Sponsored by the Foundation Fighting Blindness, the Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) studies are comprised of retrospective and prospective longitudinal observational studies.
- ProgStar study (STGD1; OMIM# 248200 [ABCA4]): Two retrospective and prospective cohort studies to evaluate the natural history Stargardt disease in patients with variants in the ABCA4 gene. Read more about this study below on this page.
- ProgStar2: This study is not existing, because the suspected mutation causing Stargardt Disease Type 2 (STGD2) could not be confirmed.
- ProgStar3: An ultra-rare variant of Stargardt disease (OMIM# 600110 [ELOV4]) for which not enough patients could be recruited, therefore no study under this name is existing.
- ProgStar4: The Natural History of the Progression of Atrophy Secondary to Stargardt Disease Type 4 (STGD4; OMIM # 603786 [PROM1]): A Prospective Longitudinal Observational Study of STGD4, a PROM1-Related Macular Dystrophy. Read more about this study in the specific ProgStar4 section of this website.
- The SMART study, an ancillary study of the ProgStar prospective study was conducted with a subset of the prospective STGT1 study patients during their regular ProgStar study visits. It exclusively evaluated the involvement and progression of rod photoreceptor damage in STGD with scotopic microperimetry. Read more about this study in the specific SMART study section of this website.
More about the ProgStar (STGT1) study:
Three hundred sixty-five unique patients aged 6 years and older at baseline harboring disease-causing variants in the ABCA4 gene and with specified ocular lesions were enrolled from 9 centers in the United States and Europe.
All published study reports can be found in section ProgStar study reports on this website.
In the retrospective ProgStar (STGT1) study, patients contributed medical record data from at least 2 and up to 4 visits for at least 1 examination modality: fundus autofluorescence (FAF), spectral-domain (SD) optical coherence tomography (SD OCT), and/or microperimetry (MP). The total observational period was at least 2 years and up to 5 years between single visits. Demographic and visual acuity (VA) data also were obtained.
In the prospective ProgStar (STGT1) study, eligible patients were examined at baseline using a standard protocol, with 6-month follow-up visits planned for a 2-year period for serial Early Treatment Diabetic Retinopathy Study (ETDRS) best-corrected VA, SD OCT, FAF, and MP.
The retrospective study includes clinical examination findings and images collected between 2008 (and earlier) and 2014 and progression was retrospectively evaluated. The prospective study consists of a 24 month observational period from 2013/2014 for two years, with one visit every six months.
The enrollment of a large population of children and adults with STGD assisted in evaluating efficacy measures for future clinical trials. The outcomes of interest were measured via imaging (i.e., spectral-domain optical coherence tomography, fundus autofluorescence) and psychophysical testing (i.e., visual acuity, microperimetry).
Images were evaluated by a central Reading Center at the Doheny Eye Institute, Los Angeles, California. Data Coordinating Center was located within the Dana Center for Preventive Ophthalmology, Johns Hopkins University School of Medicine, Baltimore, Maryland.
OBJECTIVE
The objective of the ProgStar studies is to determine the progression of disease in a large population of children and adults over at least a two year period, with a variety of potential measures.
PRIMARY AIM
- To assess the yearly rate of progression of STGD using the growth or the development of atrophic lesions as measured by FAF imaging
SECONDARY AIMS
- 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
- To assess the yearly rate of loss of retinal sensitivity as measured by microperimetry (MP)
- To assess the yearly rate of BCVA changes; in the prospective study, measurements will be based on measurements the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol
- To correlate the presence and progression of morphological abnormalities in FAF and SD-OCT images with visual function as measured by MP and VA
- 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
The study was sponsored by the Foundation Fighting Blindness, an organization dedicated to fund research that may lead to prevention, treatments, and cures for retinal degenerative diseases including STGD.