SitosterolemiaSTAIR7002NCT01584206
Sitosterolemia Metabolism
A study testing Ezetimibe in people with Sitosterolemia, run by University of Manitoba at 1 site in Canada.
Trial summary
Ezetimibe has become the treatment choice for patients with sitosterolemia. Ezetimibe is an inhibitor of cholesterol absorption from the gastrointestinal tract. The purpose of this study is to determine if ezetimibe improves whole body plant sterol and cholesterol homeostasis. Read more about this study
- Who is it for?
- Ages 16 to 99 yearsHealthy volunteers can take part
- Conditions the study is for
- Sitosterolemia
- What is being tested?
- Ezetimibe
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteCanada
- Study dates
- Apr 2012to Dec 2016
- Phase
- Interventional
- 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 16 to 99 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- confirmed diagnosis of homozygous sitosterolemia as established by genotyping and clinical parameters
Who cannot join
- pregnant
- intellectual disability
- major infectious diseases
- immunodeficiency
- cancer
- renal failure
- diabetes
Trial locations and contacts
1 site in 1 country.
Canada1 site
Richardson Centre for Functional Foods and Nutraceuticals
Winnipeg, Manitoba, R3T 2N2
About this study
Official title: Assessment of Sterol Metabolism in Sitosterolemia: A Pilot Study of Patients Treated With Ezetimibe
Ezetimibe has become the treatment choice for patients with sitosterolemia. Ezetimibe is an inhibitor of cholesterol absorption from the gastrointestinal tract. The purpose of this study is to determine if ezetimibe improves whole body plant sterol and cholesterol homeostasis.
- Conditions
- Sitosterolemia
- Being tested
- Ezetimibe
- Keywords
- Sitosterolemia, Ezetimibe, Plant sterol, Cholesterol
- Medical subject headings
- Sitosterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EzetimibeDrug
10mg ezetimibe/day, at least 4 months
| Study group | Role | What they receive |
|---|---|---|
| Ezetimibe | Experimental | Compare on and off ezetimibe Receives: Drug: Ezetimibe |
What is being measured
The primary outcome is the main question the study is designed to answer.
Plasma cholesterol level
Plasma cholesterol level will be assessed by GC
Measured: 8 months
Plasma sitosterol level
Plasma plant sterol assessment using GC
Measured: 8 months
Secondary outcomes (2)
Cholesterol absorption
Use stable isotope technique to assess cholesterol absorption
Measured: 4 month
Cholesterol synthesis
Use of stable isotope to assess cholesterol synthesis.
Measured: 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Apr 2012 (estimated)
- Study startApr 2012
- Primary completionAug 2013
- Study completionDec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for SitosterolemiaStudies 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 N/A
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Manitoba
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Semone B Myrie, PhD, Principal investigator, University of ManitobaPeter J Jones, PhD, Study chair, University of Manitoba
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01584206
- Sponsor's protocol ID
- B2011:051
- Acronym
- STAIR7002
- Status last verified
- Dec 2015
- First submitted
- 16 Apr 2012
- Last updated
- 30 Jan 2020
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Othman RA, Myrie SB, Mymin D, Roullet JB, DeBarber AE, Steiner RD, Jones PJH. Thyroid Hormone Status in Sitosterolemia Is Modified by Ezetimibe. J Pediatr. 2017 Sep;188:198-204.e1. doi: 10.1016/j.jpeds.2017.05.049. Epub 2017 Jun 16.
PubMed 28625503Othman RA, Myrie SB, Mymin D, Merkens LS, Roullet JB, Steiner RD, Jones PJ. Ezetimibe reduces plant sterol accumulation and favorably increases platelet count in sitosterolemia. J Pediatr. 2015 Jan;166(1):125-31. doi: 10.1016/j.jpeds.2014.08.069. Epub 2014 Oct 16.
PubMed 25444527
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Sterol Metabolism in Sitosterolemia: A Pilot Study of Patients Treated With Ezetimibe
- ClinicalTrials.gov ID
- NCT01584206
- Acronym / sponsor ID
- STAIR7002 / B2011:051
- Conditions
- Sitosterolemia
- Keywords
- Sitosterolemia, Ezetimibe, Plant sterol, Cholesterol
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- University of ManitobaUniversity, hospital or other
- Study start
- Apr 2012
- Primary completion
- Aug 2013 (actual)
- Study completion
- Dec 2016 (actual)
- First posted
- 24 Apr 2012 (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.
Ezetimibe has become the treatment choice for patients with sitosterolemia. Ezetimibe is an inhibitor of cholesterol absorption from the gastrointestinal tract. The purpose of this study is to determine if ezetimibe improves whole body plant sterol and cholesterol homeostasis.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ezetimibe | Experimental | Drug: EzetimibeCompare on and off ezetimibe |
Who may qualify
- Ages
- 16 to 99 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- confirmed diagnosis of homozygous sitosterolemia as established by genotyping and clinical parameters
Key exclusion criteria (7 listed)
- pregnant
- intellectual disability
- major infectious diseases
- immunodeficiency
- cancer
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 |
|---|---|---|
| Ezetimibe10mg ezetimibe/day, at least 4 months | Drug | Ezetimibe |
Primary outcome
Plasma cholesterol level
Time frame: 8 months
Plasma cholesterol level will be assessed by GC
Plasma sitosterol level
Time frame: 8 months
Plasma plant sterol assessment using GC
2 secondary outcomes
Cholesterol absorption
Time frame: 4 month
Cholesterol synthesis
Time frame: 4 months
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 30 Jan 2020.
- Derived from this study: PubMed 28625503Othman RA, Myrie SB, Mymin D, Roullet JB, DeBarber AE, Steiner RD, Jones PJH. Thyroid Hormone Status in Sitosterolemia Is Modified by Ezetimibe. J Pediatr. 2017 Sep;188:198-204.e1. doi: 10.1016/j.jpeds.2017.05.049. Epub 2017 Jun 16.
- Derived from this study: PubMed 25444527Othman RA, Myrie SB, Mymin D, Merkens LS, Roullet JB, Steiner RD, Jones PJ. Ezetimibe reduces plant sterol accumulation and favorably increases platelet count in sitosterolemia. J Pediatr. 2015 Jan;166(1):125-31. doi: 10.1016/j.jpeds.2014.08.069. Epub 2014 Oct 16.
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.
Semone B Myrie, PhD principal investigator
University of Manitoba
Peter J Jones, PhD study chair
University of Manitoba
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Ezetimibe in people with Sitosterolemia, run by University of Manitoba at 1 site in Canada.
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 7 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 30 Jan 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 Canada. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
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?
Plasma cholesterol level (time frame: 8 months). The study lists 2 primary outcomes in all. 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 NCT01584206, which also lists 2 linked publications.
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 30 Jan 2020.