Actively Recruiting
Disulfiram for Treatment of Retinal Degeneration
Led by University of Washington · Updated on 2026-05-05
30
Participants Needed
1
Research Sites
51 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Disulfiram for Treatment of Retinal Degeneration
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Clinical diagnosis of inherited retinal degeneration
- One study eye with best corrected ETDRS visual acuity letter score 70 or better (20/40 or better) within 30 days of enrollment
- Visual field constriction of 10 degrees centrally in the study eye
- Ability and willingness to provide informed consent
- Willingness and ability to abstain from alcohol during the study and for 2 weeks before and after the study endpoint
- Liver function values within normal ranges: ALT less than 40 IU/L, AST less than 40 IU/L, ALP less than 300 IU/L, Albumin less than 50 g/L, Total Protein less than 80 g/L, Total Bilirubin less than 30 umol/L
You will not qualify if you...
- Conditions precluding study participation including alcohol dependence, cardiovascular disease, hepatitis
- History of diabetes mellitus
- History of psychosis
- History of hypothyroidism
- Hypersensitivity to thiuram derivatives causing rubber contact dermatitis
- Use of anticoagulant therapy or medications affected by disulfiram
- Ocular conditions other than inherited retinal degeneration that may affect visual acuity
- History of major ocular surgery within 6 months prior or anticipated within 6 months after randomization
- Severe external ocular infection such as conjunctivitis, chalazion, or substantial blepharitis
- Participation in another investigational drug trial within 30 days prior to randomization
- Known allergy or hypersensitivity to any component of the study drug
- Women who are pregnant, lactating, or intending pregnancy within 12 months
- Expected to move out of the clinical center area during the 8-month study period
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
University of Washington-South Lake Union Retina Center
Seattle, Washington, United States, 98109
Actively Recruiting
Research Team
D
Debarshi Mustafi, MD, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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