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NCT05626920

Disulfiram for Treatment of Retinal Degeneration

Recruiting now Phase 1, PHASE2 Last updated 6 December 2024
What this trial tests

Phase 1, PHASE2 trial testing Disulfiram 250 mg in Inherited Retinal Dystrophy Primarily Involving Sensory Retina in 30 participants. Currently enrolling.

Timeline
3 January 2025
Primary endpoint
1 September 2025
1 November 2025

Quick facts

Lead sponsorUniversity of Washington
PhasePhase 1, PHASE2
StatusRecruiting now
Study typeINTERVENTIONAL
Allocationrandomized
Designcrossover
Maskingquadruple
Primary purposetreatment
Enrollment30
Start date3 January 2025
Primary completion1 September 2025
Estimated completion1 November 2025
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

University of Washington

Who can join

18 and older, any sex, with Inherited Retinal Dystrophy Primarily Involving Sensory Retina. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

  1. Off-label and repurposed use of disulfiram beyond alcohol dependence: A systematic review of clinical and preclinical evidence.
    Benkő BM, De Brito Parreirinha NS, Sebe I, Zelkó R. · · 2026 · PMID 42058920 · DOI 10.1016/j.isci.2026.115441

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