RecruitingPhase 1Phase 2NCT05626920

Disulfiram for Treatment of Retinal Degeneration

A Cross-over Randomized Control Trial to Evaluate the Retinaldehyde Dehydrogenase Inhibitor, Disulfiram, in Improving Retinal Sensitivity in Eyes Affected by Inherited Retinal Degeneration


Sponsor

University of Washington

Enrollment

30 participants

Start Date

May 3, 2026

Study Type

INTERVENTIONAL

Conditions

Summary

Aberrant retinoic acid signaling driven by the degenerating outer retina leads to pathological changes to the inner retina. The resulting hyperactivity of retinal ganglion cells leads to further diminution of the remaining vision in those afflicted with inherited retinal diseases. Inhibition of this pathway has led to improved visual function in murine models of retinal degeneration. This can be accomplished in humans with the FDA-approved irreversible inhibitor of aldehyde dehydrogenases, disulfiram.


Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study is testing whether disulfiram, a medication normally used for alcohol dependence, can slow down or halt vision loss in people with inherited retinal degeneration — a group of genetic eye conditions that progressively destroy the light-sensing cells in the retina. Researchers believe disulfiram may protect retinal cells through a different biological mechanism. Participants must be willing and able to completely avoid alcohol during and around the time of the study. You may be eligible if: - You are 18 years of age or older - You have a clinical (and ideally genetic) diagnosis of inherited retinal degeneration - Your best corrected vision in the study eye is 20/40 or better - Your visual field is constricted to 10 degrees centrally - Your liver function tests are within normal limits - You are willing to completely abstain from alcohol for the duration of the study and the 2 weeks before and after You may NOT be eligible if: - You have alcohol dependence, cardiovascular disease, or hepatitis - You have a history of diabetes, psychosis, or hypothyroidism - You have a known allergy to thiuram derivatives (found in certain rubber products) - You are taking blood thinners or other medications that interact with disulfiram - You have other eye conditions affecting your vision beyond the retinal degeneration - You have had major eye surgery within the past 6 months - You are pregnant, breastfeeding, or planning to become pregnant Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGDisulfiram 250 mg

Disulfiram treatment

DRUGPlacebo

Placebo treatment


Locations(1)

University of Washington-South Lake Union Retina Center

Seattle, Washington, United States

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NCT05626920