Phase 3
N=306
Study to Assess the Tolerability and Efficacy of Anacetrapib (MK-0859) Co-Administered With Statin in Participants With Heterozygous Familial Hypercholesterolemia (MK-0859-020)
Hyperlipoproteinemia Type II · Hypercholesterolemia, Familial
Bottom Line
View on ClinicalTrials.gov: NCT01524289 ↗Enrolled (actual)
306
Serious AEs
5.5%
Results posted
Aug 2019
Primary outcome: Primary: Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) - Treatment Phase — -36.0; 3.7 Percent Change — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Anacetrapib (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Merck Sharp & Dohme LLC
- Primary completion
- Feb 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) - Treatment Phase |
-36.0; 3.7 | <0.001 sig |
| PRIMARY Percentage of Participants With Any Adverse Event - Treatment Phase |
76.4; 78.4 | — |
| PRIMARY Percentage of Participants With Any Treatment-Related Adverse Event - Treatment Phase |
18.2; 13.7 | — |
| PRIMARY Percentage of Participants With Any Serious Adverse Event - Treatment Phase |
8.9; 9.8 | — |
| PRIMARY Percentage of Participants Discontinuing Study Treatment Due to an Adverse Event - Treatment Phase |
5.9; 4.9 | — |
| PRIMARY Percentage of Participants With Changes in Systolic Blood Pressure (SBP) >= 10 mm Hg |
45.0; 53.5 | 0.168 |
| PRIMARY Percentage of Participants With Changes in SBP >= 15 mm Hg |
26.2; 33.7 | 0.179 |
| PRIMARY Percentage of Participants With Changes in Diastolic Blood Pressure (DBP) >= 10 mm Hg |
22.8; 36.6 | 0.011 sig |
| PRIMARY Percentage of Participants With Sodium Levels > Upper Limit of Normal (ULN) |
11.4; 9.9 | 0.696 |
| PRIMARY Percentage of Participants With Chloride Levels > ULN |
0.5; 0.0 | 0.480 |
| PRIMARY Percentage of Participants With Potassium Levels < Lower Limit of Normal (LLN) |
1.5; 1.0 | 0.722 |
| PRIMARY Percentage of Participants With Bicarbonate Levels > ULN |
0.0; 0.0 | >0.999 |
| PRIMARY Percentage of Participants With Consecutive Changes in Alanine Aminotransferase (ALT) and/or Aspartate Aminotransferase (AST) of >=3 x ULN |
1.5; 1.0 | 0.722 |
| PRIMARY Percentage of Participants With Creatine Kinase (CK) Level >=10 x ULN |
0.0; 1.0 | 0.157 |
| PRIMARY Percentage of Participants With CK Level >=10 x ULN With Muscle Spasms |
0.0; 1.0 | 0.157 |
| PRIMARY Percentage of Participants Adjudicated Cardiovascular (CV) SAE |
1.5; 0.0 | 0.218 |
| PRIMARY Percentage of Participants Who Died From Any Cause - Treatment Phase |
0.0; 0.0 | >0.999 |
| SECONDARY Percent Change From Baseline in High-Density Lipoprotein Cholesterol Levels |
105.8; 3.7 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol Levels |
-32.0; 4.4 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Apolipoprotein (Apo) B Levels |
-19.6; 5.2 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Apolipoprotein (Apo) A-1 Levels |
35.8; 2.9 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Lipoprotein(a) (Lp[a]) Levels |
-31.8; 0.0 | <0.001 sig |
Summary
The objective of this study is to evaluate the efficacy and tolerability of adding anacetrapib to ongoing statin therapy in participants with heterozygous familial hypercholesterolemia (HeFH).
Eligibility Criteria
Inclusion Criteria
- If of reproductive potential, must agree to remain abstinent or use (or have their partner use) 2 acceptable methods of birth control for the duration of the study
- Diagnosed with Heterozygous Familial Hypercholesterolemia (HeFH)
- Have been treated with an optimal dose of statin for at least 6 weeks
Exclusion Criteria
- Received treatment with low-density lipoprotein (LDL) apheresis within 4 weeks of screening or expect to undergo treatment with LDL apheresis during the course of the study
- Homozygous familial hypercholesterolemia
- Severe chronic heart failure
- Uncontrolled hypertension
- Uncontrolled cardiac arrhythmias, myocardial infarction (MI), percutaneous coronary intervention (PCI), coronary artery bypass graft (CABG), unstable angina, or stroke within 3 months
- Uncontrolled endocrine or metabolic disease known to influence serum lipids or lipoproteins
- Active or chronic hepatobiliary, hepatic, or gall bladder disease
- Pregnant or breast-feeding, or plans to become pregnant during the study or within 2 years after stopping study medication
- History of ileal bypass, gastric bypass, or other significant condition associated with malabsorption
- Human immunodeficiency virus (HIV) positive
- History of malignancy ≤5 years
- 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
- Currently taking medications that are potent inhibitors or inducers of cytochrome P450 3A4 (CYP3A) (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
- Consumes more than 2 alcoholic drinks per day
- Currently participating or has participated in a study with an investigational compound or device within 3 months
- Receiving treatment with systemic corticosteroids or taking systemic anabolic agents
Data sourced from ClinicalTrials.gov (NCT01524289). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.