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Could a common diabetes drug protect eyesight in stargardt disease?

NCT ID NCT04545736

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Aug 20, 2026 · Updated 7 times

Summary

This study tests whether metformin, a widely used diabetes drug, can safely slow vision loss in people with ABCA4 retinopathy (Stargardt disease). Fifty-five participants aged 12 and older will take metformin by mouth for 24 months, with regular eye exams to track changes. The goal is to see if the drug reduces the rate of retinal damage.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
metformin (a common diabetes drug)
What this could lead to
If it works, this could point toward a treatment that slows vision loss in people with ABCA4 retinopathy.
What could go wrong
This is an early-phase trial with only 55 people, so results may not apply to everyone. Metformin can cause stomach upset and other side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

55 people

The number who actually took part.

Started

Nov 2020

Expected to finish

Feb 2029

An estimate. End dates often move.

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 100 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

* INCLUSION CRITERIA: To be eligible, the following inclusion criteria must be met, where applicable. 1. Participant must be at least 12 years of age. 2. Participant (or legal guardian) must understand and sign the protocol s informed consent document. 3. 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. 4. 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) 1. Potential participants with three natural history data points may be enrolled to obtain their fourth natural history data point on protocol. 2. 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. 3. 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. 5. Participant must agree to adhere to Lifestyle Considerations throughout study duration. 6. Any female participant of childbearing potential must have a negative urine pregnancy test at screening and be willing to undergo urine pregnancy tests throughout the study. 7. Any female participant of childbearing potential must: * Have had a surgical sterilization OR * Completely abstain from intercourse OR * Practice at least one form of contraception while actively taking IP on the study and at least one week after IP discontinuation OR * Have a partner who had a surgical sterilization OR * Have a partner who practices one form of contraception while the female participant is actively taking IP and at least one week after the female participant discontinues IP. Any male participants must: * Have had a surgical sterilization OR * Completely abstain from intercourse OR * Practice at least one form of contraception while actively taking IP on the study and at least for 3 months after IP discontinuation OR * Have a partner who had a surgical sterilization OR * Have a partner who practices one form of contraception while the male participant actively takes IP and at least 3 months after the male participant discontinues IP. 1. Acceptable forms of surgical sterilization include: vasectomy, hysterectomy, or tubal ligation. 2. Acceptable methods of contraception include: hormonal contraception (i.e., birth control pills, injected hormones, dermal patch or vaginal ring), intrauterine device, or barrier methods (diaphragm, condom) with spermicide. EXCLUSION CRITERIA: An individual who meets any of the following criteria will be excluded from participation in this study: 1. Participant is actively receiving study IP in another investigational study. 2. 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. 3. 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. 4. Participant has definitive pathogenic or likely pathogenic mutations in RDS/peripherin (PRPH2), PROM1, and/or ELOVL4. 5. 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). 6. Participant is taking any medication that could adversely interact with metformin (e.g., cimetidine, furosemide, nifedipine) and cannot switch to an alternative medication. 7. 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). 8. Participant has a known hypersensitivity to metformin. 9. Participant has not stopped taking a glucagon-like peptide 1 (GLP-1) agonist at least two weeks prior to enrollment or is currently taking a GLP-1 agonist. 10. Participant has a history of chronic lactic acidosis, including diabetic ketoacidosis, with or without coma. 11. Participant has type 1 diabetes mellitus. 12. Scarring due to choroidal neovascularization (CNV) is present in either eye. QUALIFYING EYE ELIGIBILITY CRITERIA: In order to participate in the study, the participant must have at least one qualifying eye that meets all of the inclusion and none of the exclusion criteria listed below. Qualifying Eye Inclusion Criteria: 1\. A growth rate of square root AreaEZloss \> 0.025 mm/year based on calculation from natural history data. Qualifying Eye Exclusion Criteria: 1\. Retinal degeneration has advanced beyond a point where reliable measurement of the integrity of the IS-OS on OCT is possible.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109-0624, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.