Lipid Metabolism, Inborn ErrorsPhase 3NCT00092807
Sitosterolemia Extension Study (0653-003)(COMPLETED)
A Phase 3 study testing MK0653, ezetimibe in people with Lipid Metabolism, Inborn Errors and Heart Disease, run by Organon and Co.
Trial summary
This is an extension study for patients having unusually high absorption of non-cholesterol sterols, resulting in heart-related diseases. This study will evaluate the long term safety and the ability to lower cholesterol levels with an investigational drug. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Lipid Metabolism, Inborn Errors, Heart Disease
- What is being tested?
- MK0653, ezetimibe
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 37 participants
- Where
- No sites listed yet
- Study dates
- 12 Feb 2001to 30 Nov 2004
- Phase
- Phase 3
- 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 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with elevated sitosterol levels while taking current medication
Who cannot join
- Patient has a condition which, in the opinion of the investigator, might pose a risk to the patient, interfere with participation in the study, or does not meet the additional criteria as required by the study.
About this study
Official title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of MK0653 When Added to Current Regimen in Patients With Homozygous Sitosterolemia - 1 Year Open-Label Extension
This is an extension study for patients having unusually high absorption of non-cholesterol sterols, resulting in heart-related diseases. This study will evaluate the long term safety and the ability to lower cholesterol levels with an investigational drug.
Read the detailed description
The duration of treatment is 52 weeks.
- Conditions
- Lipid Metabolism, Inborn Errors, Heart Disease
- Being tested
- MK0653, ezetimibe, Comparator: placebo
- Keywords
- Homozygous Sitosterolemia
- Medical subject headings
- Lipid Metabolism, Inborn Errors, Heart Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MK0653, ezetimibeDrug
Comparator: placeboDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the percent change relative to baseline in plasma sitosterol concentration after long-term treatment with MK0653 10 mg/day.
Secondary outcomes (1)
Assess the percent change relative to baseline in LDL-C/plasma campesterol concentrations, and safety and tolerability after long-term treatment with MK0653 10 mg/day in patients with homozygous sitosterolemia
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2004 (estimated)
- Study start12 Feb 2001
- Primary completion18 Sep 2001
- Study completion30 Nov 2004
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 Lipid Metabolism, Inborn ErrorsStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Treatment
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Organon and Co
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Merck Sharp & Dohme LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00092807
- Sponsor's protocol ID
- P02257
- Other IDs
- 2004_032
- Status last verified
- Feb 2022
- First submitted
- 23 Sep 2004
- Last updated
- 15 Aug 2024
- Results posted
- No
Publications
Salen G, von Bergmann K, Lutjohann D, Kwiterovich P, Kane J, Patel SB, Musliner T, Stein P, Musser B; Multicenter Sitosterolemia Study Group. Ezetimibe effectively reduces plasma plant sterols in patients with sitosterolemia. Circulation. 2004 Mar 2;109(8):966-71. doi: 10.1161/01.CIR.0000116766.31036.03. Epub 2004 Feb 9.
PubMed 14769702
Study at a glance
From the registry record, last updated by the sponsor on 15 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of MK0653 When Added to Current Regimen in Patients With Homozygous Sitosterolemia - 1 Year Open-Label Extension
- ClinicalTrials.gov ID
- NCT00092807
- Acronym / sponsor ID
- P02257
- Conditions
- Lipid Metabolism, Inborn Errors, Heart Disease
- Keywords
- Homozygous Sitosterolemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Organon and CoIndustry (pharma, biotech, devices)
- Study start
- 12 Feb 2001 (actual)
- Primary completion
- 18 Sep 2001 (actual)
- Study completion
- 30 Nov 2004 (actual)
- First posted
- 28 Sep 2004 (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.
This is an extension study for patients having unusually high absorption of non-cholesterol sterols, resulting in heart-related diseases. This study will evaluate the long term safety and the ability to lower cholesterol levels with an investigational drug.
Detailed description
The duration of treatment is 52 weeks.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Patients with elevated sitosterol levels while taking current medication
Key exclusion criteria (1 listed)
- Patient has a condition which, in the opinion of the investigator, might pose a risk to the patient, interfere with participation in the study, or does not meet the additional criteria as required by the study.
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 |
|---|---|---|
| MK0653, ezetimibe | Drug | – |
| Comparator: placebo | Drug | – |
Primary outcome
To assess the percent change relative to baseline in plasma sitosterol concentration after long-term treatment with MK0653 10 mg/day.
1 secondary outcome
Assess the percent change relative to baseline in LDL-C/plasma campesterol concentrations, and safety and tolerability after long-term treatment with MK0653 10 mg/day in patients with homozygous sitosterolemia
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 15 Aug 2024.
- Background: PubMed 14769702Salen G, von Bergmann K, Lutjohann D, Kwiterovich P, Kane J, Patel SB, Musliner T, Stein P, Musser B; Multicenter Sitosterolemia Study Group. Ezetimibe effectively reduces plasma plant sterols in patients with sitosterolemia. Circulation. 2004 Mar 2;109(8):966-71. doi: 10.1161/01.CIR.0000116766.31036.03. Epub 2004 Feb 9.
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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.
Medical Monitor study director
Merck Sharp & Dohme LLC
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing MK0653, ezetimibe in people with Lipid Metabolism, Inborn Errors and Heart Disease, run by Organon and Co.
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 15 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
To assess the percent change relative to baseline in plasma sitosterol concentration after long-term treatment with MK0653 10 mg/day.. 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 NCT00092807, 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 15 Aug 2024.