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Efficacy and Tolerability of Anacetrapib Added to Ongoing Lipid-Lowering Therapy in Adult Participants With Homozygous Familial Hypercholesterolemia (HoFH) (MK-0859-042)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01841684
Recruitment Status : Terminated
First Posted : April 26, 2013
Last Update Posted : June 1, 2015
Information provided by (Responsible Party):
Merck Sharp & Dohme Corp.

Brief Summary:
This study will evaluate the safety and effect of anacetrapib on low-density lipoprotein-cholesterol (LDL-C) when added to ongoing lipid-lowering therapy. The primary hypothesis is that treatment with anacetrapib 100 mg for 12 weeks will lower LDL-C to a greater extent than treatment with placebo.

Condition or disease Intervention/treatment Phase
Hyperlipoproteinemia Type II Homozygous Familial Hypercholesterolemia Drug: Anacetrapib Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-Week Study to Assess the Efficacy and Tolerability of Anacetrapib When Added to Ongoing Lipid-Lowering Therapy in Adult Patients With Homozygous Familial Hypercholesterolemia (HoFH)
Study Start Date : June 2013
Actual Primary Completion Date : June 2014
Actual Study Completion Date : June 2014

Arm Intervention/treatment
Experimental: Anacetrapib
Participants receive anacetrapib 100 mg orally once daily for 12 weeks.
Drug: Anacetrapib
100 mg tablet orally, once daily for 12 weeks
Other Name: MK-0859

Placebo Comparator: Placebo
Participants receive placebo orally once daily for 12 weeks.
Drug: Placebo
Placebo for anacetrapib orally, once daily for 12 weeks

Primary Outcome Measures :
  1. Percent change from Baseline in Low-density Lipoprotein-Cholesterol (LDL-C) using beta-quantification method [ Time Frame: Baseline and Week 12 ]
  2. Number of Participants with Alanine Transaminase (ALT) or Aspartate Aminotransferase (AST) Consecutive Elevations ≥3x Upper Limit of Normal (ULN) [ Time Frame: 12 weeks ]
  3. Number of Participants with Creatine Phosphokinase Elevations ≥10xULN with or without Muscle Symptoms [ Time Frame: 12 weeks ]
  4. Number of Participants with Sodium, Chloride, or Bicarbonate Elevations >ULN or Potassium Levels <Lower Limit of Normal (LLN) [ Time Frame: 12 weeks ]
  5. Number of Participants with Pre-specified Adjudicated Cardiovascular Serious Adverse Events or Death from Any Cause [ Time Frame: 12 weeks ]
  6. Number of Participants with Significant Increase in Blood Pressure [ Time Frame: 12 weeks ]

Secondary Outcome Measures :
  1. Percent Change from Baseline in High-density Lipoprotein-cholesterol (HDL-C) [ Time Frame: Baseline and Week 12 ]
  2. Percent Change from Baseline in Apolipoprotein A-I (apoA-I) [ Time Frame: Baseline and Week 12 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosed with HoFH by genotyping
  • If female, cannot be of reproductive potential
  • Have been stabilized on statin monotherapy or statin therapy coadministered

with other lipid medications for at least 6 weeks

Exclusion Criteria:

  • Severe chronic heart failure defined by New York Heart Association

(NYHA) Classes III or IV

  • Uncontrolled cardiac arrhythmias, myocardial infarction (MI), percutaneous coronary intervention (PCI), coronary arterial by-pass graft (CABG), unstable angina or stroke within 3 months prior to Visit 1 or has planned procedures scheduled within first 12 weeks of study
  • Uncontrolled endocrine or metabolic disease known to influence serum

lipids or lipoproteins

  • Active or chronic hepatobiliary or gall bladder disease
  • History of ileal bypass, gastric bypass, or other significant condition

associated with malabsorption

  • Human immunodeficiency virus (HIV) positive
  • Donated blood products or has had phlebotomy of >300 mL within 8 weeks or intends to donate 250_mL of blood products or receive blood products within the projected duration of the study
  • Taking medications that are potent inhibitors or inducers of cytochrome P450 3A4 (CYP3A4), including but not limited to cyclosporine, systemic itraconazole or ketoconazole, erythromycin, clarithromycin, or telithromycin, nefazodone, protease inhibitors, carbamazepine, phenobarbital, phenytoin, rifabutin, rifampin, St John's wort) or has discontinued treatment <3 weeks prior. Consumption of >1 liter of grapefruit juice per day is also prohibited.
  • Currently participating or has participated in a study with an investigational compound or device within 3 months
  • Consume more than 2 alcoholic drinks per day
  • Receiving treatment with systemic corticosteroids or systemic anabolic agents
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Responsible Party: Merck Sharp & Dohme Corp. Identifier: NCT01841684    
Other Study ID Numbers: 0859-042
2012-002434-37 ( EudraCT Number )
First Posted: April 26, 2013    Key Record Dates
Last Update Posted: June 1, 2015
Last Verified: May 2015
Keywords provided by Merck Sharp & Dohme Corp.:
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Anticholesteremic Agents
Hypolipidemic Agents
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents