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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

Tracking Information
First Submitted Date October 31, 2013
First Posted Date November 7, 2013
Results First Submitted Date May 30, 2018
Results First Posted Date November 1, 2019
Last Update Posted Date November 1, 2019
Study Start Date August 2013
Actual Primary Completion Date February 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 30, 2019)
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
Original Primary Outcome Measures
 (submitted: October 31, 2013)
To assess the yearly rate of progression of STGD using the growth of atrophic lesions as measured by fundus autofluorescence (FAF) imaging. [ Time Frame: 4-7 years ]
From 2 to 5 years of retrospective fundus autofluorescence imaging linked with 2 years of standardized prospective fundus autofluorescence imaging will be collected.
Change History
Current Secondary Outcome Measures
 (submitted: October 30, 2019)
  • 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)
  • 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
  • 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
  • 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.
  • 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.
  • 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.
  • 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.
Original Secondary Outcome Measures
 (submitted: October 31, 2013)
  • To assess the yearly rate of progression of STGD using spectral-domain optical coherence tomography (sd-OCT) to measure the rates of retinal thinning and the loss of photoreceptors [ Time Frame: 4-7 years ]
    From 2 to 5 years of retrospective spectral-domain optical coherence tomography imaging linked with 2 years of standardized prospective spectral-domain optical coherence tomography imaging will be collected
  • To assess the yearly rate of loss of retinal sensitivity as measured by microperimetry. [ Time Frame: 4 to 7 years ]
    Up to 2 to 5 years of retrospective microperimetry imaging linked with 2 years of standardized prospective microperimetry imaging will be collected.
  • To assess the yearly rate of visual acuity changes as measured by best corrected visual acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol [ Time Frame: 4-7 years ]
    From 2 to 5 years of retrospective visual acuity measurements will be linked with 2 years of standardized prospective visual acuity measurements will be collected
  • To correlate the presence and progression of morphological abnormalities in FAF and sd-OCT images with visual function as measured by microperimetry and visual acuity. [ Time Frame: 4-7 years ]
    From 2 to 5 years of retrospective spectral-domain optical coherence tomography and fundus autofluorescence imaging linked with 2 years of standardized prospective spectral-domain optical coherence tomography and fundus autofluorescence imaging will be analyzed for correlation
  • To perform exploratory analysis of factors associated with STGD progression, such as participant's use of vitamin A supplementation, and mutations in the ABCA4 gene. [ Time Frame: 4-7 years ]
    From 2 to 5 years of retrospectively collected information linked with 2 years of standardized prospectively collected information will be collected and assessed.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Natural History of the Progression of Stargardt Disease: Retrospective and Prospective Studies
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
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.

Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition Stargardt Disease
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: March 15, 2017)
259
Original Estimated Enrollment
 (submitted: October 31, 2013)
200
Actual Study Completion Date February 2017
Actual Primary Completion Date February 2017   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries France,   Germany,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT01977846
Other Study ID Numbers FFBCRI-PROGSTAR-01/02
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Foundation Fighting Blindness
Study Sponsor Foundation Fighting Blindness
Collaborators United States Department of Defense
Investigators
Study Chair: Hendrik Scholl, MD Wilmer Eye Institute at the Johns Hopkins University
PRS Account Foundation Fighting Blindness
Verification Date July 2019