Disulfiram for Treatment of Retinal Degeneration

Conditions

• Inherited Retinal Dystrophy Primarily Involving Sensory Retina

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Disulfiram 250 mg — DRUG
  Disulfiram treatment
• Placebo — DRUG
  Placebo treatment

Study Details

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.

Key Dates

Start date

May 3, 2026

Status verified

Apr 2026

Primary completion

Feb 1, 2027

Completion

May 1, 2027

Study Design

Enrollment

30 participants (estimated)

Allocation

RANDOMIZED

Intervention model

CROSSOVER

Primary purpose

TREATMENT

Arms

• Placebo Comparator: Placebo
  Placebo medication
• Active Comparator: Disulfiram
  Disulfiram medication

Primary Outcome Measure

Assess the effects of disulfiram on retinal sensitivity in eyes afflicted with inherited retinal degenerations [ Time Frame: Year 1 ]

Central Contacts

• Debarshi Mustafi, MD, PhD
  206-616-9305
  [email protected]

Locations (1)

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