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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01977846
Recruitment Status : Completed
First Posted : November 7, 2013
Results First Posted : November 1, 2019
Last Update Posted : November 1, 2019
Sponsor:
Collaborator:
United States Department of Defense
Information provided by (Responsible Party):
Foundation Fighting Blindness

Study Description
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Brief 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.


Condition or disease
Stargardt Disease

Study Design
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Study Type : Observational
Actual Enrollment : 259 participants
Observational Model: Case-Only
Time Perspective: Other
Official Title: 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
Study Start Date : August 2013
Actual Primary Completion Date : February 2017
Actual Study Completion Date : February 2017

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. Yearly Progression Rate of Atrophic Lesions Using Fundus Autofluorescence (FAF) Images [ Time Frame: 2-12 years ]
    Yearly increase in area of decreased auto-fluorescence (DAF) which is defined as the sum of definite and questionable decreased auto-fluorescence


Secondary Outcome Measures :
  1. Yearly Rate of Loss of Retinal Sensitivity as Measured by Scotopic Microperimetry (MP) [ Time Frame: 2 years ]
    The yearly rate of change in retinal sensitivity. Sensitivity tested with a Nidek MP-1 machine using a modified Humphrey 10-2 grid. The sensitivity was the average sensitivity from a 68-points test pattern (Prospective cohort only)

  2. Yearly Rate of Visual Acuity Loss [ Time Frame: 2-12 years ]
    Yearly change of visual acuity. Visual acuity measures of best-corrected or presenting VA extracted from medical record charts. Prospective cohort is best-corrected visual acuity using Early-Treatment Diabetic Retinopathy study methods

  3. Difference in the Rate of Retinal Sensitivity Change Per Year Between Photopic and Scotopic Micro-perimetry Testing [ Time Frame: 2 years ]
    Difference in the yearly rate of change in retinal sensitivity under photopic and scotopic conditions. Sensitivity tested with a Nidek MP-1. Scotopic sensitivity was obtained using a 40 points test pattern, and photopic sensitivity was obtained using a 68 points test pattern in a subset of Prospective cohort patients

  4. Yearly Rate of Loss of Overall Retinal Thickness [ Time Frame: Participants followed at Baseline, 6 months, 12 months and 24 months ]
    Yearly decrease of overall retinal thickness using spectral domain optical coherence tomography (SD-OCT) scans from a 20° x 20° scan area centered on the fovea. Data are only available for the Prospective cohort.

  5. Yearly Rate of Loss of Outer Ring Retinal Thickness [ Time Frame: Participants followed at Baseline, 6 months, 12 months and 24 months ]
    Yearly decrease of outer ring retinal thickness using SD-OCT scans from a 20° x 20° scan area centered on the fovea. Outer ring defined as ETDRS fields 1-4.

  6. Yearly Rate of Loss of the Inner Ring Retinal Thickness [ Time Frame: Participants followed at Baseline, 6 months, 12 months and 24 months ]
    Yearly decrease of the inner ring retinal thickness using SD-OCT scans from a 20° x 20° scan area centered on the fovea. Inner ring defined as ETDRS fields 5-8. Data are only available for the Prospective cohort.

  7. Yearly Rate of Loss of the Central Ring Retinal Thickness [ Time Frame: Participants followed at Baseline, 6 months, 12 months and 24 months ]
    Yearly decrease of the central ring retinal thickness using SD-OCT scans from a 20° x 20° scan area centered on the fovea. Central area defined as ETDRS fields 9. Data are only available for the Prospective cohort.


Eligibility Criteria
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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The study population shall consist of up to 250 Stargardt disease patients (minimum of 150 patients) recruited at up to 14 clinical centers across the US and Europe. Must be at least 6 years old, able to cooperate in performing the examinations and be willing to attend regular 6 month follow-up visits for up to 24 months. Must present with atrophic lesions secondary to STGD and previously genotyped (at least 2 confirmed pathogenic mutations in the ABCA4 gene). If only 1 ABCA4 allele contains a pathogenic mutation, then the patient needs typical phenotype, i.e. at least one eye must have flecks at the level of the retinal pigment epithelium typical for STGD. Best-corrected visual acuity (BCVA) must be 20 ETDRS letters (20/400 Snellen equivalent) or better.
Criteria

