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

NCT ID: NCT00346853

Last Updated: 2020-08-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2005-11-30
• Study Completion Date: 2006-09-30

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.

Conditions

Macular Dystrophy, Corneal

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

1

Group Type: EXPERIMENTAL

4-Methylpyrazole

Intervention Type: DRUG

15 mg/kg dose

2

saline

Group Type: PLACEBO_COMPARATOR

saline

Intervention Type: OTHER

Interventions

4-Methylpyrazole

15 mg/kg dose

Intervention Type: DRUG

saline

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

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

Exclusion Criteria

• Previous ocular pathologies

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

University of Utah

OTHER

Sponsor Role: lead

Responsible Party

Department of Ophthalmology

Principal Investigators

Paul S Bernstein, M.D., Ph.D.

Role: PRINCIPAL_INVESTIGATOR

University of Utah

Locations

Moran Eye Center, University of Utah

Salt Lake City, Utah, United States

Countries

United States

References

Jurgensmeier C, Bhosale P, Bernstein PS. Evaluation of 4-methylpyrazole as a potential therapeutic dark adaptation inhibitor. Curr Eye Res. 2007 Oct;32(10):911-5. doi: 10.1080/02713680701616156.

Reference Type: RESULT

PMID: 17963111 (View on PubMed)

Other Identifiers

4-MP Dark Adaptation Inhib.

Identifier Type: -

Identifier Source: org_study_id