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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
30
Disulfiram treatment
Placebo treatment
University of Washington-South Lake Union Retina Center
Seattle, Washington, United States
RECRUITINGAssess 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
Time frame: Year 1
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
Time frame: 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
Time frame: Year 1
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