Novel Quantification Methods for Fluorescence to Detect Progression in Stargardt Disease
| Status: | Completed |
|---|---|
| Conditions: | Ocular |
| Therapuetic Areas: | Ophthalmology |
| Healthy: | No |
| Age Range: | 5 - 18 |
| Updated: | 4/2/2016 |
| Start Date: | September 2012 |
| End Date: | February 2014 |
| Contact: | Jillian T. Huang, MS, MPH |
| Email: | tietjenj@umich.edu |
| Phone: | 734-936-3573 |
Novel Quantification Methods for Fundus Flavoprotein Fluorescence and Lipofuscin Fluorescence to Detect Progression in Stargardt Disease
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.
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.
This study will evaluate whether more sophisticated testing and analytic methodologies,
including fundus autofluorescence (FAF) and a novel non-invasive method to measure retinal
flavoprotein fluorescence (FPF) may be used to better predict Stargardt macular dystrophy
progression and monitor treatment effects than conventional modalities such visual acuity
and visual field. This method involves the use of novel statistical methods to assess the
heterogeneity of fundus autofluorescence images.
Participants will complete 3 visits to the University of Michigan Kellogg Eye Center. Each
visit will take approximately 2.5 hours. The initial visit will include a routine clinical
eye examination, measurement of best-corrected visual acuity, indirect ophthalmoscopy,
microperimetry, frequency-domain optical coherence tomography, Goldmann visual fields,
fundus flavoprotein fluorescence (FPF) imaging, and fundus autofluorescence (FAF) and fundus
photography. Patients will return for evaluation at 6 and 12 months after their initial
visit to repeat testing and imaging.
including fundus autofluorescence (FAF) and a novel non-invasive method to measure retinal
flavoprotein fluorescence (FPF) may be used to better predict Stargardt macular dystrophy
progression and monitor treatment effects than conventional modalities such visual acuity
and visual field. This method involves the use of novel statistical methods to assess the
heterogeneity of fundus autofluorescence images.
Participants will complete 3 visits to the University of Michigan Kellogg Eye Center. Each
visit will take approximately 2.5 hours. The initial visit will include a routine clinical
eye examination, measurement of best-corrected visual acuity, indirect ophthalmoscopy,
microperimetry, frequency-domain optical coherence tomography, Goldmann visual fields,
fundus flavoprotein fluorescence (FPF) imaging, and fundus autofluorescence (FAF) and fundus
photography. Patients will return for evaluation at 6 and 12 months after their initial
visit to repeat testing and imaging.
Inclusion Criteria:
- 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
Exclusion Criteria:
- Limited central vision, defined as visual acuity worse than 20/100
- A diagnosis of any other retinal degenerative disease
We found this trial at
1
site
Click here to add this to my saved trials
