NCT01584206

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
8

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Apr 2012

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2012

Completed
15 days until next milestone

First Submitted

Initial submission to the registry

April 16, 2012

Completed
8 days until next milestone

First Posted

Study publicly available on registry

April 24, 2012

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2013

Completed
3.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2016

Completed
Last Updated

January 30, 2020

Status Verified

December 1, 2015

Enrollment Period

1.3 years

First QC Date

April 16, 2012

Last Update Submit

January 29, 2020

Conditions

Sitosterolemia

Keywords

SitosterolemiaEzetimibePlant sterolCholesterol

Outcome Measures

Primary Outcomes (2)

  • Plasma cholesterol level

    Plasma cholesterol level will be assessed by GC

    8 months

  • Plasma sitosterol level

    Plasma plant sterol assessment using GC

    8 months

Secondary Outcomes (2)

  • Cholesterol absorption

    4 month

  • Cholesterol synthesis

    4 months

Study Arms (1)

Ezetimibe

EXPERIMENTAL

Compare on and off ezetimibe

Drug: Ezetimibe

Interventions

EzetimibeDRUG

10mg ezetimibe/day, at least 4 months

Ezetimibe

Eligibility Criteria

Age16 Years - 99 Years
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • confirmed diagnosis of homozygous sitosterolemia as established by genotyping and clinical parameters

You may not qualify if:

  • pregnant
  • intellectual disability
  • major infectious diseases
  • immunodeficiency
  • cancer
  • renal failure
  • diabetes

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Richardson Centre for Functional Foods and Nutraceuticals

Winnipeg, Manitoba, R3T 2N2, Canada

Location

Related Publications (2)

  • 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.

    PMID: 28625503DERIVED

  • Othman 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.

    PMID: 25444527DERIVED

MeSH Terms

Conditions

Sitosterolemia

Interventions

Ezetimibe

Intervention Hierarchy (Ancestors)

AzetidinesAzetinesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Study Officials

  • Semone B Myrie, PhD

    University of Manitoba

    PRINCIPAL INVESTIGATOR

  • Peter J Jones, PhD

    University of Manitoba

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
BASIC SCIENCE
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 16, 2012

First Posted

April 24, 2012

Study Start

April 1, 2012

Primary Completion

August 1, 2013

Study Completion

December 1, 2016

Last Updated

January 30, 2020

Record last verified: 2015-12

Locations

CA(1)