Open-label, Single-Arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Amgen
- 入组人数
- 163
- 试验地点
- 47
- 主要终点
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
The main purpose of this study is to describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab when added to standard of care in children 10 to 17 years of age with familial hypercholesterolemia.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 10 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Heterozygous Familial Hypercholesterolemia (HeFH):
- •Completed Study 20120123 (NCT02392559) while still on assigned investigational product and did not experience a treatment-related serious adverse event
- •Homozygous Familial Hypercholesterolemia (HoFH):
- •Male or female, ≥ 10 to ≤ 17 years of age at time of enrollment
- •Diagnosis of HoFH
- •On a low-fat diet and receiving background lipid-lowering therapy
- •Lipid-lowering therapy unchanged for ≥ 4 weeks prior to LDL-C screening; fibrates must be stable for at least 6 weeks prior to screening.
- •Fasting LDL-C at screening ≥ 130 mg/dL (3.4 mmol/L)
- •Fasting triglycerides ≤ 400 mg/dL (4.5 mmol/L)
排除标准
- •Currently receiving treatment in another investigational device or drug study, or less than 30 days since ending treatment on another investigational device or drug study(s); except Study 20120123
- •Moderate to severe renal dysfunction
- •Active liver disease or hepatic dysfunction,
- •Creatine kinase > 3 times the upper limit of normal (ULN) at screening
结局指标
主要结局
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
时间窗: From first dose of evolocumab in this study up to and including 30 days after the last dose or up to the end of study date, whichever was earlier; up to 80 weeks.
An adverse event is defined as any untoward medical occurrence in a clinical trial participant, not necessarily having a causal relationship with study treatment. A serious AE is as an AE that met at least 1 of the following criteria: * fatal; * life threatening; * required in-patient hospitalization or prolongation of existing hospitalization; * resulted in persistent or significant disability/incapacity; * congenital anomaly/birth defect; * other medically important serious event. AEs were graded for severity using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0: Grade 1: Mild; asymptomatic or mild symptoms; Grade 2: Moderate; minimal, local or noninvasive intervention indicated; Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; Grade 4: Life-threatening consequences; urgent intervention indicated; Grade 5: Death related to AE.
次要结局
- Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HoFH Participants(Baseline and week 80)
- Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HoFH Participants(Baseline and week 80)
- Change From Baseline to Week 80 in LDL-C in HeFH Participants(Baseline and week 80)
- Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HeFH Participants(Baseline and week 80)
- Percent Change From Baseline to Week 80 in Non-HDL-C in HeFH Participants(Baseline and week 80)
- Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80(Week 80)
- Number of Participants With Change in Tanner Staging From Baseline to Week 80(Baseline and week 80)
- Change From Baseline to Week 80 in Cortisol Levels(Baseline and week 80)
- Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HeFH Participants(Baseline and week 80)
- Percent Change From Baseline to Week 80 in Apolipoprotein B in HeFH Participants(Baseline and week 80)
- Percent Change From Baseline to Week 80 in Apolipoprotein B / Apolipoprotein A1 Ratio in HeFH Participants(Baseline and week 80)
- Percent Change From Baseline to Week 80 in Apolipoprotein B/Apolipoprotein A1 Ratio in HoFH Participants(Baseline and week 80)
- Change From Baseline to Week 80 in Testosterone Levels(Baseline and week 80)
- Change From Baseline to Week 80 in Follicle Stimulating Hormone (FSH) Levels(Baseline and week 80)
- Change From Baseline to Week 80 in Adenocorticotropic Hormone (ACTH) Levels(Baseline and week 80)
- Percent Change From Baseline to Week 80 in Non-HDL-C in HoFH Participants(Baseline and week 80)
- Percent Change From Baseline to Week 80 in Apolipoprotein B in HoFH Participants(Baseline and week 80)
- Change From Baseline to Week 80 in LDL-C in HoFH Participants(Baseline and week 80)
- Change From Baseline in Height at Weeks 24, 48, and 80(Baseline and weeks 24, 48, and 80)
- Change From Baseline to Week 80 in Estradiol Levels(Baseline and week 80)
- Change From Baseline to Week 80 in Luteinizing Hormone (LH) Levels(Baseline and week 80)
- Change From Baseline to Week 80 in Dehydroepiandrosterone Sulfate (DHEA-S) Levels(Baseline and week 80)
- Number of Participants With Liver Function Test Abnormalities at Week 80(Week 80)
- Change From Baseline to Week 80 in Carotid Intima-media Thickness (cIMT)(Baseline and week 80)
- Change From Baseline in Weight at Weeks 24, 48, and 80(Baseline and weeks 24, 48, and 80)