Using disulfiram to treat retinal degeneration

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial investigates the use of disulfiram, an FDA-approved medication, to inhibit aberrant retinoic acid signaling that contributes to retinal degeneration. The study aims to improve visual function in patients with inherited retinal dystrophy by targeting the hyperactivity of retinal ganglion cells. Participants will receive either disulfiram or a placebo, and the study will assess the impact on their visual acuity and field of vision. The trial is designed for adults with a specific diagnosis of inherited retinal degeneration.

Why it matters

Who can join: the full rules

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

Where it's running

• University of Washington-South Lake Union Retina Center
  Seattle, Washington, United States Recruiting

Study contacts

• Principal investigator: Debarshi Mustafi, MD, PhD — University of Washington
• Study coordinator: Debarshi Mustafi, MD, PhD
• Email: debarshi@uw.edu
• Phone: 206-616-9305

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.