A Natural History of the Progression of Stargardt Disease: Retrospective and Prospective Studies (ProgSTAR)
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|ClinicalTrials.gov Identifier: NCT01977846|
Recruitment Status : Completed
First Posted : November 7, 2013
Results First Posted : November 1, 2019
Last Update Posted : November 1, 2019
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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|
- Yearly Progression Rate of Atrophic Lesions Using Fundus Autofluorescence (FAF) Images [ Time Frame: 2-12 years ]Yearly increase in area of decreased auto-fluorescence (DAF) which is defined as the sum of definite and questionable decreased auto-fluorescence
- Yearly Rate of Loss of Retinal Sensitivity as Measured by Scotopic Microperimetry (MP) [ Time Frame: 2 years ]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)
- Yearly Rate of Visual Acuity Loss [ Time Frame: 2-12 years ]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
- Difference in the Rate of Retinal Sensitivity Change Per Year Between Photopic and Scotopic Micro-perimetry Testing [ Time Frame: 2 years ]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
- Yearly Rate of Loss of Overall Retinal Thickness [ Time Frame: Participants followed at Baseline, 6 months, 12 months and 24 months ]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.
- Yearly Rate of Loss of Outer Ring Retinal Thickness [ Time Frame: Participants followed at Baseline, 6 months, 12 months and 24 months ]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.
- Yearly Rate of Loss of the Inner Ring Retinal Thickness [ Time Frame: Participants followed at Baseline, 6 months, 12 months and 24 months ]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.
- Yearly Rate of Loss of the Central Ring Retinal Thickness [ Time Frame: Participants followed at Baseline, 6 months, 12 months and 24 months ]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.
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|Ages Eligible for Study:||6 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
|Sampling Method:||Non-Probability Sample|
- Provide a signed informed consent form and authorization allowing the disclosure and use of protected health information.
- The designated primary study eye must have at least one well-demarcated area of atrophy as imaged by fundus autofluorescence with a minimum diameter of 300 microns and all lesions together must add to less than or equal to 12 mm2 (equivalent to no more than 5 disc areas in a least one eye) and a BCVA of 20 ETDRS letters (20/400 Snellen equivalent) or better.
- Two (2) pathogenic mutations confirmed present, in the ABCA4 gene. If only one ABCA4 allele contains a pathogenic mutation, the patient shall have a typical Stargardt phenotype, namely at least one eye must have flecks at the level of the retinal pigment epithelium typical for STGD.
- The primary study eye must have clear ocular media and adequate pupillary dilation to permit good quality fundus autofluorescence (FAF) and Spectral-Domain optical coherence tomography (sd-OCT) imaging in the opinion of the investigator.
- Be able to cooperate in performing the examinations.
- Be willing to undergo ocular examinations once every 6 months for up to 24 months.
- Be at least six years old.
- Both eyes can be included if inclusion criteria are fulfilled for both eyes.
- Ocular disease, such as choroidal neovascularization, glaucoma and diabetic retinopathy, in either eye that may confound assessment of the retina morphologically and functionally.
- Intraocular surgery in the primary study eye within 90 days prior to baseline visit.
- Current or previous participation in an interventional study to treat STGD such as gene therapy or stem cell therapy. Current participation in a drug trial or previous participation in a drug trial within six months before enrollment. The use of oral supplements of vitamins and minerals are permitted although the current use of Vitamin A supplementation shall be documented.
- The site Principal Investigator may declare any patient at their site ineligible to participate in the study for a sound medical reason prior to the patient's enrollment into the study.
- Any systemic disease with a limited survival prognosis (e.g. cancer, severe/unstable cardiovascular disease).
- Any condition that would interfere with the patient attending their regular follow-up visits every 6 months for up to 24 months, e.g. personality disorder, use of major tranquilizers such as Haldol or Phenothiazine, chronic alcoholism, Alzheimer's Disease or drug abuse.
- Evidence of significant uncontrolled concomitant diseases such as cardiovascular, neurological, pulmonary, renal, hepatic, endocrine or gastro-intestinal disorders.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|
Documents provided by Foundation Fighting Blindness:
Publications of Results:
|Responsible Party:||Foundation Fighting Blindness|
|Other Study ID Numbers:||
|First Posted:||November 7, 2013 Key Record Dates|
|Results First Posted:||November 1, 2019|
|Last Update Posted:||November 1, 2019|
|Last Verified:||July 2019|
Eye Diseases, Hereditary
Genetic Diseases, Inborn