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Aldehyde Dehydrogenase Inhibitor

Disulfiram for Retinal Degeneration

Phase 1 & 2
Recruiting
Led By Debarshi Mustafi, MD, PhD
Research Sponsored by University of Washington
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up year 1

Summary

This trial showed that a drug that blocks a certain pathway in the eye led to improved vision in a mouse model of inherited retinal diseases.

Who is the study for?
Adults over 18 with a clinical diagnosis of inherited retinal degeneration, who have relatively good vision (20/40 or better) and can see within 10 degrees centrally. Participants must not drink alcohol for the duration of the study plus two weeks before and after. They should also have normal levels of albumin, total protein, ALT, AST, and ALP.
What is being tested?
The trial is testing Disulfiram (250 mg), an FDA-approved drug that may improve visual function by inhibiting a pathway linked to vision loss in retinal degeneration. It's compared against a placebo to evaluate its effectiveness in humans.
What are the potential side effects?
Disulfiram can cause side effects such as headaches, fatigue, skin rash or acne; more rarely it might lead to liver problems or neurological changes.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~year 1
This trial's timeline: 3 weeks for screening, Varies for treatment, and year 1 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Assess the effects of disulfiram on retinal sensitivity in eyes afflicted with inherited retinal degenerations
Secondary study objectives
Assess the effects of disulfiram on photoreceptor functionality in eyes afflicted with inherited retinal degenerations.
Assess the effects of disulfiram on visual acuity in eyes afflicted with inherited retinal degenerations.

Trial Design

2Treatment groups
Active Control
Placebo Group
Group I: DisulfiramActive Control1 Intervention
Disulfiram medication
Group II: PlaceboPlacebo Group1 Intervention
Placebo medication

Find a Location

Who is running the clinical trial?

University of WashingtonLead Sponsor
1,831 Previous Clinical Trials
1,907,539 Total Patients Enrolled
Debarshi Mustafi, MD, PhDPrincipal InvestigatorUniversity of Washington
~20 spots leftby Sep 2025
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