Disulfiram for Treatment of Retinal Degeneration

Launched by UNIVERSITY OF WASHINGTON · Nov 22, 2022

Keywords

ClinConnect Summary

This clinical trial is studying the use of a medication called disulfiram to treat a condition known as inherited retinal degeneration, which affects vision by damaging the retina, the light-sensitive layer at the back of the eye. Researchers believe that disulfiram can help improve vision by blocking certain harmful signals in the retina that worsen vision loss. The trial is currently recruiting participants aged 18 and older who have been diagnosed with this condition and have some remaining vision in one eye.

To be considered for the study, participants must meet specific criteria, such as having a certain level of visual acuity (meaning they can see well enough), and they must be willing to abstain from alcohol for the duration of the study. Participants will undergo assessments to ensure their liver function is normal and that they do not have certain other health conditions that might interfere with the study. If you join, you can expect regular check-ups and assessments of your vision over the course of the study. It's also important to note that if you're pregnant, breastfeeding, or planning to become pregnant, you won't be able to participate.

Gender

ALL

Eligibility criteria

• 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