A Natural History of the Progression of Stargardt Disease: Retrospective and Prospective Studies (ProgSTAR)
|ClinicalTrials.gov Identifier: NCT01977846|
Recruitment Status : Completed
First Posted : November 7, 2013
Last Update Posted : October 23, 2017
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.
|Condition or disease|
|Study Type :||Observational|
|Actual Enrollment :||259 participants|
|Official Title:||Natural History of Progression of Atrophy Secondary to Stargardt Disease: Retrospective, and Prospective Longitudinal Observational Study Incl. Ancillary SMART Study- Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease|
|Study Start Date :||August 2013|
|Actual Primary Completion Date :||February 2017|
|Actual Study Completion Date :||February 2017|
- To assess the yearly rate of progression of STGD using the growth of atrophic lesions as measured by fundus autofluorescence (FAF) imaging. [ Time Frame: 4-7 years ]From 2 to 5 years of retrospective fundus autofluorescence imaging linked with 2 years of standardized prospective fundus autofluorescence imaging will be collected.
- To assess the yearly rate of progression of STGD using spectral-domain optical coherence tomography (sd-OCT) to measure the rates of retinal thinning and the loss of photoreceptors [ Time Frame: 4-7 years ]From 2 to 5 years of retrospective spectral-domain optical coherence tomography imaging linked with 2 years of standardized prospective spectral-domain optical coherence tomography imaging will be collected
- To assess the yearly rate of loss of retinal sensitivity as measured by microperimetry. [ Time Frame: 4 to 7 years ]Up to 2 to 5 years of retrospective microperimetry imaging linked with 2 years of standardized prospective microperimetry imaging will be collected.
- To assess the yearly rate of visual acuity changes as measured by best corrected visual acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol [ Time Frame: 4-7 years ]From 2 to 5 years of retrospective visual acuity measurements will be linked with 2 years of standardized prospective visual acuity measurements will be collected
- To correlate the presence and progression of morphological abnormalities in FAF and sd-OCT images with visual function as measured by microperimetry and visual acuity. [ Time Frame: 4-7 years ]From 2 to 5 years of retrospective spectral-domain optical coherence tomography and fundus autofluorescence imaging linked with 2 years of standardized prospective spectral-domain optical coherence tomography and fundus autofluorescence imaging will be analyzed for correlation
- To perform exploratory analysis of factors associated with STGD progression, such as participant's use of vitamin A supplementation, and mutations in the ABCA4 gene. [ Time Frame: 4-7 years ]From 2 to 5 years of retrospectively collected information linked with 2 years of standardized prospectively collected information will be collected and assessed.
- To assess the yearly rate of loss of retinal sensitivity as measured by scotopic microperimetry. [ Time Frame: 2 years ]Up to 2 years of standardized prospective microperimetry imaging, gathered under scotopic conditions, will be collected in a subset of prospective patients (SMART Study).
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01977846
|United States, Maryland|
|Greater Baltimore Medical Center|
|Baltimore, Maryland, United States, 21204|
|Wilmer Eye Institute, Johns Hopkins University|
|Baltimore, Maryland, United States, 21287|
|United States, Ohio|
|Cole Eye Institute, Cleveland Clinic|
|Cleveland, Ohio, United States, 44195|
|United States, Pennsylvania|
|Scheie Eye Institute, University of Pennsylvania|
|Philadelphia, Pennsylvania, United States, 19104|
|United States, Texas|
|Retina Foundation of the Southwest|
|Dallas, Texas, United States, 75231|
|United States, Utah|
|Moran Eye Center, University of Utah|
|Salt Lake City, Utah, United States, 84132|
|Institut de la Vision|
|Paris, France, 75012|
|Center for Ophthalmic Research, University of Teubingen|
|Tübingen, Germany, 72076|
|Moorfields Eye Hospital|
|London, United Kingdom, EC1V 2PD|
|Study Chair:||Hendrik Scholl, MD||Wilmer Eye Institute at the Johns Hopkins University|