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

NCT06319872 | Recruiting Phase 1 FDA Drug

• 1 other identifier: STUDY00009118
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• mean change in Best Corrected Visual Acuity (BCVA) score assessed by ETDRS

  Visual acuity testing will be performed by Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity with refractive correction when indicated under photopic and mesopic conditions. The test will be performed by an ophthalmic technician and will be used for research purpose only. The right eye should be tested first. When it becomes evident that no further meaningful readings can be made, the examiner should stop the test. The final visual acuity should be noted. Visual acuity is a range from 20/20 to Light Perception, where 20/20 is considered the normal best visual acuity measurement.

  baseline to day 180

Secondary Outcomes (13)

• mean change in total contrast sensitivity score using Pelli-Robson charts

  baseline to day 180

• mean change in light-adapted microperimetry sensitivity assessed using standard MAIA microperimetry equipment

  baseline to day 180

• mean change in outer retinal thickness assessed using Spectral Domain Optical Coherence Tomography (SD-OCT)

  baseline to day 180

• mean change in fundus autofluorescence (FAF)

  baseline to day 180

• number of participants with retinal anatomy changes assessed using fundus photos

  baseline to day 180

Study Arms (1)

All participants

EXPERIMENTAL

Participants will receive either drug or placebo for 180 days.

Drug: Oral disulfiram

Interventions

Oral disulfiram DRUG

250 mg/day

Also known as: Antabuse

All participants

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Rochester: lead

Study Sites (1)

Flaum Eye Institute, University of Rochester Medical Center

Rochester, New York, 14642, United States

RECRUITING

MeSH Terms

Conditions

Alcoholism; Retinal Dystrophies; Macular Degeneration; Retinitis Pigmentosa; Stargardt Disease

Interventions

Disulfiram

Condition Hierarchy (Ancestors)

Alcohol-Related Disorders; Substance-Related Disorders; Chemically-Induced Disorders; Mental Disorders; Retinal Degeneration; Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Ditiocarb; Thiocarbamates; Carbamates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Disulfides; Sulfides; Sulfur Compounds

Central Study Contacts

Evan Burr

CONTACT

585- 275-5234; evan_burr@urmc.rochester.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief, Pediatric Ophthalmology and Ocular Genetics

Study Record Dates

• First Submitted: March 6, 2024
• First Posted: March 20, 2024
• Study Start: May 19, 2025
• Primary Completion (Estimated): May 19, 2029
• Study Completion (Estimated): May 19, 2029
• Last Updated: September 29, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)