Disulfiram for Treatment of Retinal Degeneration

April 28, 2026 updated by: Debarshi Mustafi, University of Washington

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

30

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Debarshi Mustafi, MD, PhD
  • Phone Number: 206-616-9305
  • Email: debarshi@uw.edu

Study Locations

  • United States
    • Washington
      • Seattle, Washington, United States, 98109
        • Recruiting
        • University of Washington-South Lake Union Retina Center
        • Contact:
          • Debarshi Mustafi, MD PhD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

Age ≥18 years Only those with a clinical diagnosis of inherited retinal degeneration. When available, supporting genetic diagnosis form a CLIA approved lab will be further considered for inclusion.

Only one eye per subject should be identified as the study eye. The study eye must meet the following criteria:

  • Best corrected ETDRS visual acuity letter score ≥ 70 (i.e., 20/40 or better) within 30 days of enrollment.
  • Goldmann visual field exhibiting constriction of visual fields to 10 degrees centrally
  • Able and willing to provide informed consent
  • Willing and able to abstain from alcohol consumption for the duration of the study and the 2 weeks preceding it and 2 weeks following the study end point

Liver function values that fall in the normal range as specified below:

  • Alanine transaminase (ALT): less than 40 IU/L
  • Aspartate transaminase (AST): less than 40 IU/L
  • Alkaline phosphatase (ALP): less than 300 IU/L
  • Albumin (Alb): less than 50 g/L
  • Total Protein: less than 80 g/L
  • Total Bilirubin: less than 30 umol/L

Exclusion Criteria:

  • A condition that, in the opinion of the investigator, would preclude participation in the study, e.g., alcohol dependence, cardiovascular disease, hepatitis.
  • Individuals with a history of diabetes mellitus
  • Individuals with a history of psychosis
  • Individuals with hypothyroidism
  • Individuals with hypersensitivity to thiuram derivatives causing rubber contact dermatitis
  • Those on anticoagulant therapy or other medications that may be affected by disulfiram.
  • An ocular condition, other than inherited retinal degeneration, is present such that, in the opinion of the investigator, visual acuity might be affected (e.g., foveal atrophy, pigment abnormalities, dense subfoveal hard exudates, non-retinal conditions such as epiretinal membrane or vitreo-macular traction, vein occlusion, uveitis or other ocular inflammatory diseases such as neovascular glaucoma, etc).
  • History of major ocular surgery within the prior 6 months or major ocular surgery anticipated within the next 6 months following randomization.
  • Exam evidence of severe external ocular infection, including conjunctivitis, chalazion, or substantial blepharitis
  • Participation in an investigational trial that involves treatment with any drug within 30 days of randomization that has not received regulatory approval at the time of study entry.

Note: study participants cannot receive another investigational drug while participating in this study.

  • Known allergy or hypersensitivity to any component of the study drug.
  • For women of child-bearing potential: pregnant or lactating or intending to become pregnant within the next 12 months. Women who are potential study participants should be questioned about the potential for pregnancy. Investigator judgment will be used to determine when a pregnancy test is needed.
  • Participants who expect to move out of the area of the clinical center during the 8 months of the study

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Placebo medication
Drug: Placebo
Placebo treatment
Active Comparator: Disulfiram
Disulfiram medication
Drug: Disulfiram 250 mg
Disulfiram treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assess the effects of disulfiram on retinal sensitivity in eyes afflicted with inherited retinal degenerations
Time Frame: Year 1
Evaluate change in contrast sensitivity measured by spatial contrast contrast sensitivity testing between disulfiram and placebo arms
Year 1

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assess the effects of disulfiram on visual acuity in eyes afflicted with inherited retinal degenerations.
Time Frame: Year 1
Evaluate change in best corrected visual acuity assessed by ETDRS chart testing between disulfiram and placebo arms
Year 1
Assess the effects of disulfiram on photoreceptor functionality in eyes afflicted with inherited retinal degenerations.
Time Frame: Year 1
Evaluate the change in retinal sensitivity as measured by standard and adaptive optics-scanning laser ophthalmoscopy based microperimetry
Year 1

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Washington

Investigators

  • Principal Investigator: Debarshi Mustafi, MD, PhD, University of Washington

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

May 3, 2026

Primary Completion (Estimated)

February 1, 2027

Study Completion (Estimated)

May 1, 2027

Study Registration Dates

First Submitted

November 15, 2022

First Submitted That Met QC Criteria

November 22, 2022

First Posted (Actual)

November 25, 2022

Study Record Updates

Last Update Posted (Actual)

May 5, 2026

Last Update Submitted That Met QC Criteria

April 28, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • STUDY00015434

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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