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

Phase 1

Completed

• Conditions: Macular Dystrophy, Corneal
• Interventions: Other: saline; Drug: 4-Methylpyrazole

• Registration Number: NCT00346853
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2005-11
• Primary Completion: 2006-05
• Study First Submitted: 2006-06-28
• Study First Submitted QC: 2006-06-28
• Study First Posted: 2006-06-30
• Study Completion: 2006-09
• Status Verified: 2020-08
• Last Update Submitted: 2020-08-03
• Last Update Posted: 2020-08-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

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

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Exclusion Criteria

• Previous ocular pathologies

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
2	saline	saline
1	4-Methylpyrazole	-

Primary Outcome Measures

Name	Time	Method
Dark adaptation inhibition measured 30 minutes after drug infusion using Goldman-Weeker adaptometer.	6 weeks

Trial Locations

Locations (1)

Moran Eye Center, University of Utah; 🇺🇸 Salt Lake City, Utah, United States