Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy
Information source: University of Utah
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Stargardt Macular Dystrophy
Intervention: 4-Methylpyrazole (Drug)
Phase: Phase 1
Sponsored by: University of Utah
Official(s) and/or principal investigator(s):
Paul S Bernstein, M.D., Ph.D., Principal Investigator, Affiliation: University of Utah
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 patientsâ vision, and may also have similar
effects in other macular degenerative diseases.
Official title: Clinical Interventions Against Stargardt Macular Dystrophy: Phase 1 Pilot Study of 4-MP as an Inhibitor of Dark Adaptation
Study design: Treatment, Randomized, Double-Blind, Placebo Control, Factorial Assignment, Efficacy Study
Primary outcome: Dark adaptation inhibition measured 30 minutes after drug infusion using Goldman-Weeker adaptometer.
Minimum age: 18 Years.
Maximum age: 65 Years.
- All nonpregnant, nonlactating adults with normal vision in both eyes
- Previous ocular pathologies
Locations and Contacts
Moran Eye Center, University of Utah, Salt Lake City, Utah 84132, United States
Starting date: November 2005
Ending date: May 2006
Last updated: January 10, 2007