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

This study has been completed.
Information provided by:
University of Utah Identifier:
First received: June 28, 2006
Last updated: July 9, 2008
Last verified: July 2008
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 Intervention Phase
Macular Dystrophy, Corneal Drug: 4-Methylpyrazole Other: saline Phase 1

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:

Further study details as provided by University of Utah:

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

Enrollment: 10
Study Start Date: November 2005
Study Completion Date: September 2006
Primary Completion Date: May 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Drug: 4-Methylpyrazole
15 mg/kg dose
Placebo Comparator: 2
Other: saline


Ages Eligible for Study:   18 Years to 65 Years   (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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00346853

United States, Utah
Moran Eye Center, University of Utah
Salt Lake City, Utah, United States, 84132
Sponsors and Collaborators
University of Utah
Principal Investigator: Paul S Bernstein, M.D., Ph.D. University of Utah
  More Information

Responsible Party: Dr. Randall Olson, Chair, Department of Ophthalmology Identifier: NCT00346853     History of Changes
Other Study ID Numbers: 4-MP Dark Adaptation Inhib.
Study First Received: June 28, 2006
Last Updated: July 9, 2008

Keywords provided by University of Utah:
Dark adaptation inhibition
Stargardt macular dystrophy
4-Methylpyrazole (4-MP)

Additional relevant MeSH terms:
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 processed this record on September 21, 2017