Homozygous Familial Hypercholesterolemia (HoFH)
Conditions
Interventions
Product Name: MK-0859
Product Code: (4S,5R)-5-[3,5-bis(trifluoromethyl)phenyl]-3-{[4’-
Pharmaceutical Form: Tablet
Pharmaceutical form of the placebo: T
Sponsors
Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Patient is male or female and 18 years of age on day of signing informed consent. -Patient is diagnosed with HoFH by genotyping as defined by documented mutations causing familial hypercholesterolemia in both alleles of LDLR including, but not limited to a LDLR null mutation. -A patient who is of reproductive potential agrees to remain abstinent* or use (or have their partner use) 2 acceptable methods of birth control for the duration of the study. -Patients have been stabilized on statin monotherapy or statin therapy coadministered with other lipid medications for at least 6 weeks prior to randomization along with adequate stabilization of LDL apheresis regimen. A new lipid-modifying therapy or apheresis regimen should not be initiated during the first 12 weeks after randomization, but can be initiated at any time during the open-label extension. -Patient provides written informed consent/assent for the trial. Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 45 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: Patient has a triglyceride value >400 mg/dL (6.78 mmol/L), creatine kinase (CK) >2x upper limit of normal (ULN), or alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2x upper limit of normal (ULN) [per central laboratory reference ranges]. -Patient has severe chronic heart failure defined by New York Heart Association (NYHA) Classes III or IV or uncontrolled cardiac arrhythmias, MI, PCI, CABG, unstable angina or stroke within 3 months prior to Visit 1 or has planned procedures scheduled within the first 12-weeks of the study. -Patient has uncontrolled endocrine or metabolic disease known to influence serum lipids or lipoproteins, thyroid stimulating hormone (TSH) values outside the central laboratory normal ranges or chronic hepatobiliary or gall bladder disease. -Patient has eGFR 300 mL within 8 weeks of signing informed consent (excluding apheresis), or intends to donate 250 mL of blood products or receive blood products within the projected duration of the study. - Patient has a history or current evidence of any condition, therapy, lab abnormality or other circumstance that might confound the results of the study, or interfere with the patient’s participation for the full duration of the study, such that it is not in the best interest of the patient to participate. -Patient is currently taking medications that are potent inhibitors or inducers of CYP3A4 or has discontinued treatment 1 liter of grapefruit juice per day is also prohibited. -Patient is currently participating or has participated in a study with an investigational compound or device within 3 months of signing informed consent. -Patient consumes more than 2 alcoholic drinks per day. - Patient is receiving treatment with systemic corticosteroids or systemic anabolic agents.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: In adults with HoFH on a stable dose of their regular medications, evaluate the effects of adding anacetrapib 100 mg for 12 weeks relative to placebo on plasma concentrations of bad cholesterol summarize the safety and tolerability of 12 weeks of treatment ; Secondary Objective: In adults with HoFH on a stable dose of their regular medications, evaluate the effects of anacetrapib 100 mg on bad and good cholesterol relative to placebo after 12 weeks of treatment and summarize the safety and tolerability of up to 64 weeks of treatment with anacetrapib 100 mg. ;Primary end point(s): The primary efficacy endpoint will be percent change in LDL-C from baseline (randomization) to week 12 measured by BL.;Timepoint(s) of evaluation of this end point: 12 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Effects of adding anacetrapib 100 mg to ongoing lipid therapy relative to placebo on plasma concentrations of HDL-C and apoA-1;Timepoint(s) of evaluation of this end point: 12 (HDL-C and apoA-1) & 64 (safety)weeks | — |
Countries
Australia, Canada, Czech Republic, Israel, Italy, Malaysia, Norway, South Africa, United Kingdom, United States
Contacts
Public ContactGlobal Clinical Trials Operations
Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.
Outcome results
None listed