Disulfiram (Antabuse) for vision in retinal degeneration

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1 interventional study gives oral disulfiram to adults with inherited retinal dystrophies or dry age-related macular degeneration who are also candidates for disulfiram treatment of alcohol use disorder. Participants will take disulfiram and undergo serial vision testing and retinal imaging (including best-corrected visual acuity, visual fields, and macular SD-OCT) to look for changes in image-forming vision and inner retinal structure. Key eligibility includes intact inner nuclear, inner plexiform, and ganglion cell layers on macular SD-OCT and a documented diagnosis of alcohol use disorder from an addiction specialist, with agreement to abstain from alcohol for 180 days. The study is conducted at the Flaum Eye Institute, University of Rochester Medical Center and is intended to detect early signals of visual benefit and safety in humans.

Why it matters

Who can join: the full rules

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

Where it's running

• Flaum Eye Institute, University of Rochester Medical Center
  Rochester, New York, United States Recruiting

Study contacts

• Study coordinator: Evan Burr
• Email: evan_burr@urmc.rochester.edu
• Phone: 585- 275-5234

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Functional visual field and navigation testing for moderate-to-profound inherited retinal disease

  This project will test whether functional visual field and navigation tests can measure real-world vision and mobility in people with moderate-to-profound inherited retinal diseases.

  Recruiting Ray Therapeutics, Inc. · Irvine, California · NCT07502664

• Assessing vision measurement methods for patients with retinal degeneration

  This study is testing different ways to measure vision in people with retinal degeneration to see how their eyesight affects daily life and how it changes over time.

  Recruiting Ray Therapeutics, Inc. · Irvine, California · NCT06375239

• Using stem cells to treat retinal and optic nerve damage

  This study is testing if using stem cells from your own bone marrow can help improve vision in people with serious eye damage from conditions like age-related macular degeneration and optic nerve problems.

  Recruiting at 4 sites MD Stem Cells · Westport, Connecticut and 3 other locations · NCT03011541

• C.001 for retinal degeneration: a first-in-human dose-escalation safety test

  A first-in-human study will test the safety and tolerability of a single subretinal injection of C.001 in adults with retinal diseases involving retinal pigment epithelium degeneration.

  Recruiting at 2 sites Phase 1 Cellio Therapeutics Inc · Beverly Hills, California and 2 other locations · NCT07594236

• Imaging of outer retinal diseases using adaptive optics

  This study is trying to take detailed pictures of the retina in people with outer retinal diseases to help find better ways to diagnose and understand these conditions.

  Recruiting at 2 sites Food and Drug Administration (FDA) · Bethesda, Maryland and 1 other locations · NCT05355415

• Understanding Age-Related Macular Degeneration and Its Progression

  This study looks at how age-related macular degeneration develops and progresses by comparing people with the condition to healthy individuals, using non-invasive tests and personal feedback.

  Recruiting Center for Eye Research Australia · East Melbourne, Victoria · NCT04739319

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.