Oral Metformin for Treatment of ABCA4 Retinopathy (NCT04545736) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Oral Metformin for Treatment of ABCA4 Retinopathy
United States55 participantsStarted 2020-11-23
Plain-language summary
Background:
ABCA4 retinopathy is a genetic disease in which the ABCA4 protein is absent or faulty. It can cause waste material to collect in the eye and may cause cells to die. The cell death can lead to vision loss. Researchers want to see if an oral drug called metformin can help.
Objective:
To see if metformin is safe and possibly helps to slow the rate of ABCA4 retinopathy.
Eligibility:
People age 12 and older who have ABCA4 retinopathy and have problems with their vision.
Design:
Participants will be screened under a separate protocol.
Participants will have a medical and family history. They will complete a questionnaire about their vision and daily activities. They will have a physical exam. They may have blood drawn through a needle in the arm.
Participants will have an eye exam. Their pupils may be dilated with eye drops. Their retina may be photographed.
Participants will have a visual field test. They will sit in front of a large dome and press a button when they see a light within the dome.
Participants will have an electroretinogram. It examines the function of the retina. They will sit in the dark for 30 minutes. Then their eyes will be numbed with eye drops. They will wear contact lenses that can sense signals from the retinas. They will watch flashing lights.
Participants will have optical coherence tomography. This non-invasive procedure makes pictures of the retina.
Participants will have fundus autofluorescence. A bright blue light will be shone into their eye.
Participants will take metformin by mouth for 24 months.
Participants will have study visits every 6 months. Participation will last for at least 36 months....
Who can participate
Age range
12 Years – 100 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Participant must be at least 12 years of age.
. Participant (or legal guardian) must understand and sign the protocol s informed consent document.
. Participant must have at least one definite pathogenic or likely pathogenic mutation in ABCA4 and a typical clinical presentation of Stargardt disease and phenotypic presentation of ABCA4 retinopathy in both eyes.
. Participant must have at least two years of natural history data from at least four data points (a) The separation between any two consecutive data points must be at least six months (b) The most recent data point must be at least 4.5 months and no more than 16 months prior to the baseline visit (c)
. Potential participants with three natural history data points may be enrolled to obtain their fourth natural history data point on protocol.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial is testing oral metformin — a drug usually used for diabetes — as a potential treatment for ABCA4 retinopathy and Stargardt disease, so can you explain why researchers think metformin might slow down retinal degeneration in my specific condition?
2The trial is measuring something called 'ellipsoid zone band loss' on retinal imaging to track disease progression — can you tell me whether my own imaging results would give us a useful baseline to compare against what this trial is finding?
3Since this is a Phase 1/2 trial, what do we currently know and not know about the safety and effectiveness of metformin at the doses being used for eye disease, as opposed to its well-established use for diabetes?
4The trial is listed as active but no longer recruiting, so if I can't enroll, are there other similar studies or compassionate-use options I should be looking into, or is watchful waiting still a reasonable path for my situation?
5Given that the primary goal is to slow the growth of retinal damage rather than restore lost vision, how would you and I track whether my disease is progressing, and what would that mean for future treatment decisions?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The difference in growth rate of square-root transformed area of EZ band loss (vAreaEZloss)
. The separation between any two consecutive data points may fall short of 6 months by no more than 45 days; however, the total separation among the data points must allow for at least two years of natural history data.
. Potential participants with four or more natural history data points completed off protocol, all of which having occurred more than 16 months prior to the baseline visit, may be enrolled to complete an additional natural history data point on protocol within the required timeframe from the baseline visit.
. Participant must agree to adhere to Lifestyle Considerations throughout study duration.
Exclusion criteria
. Participant is actively receiving study IP in another investigational study.
. Participant has a condition that would preclude participation in the study (e.g., unstable medical status including blood pressure and glycemic control) by interfering with the participant s ability to engage in the required protocol evaluation and testing and/or comply with study visits.
. Any female participant of childbearing potential that is pregnant or breast-feeding at the time of enrollment or planning to become pregnant during the study.
. Participant has definitive pathogenic or likely pathogenic mutations in RDS/peripherin (PRPH2), PROM1, and/or ELOVL4.
. Participant has a history of chronic renal impairment as measured in the acute care panel estimated glomerular filtration rate (eGFR)\<45 ml/min/1.73 m2) or severe hepatic, pulmonary, or cardiovascular disease (hypoxic state).
. Participant is taking any medication that could adversely interact with metformin (e.g., cimetidine, furosemide, nifedipine) and cannot switch to an alternative medication.
. Participant is currently taking metformin or participant has taken metformin during the period of natural history data collection that will be used for this study for a cumulative total of more than one month (\> 31 days).
. Participant has a known hypersensitivity to metformin.