Lipid Metabolism, Inborn ErrorsPhase 3NCT00092898
An Investigational Drug Study to Lower Non-Cholesterol Sterol Levels Associated With Sitosterolemia (0653-062)(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 a 6-month study with patients who have the rare disease, sitosterolemia which may result in heart-related diseases. These patients have unusually high absorption of non-cholesterol sterols, resulting in heart-related diseases. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- 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
- 30 participants
- Where
- No sites listed yet
- Study dates
- Oct 2004to Oct 2005
- 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 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 18 years or older, with a history of elevated sitosterol levels and have received treatment for at least 6 months prior to study entry.
Who cannot join
- Patient has a condition which, in the opinion of the investigator, might pose a risk to the patient or interfere with participation in the study.
About this study
Official title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of Adding Ezetimibe 30 mg to An Ongoing Regimen of Ezetimibe 10 mg in Patients Homozygous Sitosterolemia
This is a 6-month study with patients who have the rare disease, sitosterolemia which may result in heart-related diseases. These patients have unusually high absorption of non-cholesterol sterols, resulting in heart-related diseases. This study investigates whether absorption of these non-cholesterols can be reduced in these patients.
Read the detailed description
The duration of treatment is 26 weeks.
- Conditions
- Lipid Metabolism, Inborn Errors, Heart Disease
- Being tested
- MK0653, ezetimibe, Comparator: placebo
- Keywords
- Sitosterolemia
- Medical subject headings
- Lipid Metabolism, Inborn Errors, Heart Diseases, Sitosterolemia
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.
Sitosterol concentrations after 26 weeks.
Measured: After 26 weeks
Secondary outcomes (1)
Campesterol concentrations; Achilles tendon thickness; safety and tolerability.
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 startOct 2004
- Primary completionSep 2005
- Study completionOct 2005
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
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 30 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
- NCT00092898
- Sponsor's protocol ID
- 0653-062
- Other IDs
- MK0653-062, 2004_038
- Status last verified
- Feb 2022
- First submitted
- 23 Sep 2004
- Last updated
- 15 Aug 2024
- Results posted
- No
Publications
Musliner T, Cselovszky D, Sirah W, McCrary Sisk C, Sapre A, Salen G, Lutjohann D, von Bergmann K. Efficacy and safety of ezetimibe 40 mg vs. ezetimibe 10 mg in the treatment of patients with homozygous sitosterolaemia. Int J Clin Pract. 2008 Jul;62(7):995-1000. doi: 10.1111/j.1742-1241.2008.01786.x. Epub 2008 May 14.
PubMed 18484971
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 Efficacy and Safety of Adding Ezetimibe 30 mg to An Ongoing Regimen of Ezetimibe 10 mg in Patients Homozygous Sitosterolemia
- ClinicalTrials.gov ID
- NCT00092898
- Acronym / sponsor ID
- 0653-062
- Conditions
- Lipid Metabolism, Inborn Errors, Heart Disease
- Keywords
- Sitosterolemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Organon and CoIndustry (pharma, biotech, devices)
- Study start
- Oct 2004
- Primary completion
- Sep 2005 (actual)
- Study completion
- Oct 2005 (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 a 6-month study with patients who have the rare disease, sitosterolemia which may result in heart-related diseases. These patients have unusually high absorption of non-cholesterol sterols, resulting in heart-related diseases. This study investigates whether absorption of these non-cholesterols can be reduced in these patients.
Detailed description
The duration of treatment is 26 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- 18 years or older, with a history of elevated sitosterol levels and have received treatment for at least 6 months prior to study entry.
Key exclusion criteria (1 listed)
- Patient has a condition which, in the opinion of the investigator, might pose a risk to the patient or interfere with participation in 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
Sitosterol concentrations after 26 weeks.
Time frame: After 26 weeks
1 secondary outcome
Campesterol concentrations; Achilles tendon thickness; safety and tolerability.
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 18484971Musliner T, Cselovszky D, Sirah W, McCrary Sisk C, Sapre A, Salen G, Lutjohann D, von Bergmann K. Efficacy and safety of ezetimibe 40 mg vs. ezetimibe 10 mg in the treatment of patients with homozygous sitosterolaemia. Int J Clin Pract. 2008 Jul;62(7):995-1000. doi: 10.1111/j.1742-1241.2008.01786.x. Epub 2008 May 14.
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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?
Sitosterol concentrations after 26 weeks. (time frame: After 26 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 NCT00092898, 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.