NCT05626920

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: University of Washington · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Inherited Retinal Dystrophy Primarily Involving Sensory Retina

Interventions

Type	Name	Description
DRUG	Disulfiram 250 mg	Disulfiram treatment
DRUG	Placebo	Placebo treatment

Timeline

• Start date: 2025-01-03
• Primary completion: 2025-09-01
• Completion: 2025-11-01
• First posted: 2022-11-25
• Last updated: 2024-12-06

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study