Inherited Retinal Dystrophy Primarily Involving Sensory RetinaPhase 1/2NCT05626920
Disulfiram for Treatment of Retinal Degeneration
A Phase 1/2 study testing Disulfiram 250 mg in people with Inherited Retinal Dystrophy Primarily Involving Sensory Retina, run by University of Washington at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Inherited Retinal Dystrophy Primarily Involving Sensory Retina
- What is being tested?
- Disulfiram 250 mg
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 3 May 2026to 1 May 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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.
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Washington-South Lake Union Retina Center
Seattle, Washington, 98109
Debarshi Mustafi, MD PhD
About this study
Official title: A Cross-over Randomized Control Trial to Evaluate the Retinaldehyde Dehydrogenase Inhibitor, Disulfiram, in Improving Retinal Sensitivity in Eyes Affected by Inherited Retinal Degeneration
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.
- Being tested
- Disulfiram 250 mg, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Disulfiram 250 mgDrug
Disulfiram treatment
PlaceboDrug
Placebo treatment
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo medication Receives: Drug: Placebo |
| Disulfiram | Active comparator | Disulfiram medication Receives: Drug: Disulfiram 250 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assess the effects of disulfiram on retinal sensitivity in eyes afflicted with inherited retinal degenerations
Evaluate change in contrast sensitivity measured by spatial contrast contrast sensitivity testing between disulfiram and placebo arms
Measured: Year 1
Secondary outcomes (2)
Assess the effects of disulfiram on visual acuity in eyes afflicted with inherited retinal degenerations.
Evaluate change in best corrected visual acuity assessed by ETDRS chart testing between disulfiram and placebo arms
Measured: Year 1
Assess the effects of disulfiram on photoreceptor functionality in eyes afflicted with inherited retinal degenerations.
Evaluate the change in retinal sensitivity as measured by standard and adaptive optics-scanning laser ophthalmoscopy based microperimetry
Measured: Year 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2022
- Study start3 May 2026 (estimated)
- Primary completion1 Feb 2027 (estimated)
- Study completion1 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Inherited Retinal Dystrophy Primarily Involving Sensory RetinaStudies of the same condition, open and under way.
- More trials run by University of WashingtonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- Randomized Cross-over study
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Washington
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Debarshi Mustafi, University of Washington
- Investigators
- Debarshi Mustafi, MD, PhD, Principal investigator, University of Washington
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05626920
- Sponsor's protocol ID
- STUDY00015434
- Status last verified
- Apr 2026
- First submitted
- 15 Nov 2022
- Last updated
- 5 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Cross-over Randomized Control Trial to Evaluate the Retinaldehyde Dehydrogenase Inhibitor, Disulfiram, in Improving Retinal Sensitivity in Eyes Affected by Inherited Retinal Degeneration
- ClinicalTrials.gov ID
- NCT05626920
- Acronym / sponsor ID
- STUDY00015434
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- University of WashingtonUniversity, hospital or other
- Study start
- 3 May 2026 (estimated)
- Primary completion
- 1 Feb 2027 (estimated)
- Study completion
- 1 May 2027 (estimated)
- First posted
- 25 Nov 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Randomized Cross-over study
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPlacebo medication |
| Disulfiram | Active comparator | Drug: Disulfiram 250 mgDisulfiram medication |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Disulfiram 250 mgDisulfiram treatment | Drug | Disulfiram |
| PlaceboPlacebo treatment | Drug | Placebo |
Primary outcome
Assess the effects of disulfiram on retinal sensitivity in eyes afflicted with inherited retinal degenerations
Time frame: Year 1
Evaluate change in contrast sensitivity measured by spatial contrast contrast sensitivity testing between disulfiram and placebo arms
2 secondary outcomes
Assess the effects of disulfiram on visual acuity in eyes afflicted with inherited retinal degenerations.
Time frame: Year 1
Assess the effects of disulfiram on photoreceptor functionality in eyes afflicted with inherited retinal degenerations.
Time frame: Year 1
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2026.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Debarshi Mustafi, MD, PhD principal investigator
University of Washington
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Disulfiram 250 mg in people with Inherited Retinal Dystrophy Primarily Involving Sensory Retina, run by University of Washington at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Debarshi Mustafi, MD, PhD (206-616-9305, debarshi@uw.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assess the effects of disulfiram on retinal sensitivity in eyes afflicted with inherited retinal degenerations (time frame: Year 1). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 May 2026.