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)
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 2
- 主要终点
- Percent change from Baseline in Low-density Lipoprotein-Cholesterol (LDL-C) using beta-quantification method
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Anacetrapib
Participants receive anacetrapib 100 mg orally once daily for 12 weeks.
干预措施: Anacetrapib (Drug)
Placebo
Participants receive placebo orally once daily for 12 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Percent change from Baseline in Low-density Lipoprotein-Cholesterol (LDL-C) using beta-quantification method
时间窗: Baseline and Week 12
Number of Participants with Alanine Transaminase (ALT) or Aspartate Aminotransferase (AST) Consecutive Elevations ≥3x Upper Limit of Normal (ULN)
时间窗: 12 weeks
Number of Participants with Creatine Phosphokinase Elevations ≥10xULN with or without Muscle Symptoms
时间窗: 12 weeks
Number of Participants with Sodium, Chloride, or Bicarbonate Elevations >ULN or Potassium Levels <Lower Limit of Normal (LLN)
时间窗: 12 weeks
Number of Participants with Pre-specified Adjudicated Cardiovascular Serious Adverse Events or Death from Any Cause
时间窗: 12 weeks
Number of Participants with Significant Increase in Blood Pressure
时间窗: 12 weeks
次要结局
- Percent Change from Baseline in Apolipoprotein A-I (apoA-I)(Baseline and Week 12)
- Percent Change from Baseline in High-density Lipoprotein-cholesterol (HDL-C)(Baseline and Week 12)