Sitosterolemia Extension Study - Full Text View

Purpose

This is an extension study for patients having unusually high absorption of non-cholesterol sterols, resulting in heart-related diseases. This study will evaluate the long term safety and the ability to lower cholesterol levels with an investigational drug.

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

ChemIDplus related topics:

Ezetimibe

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Safety Study

Official Title:	A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of MK-0653 When Added to Current Regimen in Patients With Homozygous Sitosterolemia

Further study details as provided by Merck:

Primary Outcome Measures:

Plasma sitosterol levels after 52 weeks.

Secondary Outcome Measures:

Plasma LDL-C and plasma campesterol. Safety and tolerability.

Estimated Enrollment:	25
Study Start Date:	November 2001

Detailed Description:

The duration of treatment is 52 weeks.

Eligibility

Ages Eligible for Study:	10 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Patients with elevated sitosterol levels while taking current medication

Exclusion Criteria:

Patient has a condition which, in the opinion of the investigator, might pose a risk to the patient, interfere with participation in the study, or does not meet the additional criteria as required by the study.

Contacts and Locations

No Contacts or Locations Provided

More Information

Publications:

Salen G, von Bergmann K, Lutjohann D, Kwiterovich P, Kane J, Patel SB, Musliner T, Stein P, Musser B; Multicenter Sitosterolemia Study Group. Ezetimibe effectively reduces plasma plant sterols in patients with sitosterolemia. Circulation. 2004 Mar 2;109(8):966-71. Epub 2004 Feb 09.

Study ID Numbers:	2004_033
First Received:	September 23, 2004
Last Updated:	June 2, 2006
ClinicalTrials.gov Identifier:	NCT00092820
Health Authority:	United States: Food and Drug Administration

Keywords provided by Merck:

Homozygous 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:

Cardiovascular Diseases

ClinicalTrials.gov processed this record on October 09, 2008

Sponsored by:	Merck
Information provided by:	Merck
ClinicalTrials.gov Identifier:	NCT00092820