Oral Metformin for Treatment of ABCA4 Retinopathy

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Eye Institute (NEI)
Study ID
NCT04545736
Phase
PHASE1/PHASE2
Status
Active Not Recruiting

Conditions

  • ABCA4 Retinopathy
  • Retinal Degeneration
  • Retinal Dystrophy
  • Stargardt Disease

Eligibility Criteria

Sex
ALL
Age
12 Years - 100 Years
Healthy Volunteers
Not accepted

Interventions

  • Metformin hydrochloride — DRUG
    Metformin is commercially produced in immediate and extended release. Participants will receive an immediate release formulation of metformin of 500mg daily at study entry. This dose will be titrated up weekly in 500mg increments to reach a goal of 2000mg daily maximum. Once participants \>=17 years of age reach 2000mg metformin immediate release they will switch to an extended-release formulation (1000mg twice a day by mouth). Participants \>= 17 years of age that cannot tolerate 2000mg will be permitted to reduce their daily dose to a minimum of 1000mg/day. Because metformin extended release is not FDA-approved for children under the age of 17, participants under 17 will remain on the immediate release formulation. For these participants who remain on standard formulation, the maximum tolerated dose between 1000mg and 2000mg/day will be given.

Study Details

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....

Key Dates

Start date
Nov 23, 2020
Status verified
Jun 2026
Primary completion
Feb 2, 2028
Completion
Feb 2, 2029

Study Design

Enrollment
55 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Metformin
    Oral administration of metformin

Primary Outcome Measure

The difference in growth rate of square-root transformed area of EZ band loss (vAreaEZloss) [ Time Frame: Pre-treatment, Baseline, Month 24 ]

Locations (2)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892-
University of MichiganAnn ArborMichigan48109-0624-

Find similar trials in Bethesda, MD

By research site
National Institutes of Health Clinical Center· Bethesda, MDUniversity of Michigan· Ann Arbor, MI

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