Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy
Status:
Completed
Trial end date:
2006-09-01
Target enrollment:
Participant gender:
Summary
The purpose of this study is to investigate whether taking 4-methylpyrazole (4-MP,
fomepizole, Antizol™) inhibits dark adaptation of the eye. In other words, we are testing if
4-MP slows the processing of vitamin A derivatives in the eye. By slowing down these
processes, individuals with Stargardt disease may have better chances of saving their
remaining vision. 4-MP has been shown to slow dark adaptation in animals, and is FDA approved
for human use to treat individuals with methanol or ethylene glycol (antifreeze) poisoning by
shutting down the body's ability to process alcohols. This medication has an excellent safety
profile and has been reported to have no short-term or long-term side effects, as long as
patients refrain from any alcohol while the medication is in the body. A single dose of 4-MP
remains in the body for about 12 hours, and therefore, it may inhibit dark adaptation of your
eyes for up to 12 hours. Studying the effects of 4-MP may lead to effective medical treatment
to save Stargardt patients' vision, and may also have similar effects in other macular
degenerative diseases.