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Novel Quantification Methods for Fluorescence to Detect Progression in Stargardt Disease

This study is currently recruiting participants.
Verified September 2012 by University of Michigan
Sponsor:
Collaborator:
Midwest Eye Banks
Information provided by (Responsible Party):
K. Thiran Jayasundera, University of Michigan
ClinicalTrials.gov Identifier:
NCT01676766
First received: August 23, 2012
Last updated: September 19, 2012
Last verified: September 2012
History of Changes
  Purpose

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.


Condition
Stargardt Disease

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Novel Quantification Methods for Fundus Flavoprotein Fluorescence and Lipofuscin Fluorescence to Detect Progression in Stargardt Disease

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by University of Michigan:

Primary Outcome Measures:
  • Change from baseline in pixel intensity quantification of fundus autofluorescence at 6 months [ Time Frame: 0 months, 6 months ] [ Designated as safety issue: No ]
  • Change from baseline in pixel intensity quantification of flavoprotein autofluorescence at 6 months [ Time Frame: 0 months, 6 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change from baseline in pixel intensity quantification of fundus autofluorescence at 12 months [ Time Frame: 0 months, 12 months ] [ Designated as safety issue: No ]
  • Change from baseline in pixel intensity quantification of flavoprotein autofluorescence at 12 months [ Time Frame: 0 months, 12 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 25
Study Start Date: September 2012
Estimated Study Completion Date: February 2014
Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   5 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

We will perform an observational clinical study of 25 pediatric patients with Stargardt Disease recruited from the retinal degeneration clinic at the University of Michigan who have two mutations in ABCA4.

Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01676766

Contacts
Contact: Jillian T. Huang, MS, MPH 734-936-3573 tietjenj@umich.edu

Locations
United States, Michigan
Kellogg Eye Center Recruiting
Ann Arbor, Michigan, United States, 48105
Contact: Jillian T. Huang, MS, MPH     734-936-3573        
Sponsors and Collaborators
University of Michigan
Midwest Eye Banks
Investigators
Principal Investigator: K. Thiran Jayasundera, MD University of Michigan Kellogg Eye Center
  More Information

Publications:

Responsible Party: K. Thiran Jayasundera, Assistant Professor, Retina and Uveitis, University of Michigan
ClinicalTrials.gov Identifier: NCT01676766     History of Changes
Other Study ID Numbers: HUM 64388
Study First Received: August 23, 2012
Last Updated: September 19, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by University of Michigan:
Stargardt Disease
Macular degeneration
Retinal dystrophy

ClinicalTrials.gov processed this record on May 19, 2013