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Disulfiram for Treatment of Retinal Degeneration

NCT05626920 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2026-05-05

No results posted yet for this study

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

DRUG

Disulfiram 250 mg

Disulfiram treatment

DRUG

Placebo

Placebo treatment

Sponsors & Collaborators

Principal Investigators

  • Debarshi Mustafi, MD, PhD · University of Washington

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-05-03
Primary Completion
2027-02-01
Completion
2027-05-01
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05626920 on ClinicalTrials.gov