Recruiting
Phase 1
Phase 2

Disulfiram

Sponsor:

University of Washington

Code:

NCT05626920

Conditions

Inherited Retinal Dystrophy Primarily Involving Sensory Retina

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Disulfiram 250 mg

Placebo

Study Details

Brief 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

Study ID

NCT05626920

Start date

May 3, 2026

Status verified date

Apr, 2026

Completion date

May 1, 2027

Anticipated

Primary completion date

Feb 1, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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 Design

Enrollment

30 participants

Anticipated

Allocation

Randomized

Intervention Model

Crossover

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

placebo comparator: Placebo

Placebo medication

active comparator: Disulfiram

Disulfiram medication

Interventions

Disulfiram 250 mg

Disulfiram treatment

Placebo

Placebo treatment

Primary outcome measure

  • Assess the effects of disulfiram on retinal sensitivity in eyes afflicted with inherited retinal degenerations [ Time Frame: Year 1 ]

Central Contacts and Locations

Central contacts

Debarshi Mustafi, MD, PhD

206-616-9305debarshi@uw.edu

Locations

University of Washington-South Lake Union Retina Center

Recruiting

Seattle, Washington, United States, 98109

More Information

Sponsor

University of Washington

Last update posted

May 5, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of Washington on 2026-05-05.