The Effects of Disulfiram (Antabuse®) on Visual Acuity in Patients With Retinal Degeneration

NCT ID: NCT06319872

Last Updated: 2025-09-29

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-05-19
• Study Completion Date: 2029-05-19

Brief Summary

Oral disulfiram (Antabuse®) has been shown to improve image-forming vision in animal models with retinal degeneration due to its ability to decrease Retinoic Acid synthesis and consequently reduce hyperactivity in the inner retina. The investigator will aim to evaluate the impact of oral disulfiram on the vision of patients with retinal degeneration who are being treated with the drug in the management of their concurrent alcohol use disorder.

Conditions

Alcohol Use Disorder; Retinal Dystrophies; Age-Related Macular Degeneration; Retinitis Pigmentosa; Stargardt Disease

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

All participants

Participants will receive either drug or placebo for 180 days.

Group Type: EXPERIMENTAL

Oral disulfiram

Intervention Type: DRUG

250 mg/day

Interventions

Oral disulfiram

250 mg/day

Intervention Type: DRUG

Other Intervention Names

Antabuse

Eligibility Criteria

Inclusion Criteria

• All sexes, 18 years and older.
• Participants must speak English, understand, and sign the informed consent document.
• Stated willingness to comply with all study procedures and availability for the duration of the study.
• In good general health as evidenced by medical history and with a clinical diagnosis of inherited retinal dystrophy or dry-AMD.
• Best Corrected Visual Acuity (BCVA) of 20/20 (with constriction or other defects of Goldmann visual field) to Light Perception in the better eye.
• Intact inner nuclear layer, inner plexiform, and ganglion cell layer on macular SD-OCT.
• Ability to take oral medication and be willing to adhere to the disulfiram regimen.
• Patients must have the diagnosis of alcohol use disorder provided by an addiction specialist and be a candidate for therapeutic use of disulfiram for that condition.
• Patients must agree to refrain from all alcohol consumption for 180 days.
• Any female participant of childbearing potential must have a negative urine pregnancy test at screening.
• Any female participant of childbearing potential must have (or have a partner who has) had a surgical sterilization (vasectomy, hysterectomy, or tubal ligation), be completely abstinent from intercourse or must agree to practice two acceptable methods of contraception throughout the course of the study and for at least one week after disulfiram discontinuation. Acceptable methods of contraception include hormonal contraception (i.e., birth control pills, injected hormones, dermal patch, or vaginal ring); intrauterine device; barrier methods (diaphragm, condom) with spermicide.

Exclusion Criteria

• A condition that, in the opinion of the investigator, would preclude participation in the study, e.g., 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.
• Ophthalmic conditions with independent effect upon visual function (e.g. diabetic retinopathy, glaucoma, cataract, vitreous hemorrhage, retinal detachment, active intraocular inflammation or active infectious ocular diseases, choroidal neovascularization).
• Patients with No Light Perception (NLP) in both eyes.
• 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.
• Participants who expect to move out of the area of the clinical center during the 8 months of the study

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Rochester

OTHER

Sponsor Role: lead

Responsible Party

Alex Levin

Chief, Pediatric Ophthalmology and Ocular Genetics

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Flaum Eye Institute, University of Rochester Medical Center

Rochester, New York, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Evan Burr

Role: CONTACT

evan_burr@urmc.rochester.edu

585- 275-5234

Facility Contacts

Evan Burr

Role: primary

evan_burr@urmc.rochester.edu

585- 275-5234

Other Identifiers

STUDY00009118

Identifier Type: -

Identifier Source: org_study_id