A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 480
- 试验地点
- 4
- 主要终点
- Percent change from Baseline in low-density lipoprotein cholesterol (LDL-C) in Cohort 1
研究概览
简要总结
This study is a phase 3, randomized, double-blind,placebo-controlled, parallel-group study to assess the safety and efficacy oftwo different regimens of mipomersen in patients with familialhypercholesterolemia and inadequately controlled low-density lipoproteincholesterol. This study will be conducted in about 30 countries globally,enrolling 480 patients. From India,40 patients will be enrolled in to the study at 4 centers.
Theprimary objective of this study is to determine whether mipomersen (ISIS301012) significantly reduces atherogenic lipid levels in patients with severeheterozygous familial hypercholesterolemia (severe HeFH), defined aslow-density lipoprotein cholesterol (LDL-C) levels ≥200 mg/dL plus the presenceof coronary heart disease (CHD)/risk equivalents or LDL-C levels ≥300 mg/dLregardless of the presence of CHD/risk equivalents compared to placebo. Twodifferent mipomersen dosing regimens will be studied: subcutaneous (SC)mipomersen 200 mg once weekly versus placebo, and SC mipomersen 70 mg thriceweekly versus placebo.
Primary outcome measure will be percent change fromBaseline in low-density lipoprotein cholesterol (LDL-C) in Cohort 1 [ TimeFrame: Baseline and Week 60 ]
研究设计
- 研究类型
- Interventional
- 分配方式
- Computer generated randomization
- 盲法
- Participant, Investigator and Outcome Assessor Blinded
入排标准
- 年龄范围
- 18.00 Year(s) 至 99.00 Year(s)（—）
- 性别
- All
入选标准
- •Severe hypercholesterolemia (LDL-C ≥300 mg/dL (7.77 mmol/L) or LDL-C ≥200 mg/dL (5.18 mmol/L) with documented coronary heart disease (CHD) or CHD risk equivalents, or diagnosis of Heterozygous Familial Hypercholesterolemia and LDL-C ≥160 mg/dL (4.14 mmol/L) and <200 mg/dL (5.18 mmol/L))
- •On stable, maximally tolerated, statin therapy for at least 12 weeks or if statin intolerant, on at least 1 medication from another class of hypolipidemic agents (i.e., bile acid sequestrants, niacin/nicotinic acid, cholesterol absorption inhibitors, fibrates).
- •On stable, low fat diet for 12 weeks
- •Body mass index (BMI) ≤40 kg/m2 and stable weight for 6 weeks In INDIA the upper age limit is 65 YEARS.
排除标准
- •â€¢Significant health problems in the recent past including heart attack, stroke, coronary syndrome, unstable angina, heart failure, significant arrhythmia, hypertension, blood disorders, liver disease, cancer, digestive disorders, Type I diabetes, or uncontrolled Type II diabetes •Apheresis within 3 months prior to Screening or expected to start apheresis during the treatment phase.
结局指标
主要结局
Percent change from Baseline in low-density lipoprotein cholesterol (LDL-C) in Cohort 1
时间窗: Baseline and Week 60
次要结局
- • Percent Change from Baseline in Apolipoprotein B (Apo B)(• Percent Change from Baseline in Lipoprotein a)