Stargardt DiseaseNCT01676766
Novel Quantification Methods for Fluorescence to Detect Progression in Stargardt Disease
An observational study of Stargardt Disease, run by University of Michigan at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 5 to 18 years
- Conditions the study is for
- Stargardt Disease
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2012to Oct 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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
Who cannot join
- Limited central vision, defined as visual acuity worse than 20/100
- A diagnosis of any other retinal degenerative disease
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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.
- Conditions
- Stargardt Disease
- Keywords
- Stargardt Disease, Macular degeneration, Retinal dystrophy
- Medical subject headings
- Stargardt Disease, Macular Degeneration, Retinal Dystrophies
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in pixel intensity quantification of fundus autofluorescence at 6 months
Measured: 0 months, 6 months
Change from baseline in pixel intensity quantification of flavoprotein autofluorescence at 6 months
Measured: 0 months, 6 months
Secondary outcomes (2)
Change from baseline in pixel intensity quantification of fundus autofluorescence at 12 months
Measured: 0 months, 12 months
Change from baseline in pixel intensity quantification of flavoprotein autofluorescence at 12 months
Measured: 0 months, 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2012 (estimated)
- Study startSep 2012
- Primary completionOct 2016
- Study completionOct 2016
Why it stopped: Flavoprotein fluorescence machine for the trial is not working so the trial was terminated. No study data was collected prior to termination.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Stargardt DiseaseStudies of the same condition, open and under way.
- More trials run by University of MichiganThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Michigan
- Sponsor type
- University, hospital or other
- Collaborators
- Midwest Eye Banks
- Responsible party
- Principal investigator, K. Thiran Jayasundera, University of Michigan
- Investigators
- K. Thiran Jayasundera, MD, Principal investigator, University of Michigan Kellogg Eye Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01676766
- Sponsor's protocol ID
- HUM 64388
- Status last verified
- Nov 2016
- First submitted
- 23 Aug 2012
- Last updated
- 8 Nov 2016
- Results posted
- No
- Data monitoring committee
- No
Publications
Elner SG, Elner VM, Field MG, Park S, Heckenlively JR, Petty HR. Retinal flavoprotein autofluorescence as a measure of retinal health. Trans Am Ophthalmol Soc. 2008;106:215-22; discussion 222-4.
PubMed 19277237Field MG, Elner VM, Park S, Hackel R, Heckenlively JR, Elner SG, Petty HR. Detection of retinal metabolic stress resulting from central serous retinopathy. Retina. 2009 Sep;29(8):1162-6. doi: 10.1097/IAE.0b013e3181a3b923.
PubMed 19491721Field MG, Elner VM, Puro DG, Feuerman JM, Musch DC, Pop-Busui R, Hackel R, Heckenlively JR, Petty HR. Rapid, noninvasive detection of diabetes-induced retinal metabolic stress. Arch Ophthalmol. 2008 Jul;126(7):934-8. doi: 10.1001/archopht.126.7.934.
PubMed 18625939Chen B, Tosha C, Gorin MB, Nusinowitz S. Analysis of autofluorescent retinal images and measurement of atrophic lesion growth in Stargardt disease. Exp Eye Res. 2010 Aug;91(2):143-52. doi: 10.1016/j.exer.2010.03.021. Epub 2010 Apr 14.
PubMed 20398653
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Novel Quantification Methods for Fundus Flavoprotein Fluorescence and Lipofuscin Fluorescence to Detect Progression in Stargardt Disease
- ClinicalTrials.gov ID
- NCT01676766
- Acronym / sponsor ID
- HUM 64388
- Conditions
- Stargardt Disease
- Keywords
- Stargardt Disease, Macular degeneration, Retinal dystrophy
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Flavoprotein fluorescence machine for the trial is not working so the trial was terminated. No study data was collected prior to termination.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- University of MichiganUniversity, hospital or other
- Collaborators
- Midwest Eye Banks
- Study start
- Sep 2012
- Primary completion
- Oct 2016 (actual)
- Study completion
- Oct 2016 (actual)
- First posted
- 31 Aug 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 5 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (2 listed)
- Limited central vision, defined as visual acuity worse than 20/100
- A diagnosis of any other retinal degenerative disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change from baseline in pixel intensity quantification of fundus autofluorescence at 6 months
Time frame: 0 months, 6 months
Change from baseline in pixel intensity quantification of flavoprotein autofluorescence at 6 months
Time frame: 0 months, 6 months
2 secondary outcomes
Change from baseline in pixel intensity quantification of fundus autofluorescence at 12 months
Time frame: 0 months, 12 months
Change from baseline in pixel intensity quantification of flavoprotein autofluorescence at 12 months
Time frame: 0 months, 12 months
Results and safety
The study was stopped early (reason given: Flavoprotein fluorescence machine for the trial is not working so the trial was terminated. No study data was collected prior to termination.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2016.
- Background: PubMed 19277237Elner SG, Elner VM, Field MG, Park S, Heckenlively JR, Petty HR. Retinal flavoprotein autofluorescence as a measure of retinal health. Trans Am Ophthalmol Soc. 2008;106:215-22; discussion 222-4.
- Background: PubMed 19491721Field MG, Elner VM, Park S, Hackel R, Heckenlively JR, Elner SG, Petty HR. Detection of retinal metabolic stress resulting from central serous retinopathy. Retina. 2009 Sep;29(8):1162-6. doi: 10.1097/IAE.0b013e3181a3b923.
- Background: PubMed 18625939Field MG, Elner VM, Puro DG, Feuerman JM, Musch DC, Pop-Busui R, Hackel R, Heckenlively JR, Petty HR. Rapid, noninvasive detection of diabetes-induced retinal metabolic stress. Arch Ophthalmol. 2008 Jul;126(7):934-8. doi: 10.1001/archopht.126.7.934.
- Background: PubMed 20398653Chen B, Tosha C, Gorin MB, Nusinowitz S. Analysis of autofluorescent retinal images and measurement of atrophic lesion growth in Stargardt disease. Exp Eye Res. 2010 Aug;91(2):143-52. doi: 10.1016/j.exer.2010.03.021. Epub 2010 Apr 14.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
K. Thiran Jayasundera, MD principal investigator
University of Michigan Kellogg Eye Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Stargardt Disease, run by University of Michigan at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Flavoprotein fluorescence machine for the trial is not working so the trial was terminated. No study data was collected prior to termination.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in pixel intensity quantification of fundus autofluorescence at 6 months (time frame: 0 months, 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01676766, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Nov 2016.