NCT05626920 Disulfiram for Treatment of Retinal Degeneration
| NCT ID | NCT05626920 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | University of Washington |
| Condition | Inherited Retinal Dystrophy Primarily Involving Sensory Retina |
| Study Type | INTERVENTIONAL |
| Enrollment | 30 participants |
| Start Date | 2026-05-03 |
| Primary Completion | 2027-02-01 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 30 participants in total. It began in 2026-05-03 with a primary completion date of 2027-02-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
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
Contact & Investigator
Debarshi Mustafi, MD, PhD
PRINCIPAL INVESTIGATOR
University of Washington
Frequently Asked Questions
Who can join the NCT05626920 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Inherited Retinal Dystrophy Primarily Involving Sensory Retina. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05626920 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05626920 currently recruiting?
Yes, NCT05626920 is actively recruiting participants. Contact the research team at debarshi@uw.edu for enrollment information.
Where is the NCT05626920 trial being conducted?
This trial is being conducted at Seattle, United States.
Who is sponsoring the NCT05626920 clinical trial?
NCT05626920 is sponsored by University of Washington. The principal investigator is Debarshi Mustafi, MD, PhD at University of Washington. The trial plans to enroll 30 participants.