An Investigational Drug Study to Lower Non-Cholesterol Sterol Levels Associated With Sitosterolemia - Full Text View

Sponsored by:	Merck
Information provided by:	Merck
ClinicalTrials.gov Identifier:	NCT00092898

Purpose

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.

Condition	Intervention	Phase
Lipid Metabolism, Inborn Errors Heart Disease	Drug: MK0653, ezetimibe Drug: Comparator: placebo	Phase III

Genetics Home Reference related topics: cholesteryl ester storage disease Farber lipogranulomatosis long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency mitochondrial trifunctional protein deficiency primary carnitine deficiency

MedlinePlus related topics: Heart Diseases

Drug Information available for: Ezetimibe

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double Blind (Subject, Investigator), Placebo Control, Parallel Assignment
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

Further study details as provided by Merck:

Primary Outcome Measures:

Sitosterol concentrations after 26 weeks. [ Time Frame: After 26 weeks ]

Secondary Outcome Measures:

Campesterol concentrations; Achilles tendon thickness; safety and tolerability.

Enrollment:	30
Study Start Date:	November 2004
Study Completion Date:	March 2006
Primary Completion Date:	October 2005 (Final data collection date for primary outcome measure)

Detailed Description:

The duration of treatment is 26 weeks.

Eligibility

Ages Eligible for Study:	18 Years to 85 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00092898

Sponsors and Collaborators

Merck

Investigators

Study Director:

Medical Monitor

Merck

More Information

Publications:

Musliner T, Cselovszky D, Sirah W, McCrary Sisk C, Sapre A, Salen G, Lütjohann 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. Epub 2008 May 14.

Responsible Party:	Merck & Co., Inc. ( Executive Vice President, Clinical and Quantitative Sciences )
Study ID Numbers:	2004_038, MK0653-062
Study First Received:	September 23, 2004
Last Updated:	September 24, 2008
ClinicalTrials.gov Identifier:	NCT00092898
Health Authority:	United States: Food and Drug Administration

Keywords provided by Merck:

Sitosterolemia

Study placed in the following topic categories:

Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Sitosterolemia
Metabolic Diseases
Heart Diseases

Genetic Diseases, Inborn
Ezetimibe
Metabolic disorder
Lipid Metabolism Disorders

Additional relevant MeSH terms:

Antimetabolites
Molecular Mechanisms of Pharmacological Action
Therapeutic Uses
Antilipemic Agents

Cardiovascular Diseases
Anticholesteremic Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 14, 2009