Sitosterolemia Extension Study (0653-004)(COMPLETED)

• ClinicalTrials.gov Identifier: NCT00092820
• Recruitment Status: Completed
• First Posted: September 28, 2004
• Last Update Posted: May 11, 2017
• Sponsor: Merck Sharp & Dohme Corp.
• Information provided by (Responsible Party): Merck Sharp & Dohme Corp.

Study Description

Brief Summary:

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 or disease	Intervention/treatment	Phase
Lipid Metabolism, Inborn Errors; Heart Disease	Drug: MK0653, ezetimibe; Drug: Comparator: placebo	Phase 3

Detailed Description:

The duration of treatment is 52 weeks.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 58 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of MK0653 When Added to Current Regimen in Patients With Homozygous Sitosterolemia
• Actual Study Start Date: February 12, 2001
• Actual Primary Completion Date: September 18, 2001
• Actual Study Completion Date: November 30, 2004

Arms and Interventions

Outcome Measures

Primary Outcome Measures :

1. Plasma sitosterol levels after 52 weeks.

Secondary Outcome Measures :

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

Eligibility Criteria

• Ages Eligible for Study: 10 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• 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

Sponsors and Collaborators

Merck Sharp & Dohme Corp.

Investigators

• Study Director: Medical Monitor; Merck Sharp & Dohme Corp.

More Information

Study Data/Documents: CSR Synopsis 

Publications:

Salen G, von Bergmann K, Lütjohann 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 9.

Lütjohann D, von Bergmann K, Sirah W, Macdonell G, Johnson-Levonas AO, Shah A, Lin J, Sapre A, Musliner T. Long-term efficacy and safety of ezetimibe 10 mg in patients with homozygous sitosterolemia: a 2-year, open-label extension study. Int J Clin Pract. 2008 Oct;62(10):1499-510. doi: 10.1111/j.1742-1241.2008.01841.x.

• Responsible Party: Merck Sharp & Dohme Corp.
• ClinicalTrials.gov Identifier: NCT00092820 History of Changes
• Other Study ID Numbers: P02243; 2004_033
• First Posted: September 28, 2004
• Last Update Posted: May 11, 2017
• Last Verified: May 2017

Keywords provided by Merck Sharp & Dohme Corp.:

Homozygous Sitosterolemia

Additional relevant MeSH terms:

Heart Diseases; Metabolism, Inborn Errors; Lipid Metabolism, Inborn Errors; Cardiovascular Diseases; Genetic Diseases, Inborn; Metabolic Diseases; Lipid Metabolism Disorders; Ezetimibe; Anticholesteremic Agents; Hypolipidemic Agents; Antimetabolites; Molecular Mechanisms of Pharmacological Action; Lipid Regulating Agents