Stargardt disease is the most prevalent form of juvenile-onset macular dystrophy, predominantly due to gene mutations in an autosomal recessive inherited pattern. It is currently incurable as there are no therapeutic treatments for Stargardt Disease approved by the Food and Drug Administration. The Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) is sponsored by the Foundation Fighting Blindness.
Principal Investigator: Dr. Xiangrong Kong
The objective of the ProgStar studies is to determine the natural progression of Stargardt disease in a large population of children and adults. The studies will assist with determining clinically significant efficacy measures for future randomized controlled trials.
ProgStar is comprised of retrospective and prospective observational studies currently conducted at nine clinical centers across the U.S. and Europe.
In the retrospective study, clinical examination findings and images of 250 Stargardt disease patients collected between 2008 or earlier and 2015 will be evaluated. The prospective study consists of a 24-month observational period of 250 Stargardt disease participants meeting specific eligibility criteria with one clinical visit every six months.
The outcomes of interest for the ProgStar studies are measured via imaging and psychophysical testing.
- To assess the yearly rate of progression of Stargardt Disease using the growth or the development of atrophic lesions as measured by funds autofluorescence (FAF) imaging
- To assess the progression of Stargardt Disease using spectral domain optical coherence tomography (SD-OCT) to measure the rates of retinal thinning and the loss of photoreceptors
- To assess loss of retinal sensitivity as measured by microperimetry (MP)
- To assess change in best corrected visual acuity (BCVA)
- To correlate the presence and progression of morphological abnormalities in FAF and SD-OCT images with visual function as measured by MP and BCVA
- To perform exploratory analysis of factors associated with Stargardt Disease progression, such as participant’s use of vitamin A supplementation and mutations in the ABCA4 gene