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

NCT00346853 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2020-08-05

No results posted yet for this study

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.

Conditions

  • Macular Dystrophy, Corneal

Interventions

DRUG

4-Methylpyrazole

15 mg/kg dose

OTHER

saline

Sponsors & Collaborators

Principal Investigators

  • Paul S Bernstein, M.D., Ph.D. · University of Utah

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2005-11-30
Primary Completion
2006-05-31
Completion
2006-09-30

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00346853 on ClinicalTrials.gov