Novel Quantification Methods for Fluorescence to Detect Progression in Stargardt Disease

Stopped early

Conditions studied: Stargardt Disease

In brief

The purpose of this study is to utilize flavoprotein fluorescence and fundus autofluorescence to detect progression of Stargardt macular dystrophy in a pediatric population over the course of a year with the hope of aiding future therapeutic risk-benefit decisions and assessment of outcomes. Stargardt macular dystrophy is the most common of the juvenile-onset macular dystrophies. Despite determination of ABCA4 as the causative gene, clinicians have been challenged by variability in clinical phenotypes. Given the recent initiation of clinical trials to assess novel treatments (e.g. gene therapy), there is a need to identify patients with the worst prognosis. The investigators have observed that pediatric patients lose central visual function faster than their adult counterparts. Thus, they present an ideal cohort with which to determine the utility of novel modalities to detect early change. These include flavoprotein fluorescence, a new imaging technique for detecting mitochondrial dysfunction developed at the University of Michigan. Fundus autofluorescence (FAF) is another commonly utilized technique of evaluating hereditary eye diseases. The investigators have developed a novel means of quantifying FAF signatures that will allow documentation of severity as well as detection of progression.

Key facts

Study ID

NCT01676766

Run by

University of Michigan

People needed

2

Starts

2012-09-01

Expected to finish

2016-10-01

Last updated by the study team

2016-11-08

Who can join

Age: 5 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Between the age of 5 and 18 years old
• Clinical diagnosis of Stargardt Disease
• Molecular confirmation of Stargardt Disease (with 2 identified mutations in ABCA4)
• Visual acuity better than 20/100

You may not qualify if…

• Limited central vision, defined as visual acuity worse than 20/100
• A diagnosis of any other retinal degenerative disease

Where it is running

• Kellogg Eye Center — Ann Arbor, Michigan, United States

Full record on ClinicalTrials.gov

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