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Oral Metformin for Treatment of ABCA4 Retinopathy

Sponsored by National Eye Institute (NEI)

Quick Self-Assessment

See if you qualify and express your interest

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Current Status

Recruiting

Auto-verified from ClinicalTrials.gov

Verified Oct 27, 2025

Phase

Clinical Trial

Sponsor

National Eye Institute (NEI)

Enrollment Target

56

Start Date

Nov 2020

๐Ÿ“Š
1 of 56 participants interested
2% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

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.

Who Can Participate

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

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

๐Ÿ“‹

Study Type

INTERVENTIONAL

๐Ÿ‘ฅ

Target Enrollment

56 participants

๐ŸŽ‚

Age Range

12 Years - 100 Years

โšง

Gender

ALL

๐Ÿข

Sponsor

National Eye Institute (NEI)

Study Locations

Choose your preferred location or select flexible during enrollment

RECRUITING

Bethesda

Maryland

Location available
View Bethesda location page
RECRUITING

Ann Arbor

Michigan

Location available
View Ann Arbor location page

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

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Looking for Stargardt Disease Treatment in Bethesda?

Join others in Maryland exploring innovative treatment options through clinical research

Stargardt Disease Treatment Options in Bethesda, Maryland

If you're searching for Stargardt Disease treatment in Bethesda, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Bethesda, Ann Arbor and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Stargardt Disease. All study-related care is provided at no cost to participants.

Local Sites
2 locations in Maryland
Now Enrolling
Up to 56 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Stargardt Disease?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Stargardt Disease

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Stargardt Disease Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT04545736. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.