Double-blind, Randomized, Multicenter, Placebo-Controlled Study to Characterize the Efficacy, Safety, and Tolerability of 24 Weeks of Evolocumab for LDL-C Reduction in Pediatric Subjects 10 to 17 Years of Age With HeFH
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Amgen
- 入组人数
- 158
- 试验地点
- 1
- 主要终点
- Percent Change From Baseline to Week 24 in LDL-C
研究概览
简要总结
A study to assess safety and efficacy of evolocumab (AMG-145) in paediatric subjects aged 10-17 years diagnosed with heterozygous familial hypercholesterolemia.
详细描述
A study to evaluate the effect of 24 weeks of subcutaneous (SC) evolocumab compared with placebo, when added to standard of care, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in pediatric subjects 10 to 17 years of age with heterozygous familial hypercholesterolemia (HeFH).
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
- Triple (Participant, Care Provider, Investigator)
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
- •Male or female ≥ 10 to ≤ 17 years of age (before 18th birthday)
- •Diagnosis of heterozygous familial hypercholesterolemia
- •On an approved statin with stable optimized dose for ≥ 4 weeks
- •Other lipid-lowering therapy stable for ≥ 4 weeks (fibrates must be stable for ≥ 6 weeks)
- •Fasting LDL-C ≥ 130 mg/dL (3.4 mmol/L)
- •Fasting triglycerides ≤ 400 mg/dL (4.5 mmol/L)
排除标准
- •Type 1 diabetes, or type 2 diabetes that is or poorly controlled
- •Uncontrolled hyperthyroidism or hypothyroidism
- •Cholesterylester transfer protein (CETP) inhibitor in the last 12 months, or mipomersen or lomitapide in the last 5 months
- •Previously received evolocumab or any other investigational therapy to inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9).
- •Lipid apheresis within the last 12 weeks prior to screening.
- •Homozygous familial hypercholesterolemia
研究组 & 干预措施
Placebo
Matching subcutaneous injection every 4 weeks (QM)
干预措施: Placebo (Drug)
EvoMab 420 mg QM
Evolocumab subcutaneous injection QM
干预措施: Evolocumab (Drug)
结局指标
主要结局
Percent Change From Baseline to Week 24 in LDL-C
时间窗: Baseline, Week 24
Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from interactive voice response system \[IVRS\]), scheduled visit and the interaction of treatment with scheduled visit as covariates. The model uses an unstructured covariance.
次要结局
- Mean Percent Change From Baseline to Mean of Weeks 22 and 24 in LDL-C(Baseline, Week 22, Week 24)
- Change From Baseline to Week 24 in LDL-C(Baseline, Week 24)
- Change From Baseline Over Time in Heart Rate(Baseline, Week 4, Week 12, Week 20, Week 22, Week 24)
- Percent Change From Baseline to Week 24 in ApoB:ApoA1 Ratio(Baseline, Week 24)
- Percent Change From Baseline to Week 24 in Non-HDL-C(Baseline, Week 24)
- Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB)(Baseline, Week 24)
- Percent Change From Baseline to Week 24 in Total Cholesterol/HDL-C Ratio(Baseline, Week 24)
- Number of Participants With Maximum Post-Baseline Laboratory Toxicities of Grade ≥ 3(Week 24)
- Change From Baseline Over Time in Systolic Blood Pressure(Baseline, Week 4, Week 12, Week 20, Week 22, Week 24)
- Change From Baseline Over Time in Diastolic Blood Pressure(Baseline, Week 4, Week 12, Week 20, Week 22, Week 24)
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation (DC), Fatal TEAEs, and Device-Related TEAEs(From first dose of study drug up to and including 30 days after the last dose or end of study date (Week 24), whichever was earlier. Mean (SD) duration on study was 5.664 (0.278) and 5.608 (0.137) months for Placebo and EvoMab arms, respectively.)
- Number of Participants Testing Positive for Anti-Evolocumab Antibodies(up to Week 24)
- Serum Evolocumab Concentrations Over Time(Week 12, Week 22, Week 24)