ProgSTAR: A Prospective Observational Study of Stargardt Disease
Research type
Research Study
Full title
The Natural History of the Progression of Atrophy Secondary to Stargardt Disease: A Prospective Longitudinal Observational Study
IRAS ID
126226
Contact name
Michel Michaelides
Contact email
Sponsor organisation
Foundation Fighting Blindness Clinical Research Institute
Research summary
Stargardt Disease (STGD) is the most prevalent form of juvenile-onset macular dystrophy, with an estimated incidence of 10 – 12.5 per 100,000. However, it is still relatively rare and there is very limited information available about the natural course of the disease in larger numbers of STGD patients. The information currently available is predominantly based on individual reports of patient data collected from single centers. However, this limited data clearly points to a disease which is very heterogeneous with significant variation in the age of onset and rate of progression, with limited clear correlations between phenotype and genotype. These limitations highlight the urgent need for a (multi-center) study documenting the natural course of the disease. The information gathered from this proposed study should provide guidance and support to the identification of the most appropriate clinical outcome measures for anticipated clinical trials. It may also be used to identify and support the validation of surrogate clinical trial endpoints that are offered by recent technological advances in retinal imaging capabilities, such as fundus autofluorescence (FAF) and spectral-domain optical coherence tomography (sd-OCT).
REC name
London - Queen Square Research Ethics Committee
REC reference
13/LO/0886
Date of REC Opinion
23 Jul 2013
REC opinion
Further Information Favourable Opinion