Trials / NCT05626920
Disulfiram for Treatment of Retinal Degeneration
Recruiting now Phase 1 / Phase 2 Drug / neuroprotection
- Sponsor
- University of Washington
- Treatment
- Disulfiram 250 mg; Placebo
- Gene
- Not stated
- Conditions
- Inherited Retinal Dystrophy Primarily Involving Sensory Retina
- Ages
- 18 Years to no upper limit
- Enrollment
- 30 participants
- Started
- 2026-05-03
- Main results expected
- 2027-02-01
- Results posted
- Not yet
- Record last updated
- 2026-05-05
What the study is testing
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.
Where it runs
United States (1)
- University of Washington-South Lake Union Retina Center, Seattle, Washington (recruiting now)