NCT01977846

A Natural History of the Progression of Stargardt Disease: Retrospective and Prospective Studies

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

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 259 (actual)
• Sponsor: Foundation Fighting Blindness · Academic / Other
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Stargardt Disease

Timeline

• Start date: 2013-08-01
• Primary completion: 2017-02-01
• Completion: 2017-02-01
• First posted: 2013-11-07
• Last updated: 2019-11-01
• Results posted: 2019-11-01

Locations

9 sites across 4 countries: United States, France, Germany, United Kingdom