined as ETDRS fields 5-8. Data are only available for the Prospective cohort.
Time frame: Participants followed at Baseline, 6 months, 12 months and 24 months
Yearly Rate of Loss of the Central Ring Retinal Thickness
Yearly decrease of the central ring retinal thickness using SD-OCT scans from a 20° x 20° scan area centered on the fovea. Central area defined as ETDRS fields 9. Data are only available for the Prospective cohort.
Time frame: Participants followed at Baseline, 6 months, 12 months and 24 months
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