Inclusion Criteria:

  • Provide a signed informed consent form and authorization allowing the disclosure and use of protected health information.
  • The designated primary study eye must have at least one well-demarcated area of atrophy as imaged by fundus autofluorescence with a minimum diameter of 300 microns and all lesions together must add to less than or equal to 12 mm2 (equivalent to no more than 5 disc areas in a least one eye) and a BCVA of 20 ETDRS letters (20/400 Snellen equivalent) or better.
  • Two (2) pathogenic mutations confirmed present, in the ABCA4 gene. If only one ABCA4 allele contains a pathogenic mutation, the patient shall have a typical Stargardt phenotype, namely at least one eye must have flecks at the level of the retinal pigment epithelium typical for STGD.
  • The primary study eye must have clear ocular media and adequate pupillary dilation to permit good quality fundus autofluorescence (FAF) and Spectral-Domain optical coherence tomography (sd-OCT) imaging in the opinion of the investigator.
  • Be able to cooperate in performing the examinations.
  • Be willing to undergo ocular examinations once every 6 months for up to 24 months.
  • Be at least six years old.
  • Both eyes can be included if inclusion criteria are fulfilled for both eyes.

Exclusion Criteria:

  • Ocular disease, such as choroidal neovascularization, glaucoma and diabetic retinopathy, in either eye that may confound assessment of the retina morphologically and functionally.
  • Intraocular surgery in the primary study eye within 90 days prior to baseline visit.
  • Current or previous participation in an interventional study to treat STGD such as gene therapy or stem cell therapy. Current participation in a drug trial or previous participation in a drug trial within six months before enrollment. The use of oral supplements of vitamins and minerals are permitted although the current use of Vitamin A supplementation shall be documented.
  • The site Principal Investigator may declare any patient at their site ineligible to participate in the study for a sound medical reason prior to the patient's enrollment into the study.
  • Any systemic disease with a limited survival prognosis (e.g. cancer, severe/unstable cardiovascular disease).
  • Any condition that would interfere with the patient attending their regular follow-up visits every 6 months for up to 24 months, e.g. personality disorder, use of major tranquilizers such as Haldol or Phenothiazine, chronic alcoholism, Alzheimer's Disease or drug abuse.
  • Evidence of significant uncontrolled concomitant diseases such as cardiovascular, neurological, pulmonary, renal, hepatic, endocrine or gastro-intestinal disorders.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01977846


Locations
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United States, Maryland
Greater Baltimore Medical Center
Baltimore, Maryland, United States, 21204
Wilmer Eye Institute, Johns Hopkins University
Baltimore, Maryland, United States, 21287
United States, Ohio
Cole Eye Institute, Cleveland Clinic
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
Scheie Eye Institute, University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
United States, Utah
Moran Eye Center, University of Utah
Salt Lake City, Utah, United States, 84132
France
Institut de la Vision
Paris, France, 75012
Germany
Center for Ophthalmic Research, University of Teubingen
Tübingen, Germany, 72076
United Kingdom
Moorfields Eye Hospital
London, United Kingdom, EC1V 2PD
Sponsors and Collaborators
Foundation Fighting Blindness
United States Department of Defense
Investigators
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Study Chair: Hendrik Scholl, MD Wilmer Eye Institute at the Johns Hopkins University
  Study Documents (Full-Text)

Documents provided by Foundation Fighting Blindness:
Study Protocol  [PDF] March 11, 2013
Statistical Analysis Plan  [PDF] May 19, 2018

More Information
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Additional Information:

Publications of Results:

Other Publications:

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Responsible Party: Foundation Fighting Blindness
ClinicalTrials.gov Identifier: NCT01977846    
Other Study ID Numbers: FFBCRI-PROGSTAR-01/02
First Posted: November 7, 2013    Key Record Dates
Results First Posted: November 1, 2019
Last Update Posted: November 1, 2019
Last Verified: July 2019
Keywords provided by Foundation Fighting Blindness:
genetic testing
ABCA4
Stargardt
retina
retinal degeneration
Additional relevant MeSH terms:
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Stargardt Disease
Macular Degeneration
Eye Diseases, Hereditary
Eye Diseases
 Retinal Degeneration
Retinal Diseases
Genetic Diseases, Inborn