Macular Dystrophy, CornealPhase 1NCT00346853
Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy
A Phase 1 study testing 4-Methylpyrazole and saline in people with Macular Dystrophy, Corneal, run by University of Utah at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Macular Dystrophy, Corneal
- What is being tested?
- 4-Methylpyrazole and saline
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2005to Sep 2006
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- All nonpregnant, nonlactating adults with normal vision in both eyes
Who cannot join
- Previous ocular pathologies
Trial locations and contacts
1 site in 1 country.
United States1 site
Moran Eye Center, University of Utah
Salt Lake City, Utah, 84132
About this study
Official title: Clinical Interventions Against Stargardt Macular Dystrophy: Phase 1 Pilot Study of 4-MP as an Inhibitor of Dark Adaptation
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
- Being tested
- 4-Methylpyrazole, saline
- Keywords
- Dark adaptation inhibition, Stargardt macular dystrophy, 4-Methylpyrazole (4-MP), Antizol, Fomepizole
- Medical subject headings
- Corneal Dystrophies, Hereditary, Stargardt Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
4-MethylpyrazoleDrug
15 mg/kg dose
salineOther
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Not described Receives: Drug: 4-Methylpyrazole |
| 2 | Placebo comparator | saline Receives: Other: saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dark adaptation inhibition measured 30 minutes after drug infusion using Goldman-Weeker adaptometer.
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2006 (estimated)
- Study startNov 2005
- Primary completionMay 2006
- Study completionSep 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Macular Dystrophy, CornealStudies of the same condition, open and under way.
- More trials run by University of UtahThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Utah
- Sponsor type
- University, hospital or other
- Investigators
- Paul S Bernstein, M.D., Ph.D., Principal investigator, University of Utah
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00346853
- Sponsor's protocol ID
- 4-MP Dark Adaptation Inhib.
- Status last verified
- Aug 2020
- First submitted
- 28 Jun 2006
- Last updated
- 5 Aug 2020
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 17963111
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Interventions Against Stargardt Macular Dystrophy: Phase 1 Pilot Study of 4-MP as an Inhibitor of Dark Adaptation
- ClinicalTrials.gov ID
- NCT00346853
- Acronym / sponsor ID
- 4-MP Dark Adaptation Inhib.
- Conditions
- Macular Dystrophy, Corneal
- Keywords
- Dark adaptation inhibition, Stargardt macular dystrophy, 4-Methylpyrazole (4-MP), Antizol, Fomepizole
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University of UtahUniversity, hospital or other
- Study start
- Nov 2005
- Primary completion
- May 2006 (actual)
- Study completion
- Sep 2006 (actual)
- First posted
- 30 Jun 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: 4-Methylpyrazole |
| 2 | Placebo comparator | Other: salinesaline |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- All nonpregnant, nonlactating adults with normal vision in both eyes
Key exclusion criteria (1 listed)
- Previous ocular pathologies
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 4-Methylpyrazole15 mg/kg dose | Drug | 1 |
| saline | Other | 2 |
Primary outcome
Dark adaptation inhibition measured 30 minutes after drug infusion using Goldman-Weeker adaptometer.
Time frame: 6 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2020.
- Results publication: PubMed 17963111Jurgensmeier 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Paul S Bernstein, M.D., Ph.D. principal investigator
University of Utah
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 4-Methylpyrazole and saline in people with Macular Dystrophy, Corneal, run by University of Utah at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dark adaptation inhibition measured 30 minutes after drug infusion using Goldman-Weeker adaptometer. (time frame: 6 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00346853, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Aug 2020.