NCT00092898

Brief 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. This study investigates whether absorption of these non-cholesterols can be reduced in these patients.

Trial Health

100
On Track

Trial Health Score

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

Enrollment
30

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Oct 2004

Shorter than P25 for phase_3

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

First Submitted

Initial submission to the registry

September 23, 2004

Completed
5 days until next milestone

First Posted

Study publicly available on registry

September 28, 2004

Completed
3 days until next milestone

Study Start

First participant enrolled

October 1, 2004

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2005

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2005

Completed
Last Updated

August 15, 2024

Status Verified

February 1, 2022

Enrollment Period

11 months

First QC Date

September 23, 2004

Last Update Submit

August 12, 2024

Conditions

Lipid Metabolism, Inborn ErrorsHeart Disease

Keywords

Sitosterolemia

Outcome Measures

Primary Outcomes (1)

  • Sitosterol concentrations after 26 weeks.

    After 26 weeks

Secondary Outcomes (1)

  • Campesterol concentrations; Achilles tendon thickness; safety and tolerability.

Interventions

MK0653, ezetimibeDRUG
Comparator: placeboDRUG

Eligibility Criteria

Age18 Years - 85 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • years or older, with a history of elevated sitosterol levels and have received treatment for at least 6 months prior to study entry.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (1)

  • 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.

    PMID: 18484971BACKGROUND

MeSH Terms

Conditions

Lipid Metabolism, Inborn ErrorsHeart DiseasesSitosterolemia

Interventions

Ezetimibe

Condition Hierarchy (Ancestors)

Metabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesCardiovascular Diseases

Intervention Hierarchy (Ancestors)

AzetidinesAzetinesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Study Officials

  • Medical Monitor

    Merck Sharp & Dohme LLC

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 23, 2004

First Posted

September 28, 2004

Study Start

October 1, 2004

Primary Completion

September 1, 2005

Study Completion

October 1, 2005

Last Updated

August 15, 2024

Record last verified: 2022-02