An observational study in Stargardt Disease, sponsored by Foundation Fighting Blindness. Completed at 9 sites in 4 countries. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2019-11-01.
Sponsored by Foundation Fighting Blindness · Observational
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.
66 studies on the registry are indexed under Stargardt Disease; 25 are open to participants now.
This study's enrollment of 259 is above the median of 50 across 23 observational studies indexed under Stargardt Disease.
Browse Stargardt Disease studies →Foundation Fighting Blindness is the lead sponsor of 3 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Yearly Progression Rate of Atrophic Lesions Using Fundus Autofluorescence (FAF) Images
Yearly increase in area of decreased auto-fluorescence (DAF) which is defined as the sum of definite and questionable decreased auto-fluorescence
Time frame: 2-12 years
Yearly Rate of Loss of Retinal Sensitivity as Measured by Scotopic Microperimetry (MP)
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)
Time frame: 2 years
Yearly Rate of Visual Acuity Loss
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
Time frame: 2-12 years
Difference in the Rate of Retinal Sensitivity Change Per Year Between Photopic and Scotopic Micro-perimetry Testing
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
Time frame: 2 years
Yearly Rate of Loss of Overall Retinal Thickness
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.
Time frame: Participants followed at Baseline, 6 months, 12 months and 24 months
Yearly Rate of Loss of Outer Ring Retinal Thickness
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.
Time frame: Participants followed at Baseline, 6 months, 12 months and 24 months
Yearly Rate of Loss of the Inner Ring Retinal Thickness
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.
Time frame: Participants followed at Baseline, 6 months, 12 months and 24 months
Yearly Rate of Loss of the Central Ring Retinal Thickness
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.
Time frame: Participants followed at Baseline, 6 months, 12 months and 24 months
| Milestone | Retrospective Cohort | Prospective Cohort |
|---|---|---|
| Started | 251 | 0 |
| Completed | 251 | 0 |
| Not completed | 0 | 0 |
| Milestone | Retrospective Cohort | Prospective Cohort |
|---|---|---|
| Started | 0 | 259 |
| Completed | 0 | 230 |
| Not completed | 0 | 29 |
| Withdrew: Death | 0 | 1 |
| Withdrew: Lost to follow-up | 0 | 8 |
| Withdrew: Withdrawal by subject | 0 | 13 |
| Withdrew: Not available at time of visit | 0 | 5 |
| Withdrew: No show | 0 | 1 |
| Withdrew: Protocol violation | 0 | 1 |
Yearly increase in area of decreased auto-fluorescence (DAF) which is defined as the sum of definite and questionable decreased auto-fluorescence
| mm^2/year | Retrospective Cohort | Prospective Cohort |
|---|---|---|
| Yearly Progression Rate of Atrophic Lesions Using Fundus Autofluorescence (FAF) Images | 0.35 (0.28 to 0.43) | 0.64 (0.57 to 0.71) |
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)
| dB/year | Retrospective Cohort | Prospective Cohort |
|---|---|---|
| Yearly Rate of Loss of Retinal Sensitivity as Measured by Scotopic Microperimetry (MP) | — | -0.76 (-0.87 to -0.66) |
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
| logMAR/year | Retrospective Cohort | Prospective Cohort |
|---|---|---|
| Yearly Rate of Visual Acuity Loss | 0.030 (0.026 to 0.043) | 0.011 (0.004 to 0.018) |
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
| dB/year | Retrospective Cohort | Prospective Cohort |
|---|---|---|
| Difference in the Rate of Retinal Sensitivity Change Per Year Between Photopic and Scotopic Micro-perimetry Testing | — | -0.78 (-1.16 to -0.41) |
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.
| microns/year | Prospective Cohort |
|---|---|
| Yearly Rate of Loss of Overall Retinal Thickness | -2.85 (-3.19 to -2.52) |
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.
| microns/year | Prospective Cohort |
|---|---|
| Yearly Rate of Loss of Outer Ring Retinal Thickness | -2.84 (-3.19 to -2.50) |
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.
| microns/year | Prospective Cohort |
|---|---|
| Yearly Rate of Loss of the Inner Ring Retinal Thickness | -3.20 (-3.68 to -2.72) |
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.
| microns/year | Prospective Cohort |
|---|---|
| Yearly Rate of Loss of the Central Ring Retinal Thickness | -2.24 (-3.06 to -1.42) |
Collected over Adverse events were not collected in this natural history study. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Retrospective Cohort | 0/251 (0%) | — | — |
| Prospective Cohort | 1/259 (0.4%) | — | — |
In Retrospective cohort, 251 participants contributed 433 eyes. In Prospecitive cohort, 259 participants contributed 489 eyes.
| Age, Categorical(Participants) | Retrospective Cohort | Prospective Cohort | Total |
|---|---|---|---|
| <=18 years | 69 | 51 | 120 |
| Between 18 and 65 years | 179 | 201 | 380 |
| >=65 years | 3 | 7 | 10 |
| Sex: Female, Male(Participants) | Retrospective Cohort | Prospective Cohort | Total |
|---|---|---|---|
| Female | 149 | 141 | 290 |
| Male | 102 | 118 | 220 |
| Race/Ethnicity, Customized(Participants) | Retrospective Cohort | Prospective Cohort | Total |
|---|---|---|---|
| Race — White/Middle Eastern | 174 | 222 | 396 |
| Race — Black or African American | 14 | 20 | 34 |
| Race — Asian/Indian | 10 | 10 | 20 |
| Race — Other | 5 | 1 | 6 |
| Race — More than one race | 2 | 2 | 4 |
| Race — Don't know/missing | 46 | 4 | 50 |
| Region of Enrollment(participants) | Retrospective Cohort | Prospective Cohort | Total |
|---|---|---|---|
| United States | 90 | 137 | 227 |
| United Kingdom | 79 | 30 | 109 |
| France | 49 | 48 | 97 |
| Germany | 33 | 44 | 77 |
PDFs hosted by the registry, ClinicalTrials.gov.
The record gives a start of Aug 2013, a primary completion of Feb 2017 and a completion of Feb 2017. It does not say whether these dates are actual or estimated.
Results were first posted in Nov 2019, 2 years 9 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jul 2019. You cannot join it, but the record below documents what was studied.
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Foundation Fighting Blindness