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

NCT01841684 | Terminated Phase 3

• 2 other identifiers: 0859-0422012-002434-37
• Study Type: interventional
• Target: 2
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Hyperlipoproteinemia Type II; Homozygous Familial Hypercholesterolemia

Keywords

HoFH

Outcome Measures

Primary Outcomes (6)

• Percent change from Baseline in Low-density Lipoprotein-Cholesterol (LDL-C) using beta-quantification method

  Baseline and Week 12

• Number of Participants with Alanine Transaminase (ALT) or Aspartate Aminotransferase (AST) Consecutive Elevations ≥3x Upper Limit of Normal (ULN)

  12 weeks

• Number of Participants with Creatine Phosphokinase Elevations ≥10xULN with or without Muscle Symptoms

  12 weeks

• Number of Participants with Sodium, Chloride, or Bicarbonate Elevations >ULN or Potassium Levels <Lower Limit of Normal (LLN)

  12 weeks

• Number of Participants with Pre-specified Adjudicated Cardiovascular Serious Adverse Events or Death from Any Cause

  12 weeks

• Number of Participants with Significant Increase in Blood Pressure

  12 weeks

Secondary Outcomes (2)

• Percent Change from Baseline in High-density Lipoprotein-cholesterol (HDL-C)

  Baseline and Week 12

• Percent Change from Baseline in Apolipoprotein A-I (apoA-I)

  Baseline and Week 12

Study Arms (2)

Anacetrapib

EXPERIMENTAL

Participants receive anacetrapib 100 mg orally once daily for 12 weeks.

Drug: Anacetrapib

Placebo

PLACEBO COMPARATOR

Participants receive placebo orally once daily for 12 weeks.

Drug: Placebo

Interventions

Anacetrapib DRUG

100 mg tablet orally, once daily for 12 weeks

Also known as: MK-0859

Anacetrapib

Placebo DRUG

Placebo for anacetrapib orally, once daily for 12 weeks

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Merck Sharp & Dohme LLC: lead

MeSH Terms

Conditions

Hyperlipoproteinemia Type II; Homozygous Familial Hypercholesterolemia

Interventions

anacetrapib

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

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: April 24, 2013
• First Posted: April 26, 2013
• Study Start: June 1, 2013
• Primary Completion: June 1, 2014
• Study Completion: June 1, 2014
• Last Updated: June 1, 2015

Record last verified: 2015-05