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Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00346853
Recruitment Status : Completed
First Posted : June 30, 2006
Last Update Posted : July 10, 2008
Information provided by:
University of Utah

Brief 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.

Condition or disease Intervention/treatment Phase
Macular Dystrophy, Corneal Drug: 4-Methylpyrazole Other: saline Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Clinical Interventions Against Stargardt Macular Dystrophy: Phase 1 Pilot Study of 4-MP as an Inhibitor of Dark Adaptation
Study Start Date : November 2005
Actual Primary Completion Date : May 2006
Actual Study Completion Date : September 2006

Arm Intervention/treatment
Experimental: 1 Drug: 4-Methylpyrazole
15 mg/kg dose

Placebo Comparator: 2
Other: saline

Primary Outcome Measures :
  1. Dark adaptation inhibition measured 30 minutes after drug infusion using Goldman-Weeker adaptometer. [ Time Frame: 6 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

Inclusion Criteria:

  • All nonpregnant, nonlactating adults with normal vision in both eyes

Exclusion Criteria:

  • Previous ocular pathologies

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00346853

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United States, Utah
Moran Eye Center, University of Utah
Salt Lake City, Utah, United States, 84132
Sponsors and Collaborators
University of Utah
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Principal Investigator: Paul S Bernstein, M.D., Ph.D. University of Utah
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Responsible Party: Dr. Randall Olson, Chair, Department of Ophthalmology Identifier: NCT00346853    
Other Study ID Numbers: 4-MP Dark Adaptation Inhib.
First Posted: June 30, 2006    Key Record Dates
Last Update Posted: July 10, 2008
Last Verified: July 2008
Keywords provided by University of Utah:
Dark adaptation inhibition
Stargardt macular dystrophy
4-Methylpyrazole (4-MP)
Additional relevant MeSH terms:
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Macular Degeneration
Corneal Dystrophies, Hereditary
Retinal Degeneration
Retinal Diseases
Eye Diseases
Corneal Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Protective Agents
Physiological Effects of Drugs
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action