A Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of SAR236553/REGN727 in Patients With Heterozygous Familial Hypercholesterolemia and LDL-C Higher or Equal to 160mg/dL With Their Lipid-Modifying Therapy
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Sanofi
- 入组人数
- 107
- 试验地点
- 35
- 主要终点
- Percent Change From Baseline in Calculated LDL-C at Week 24 - ITT Analysis
研究概览
简要总结
Alirocumab (SAR236553/REGN727) is a fully human monoclonal antibody that binds PCSK9 (proprotein convertase subtilisin/kexin type 9).
Primary Objective of the study:
To evaluate the effect of alirocumab on low-density lipoprotein cholesterol (LDL-C) levels after 24 weeks of treatment in comparison with placebo.
Secondary Objectives:
- To evaluate the effect of alirocumab in comparison with placebo on LDL-C at other time points
- To evaluate the effects of alirocumab on other lipid parameters
- To evaluate the safety and tolerability of alirocumab
详细描述
The maximum study duration was planned to be 89 weeks per participant including participants who successfully completed the 78-week treatment period had the possibility to join an open-label extension study (LTS13463, NCT01954394) at the end of the treatment period.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Placebo Q2W
Placebo for alirocumab subcutaneous (SC) injection every two weeks (Q2W) on top of stable lipid-modifying therapy (LMT) for 78 weeks.
干预措施: Placebo (for alirocumab) (Drug)
Placebo Q2W
Placebo for alirocumab subcutaneous (SC) injection every two weeks (Q2W) on top of stable lipid-modifying therapy (LMT) for 78 weeks.
干预措施: Lipid Modifying Therapy (LMT) (Drug)
Alirocumab 150 mg Q2W
Alirocumab 150 mg SC injection Q2W on top of stable LMT for 78 weeks.
干预措施: Alirocumab (Drug)
Alirocumab 150 mg Q2W
Alirocumab 150 mg SC injection Q2W on top of stable LMT for 78 weeks.
干预措施: Lipid Modifying Therapy (LMT) (Drug)
结局指标
主要结局
Percent Change From Baseline in Calculated LDL-C at Week 24 - ITT Analysis
时间窗: From Baseline to Week 52
Adjusted Least-squares (LS) means and standard errors at Week 24 were obtained from a mixed-effect model with repeated measures (MMRM) to account for missing data. All available post-baseline data from Week 4 to Week 52 regardless of status on- or off-treatment were used in the model (ITT analysis).
次要结局
- Percent Change From Baseline in Calculated LDL-C at Week 12 - On-Treatment Analysis(From Baseline to Week 52)
- Percentage of Very High CV Risk Participants Achieving Calculated LDL-C < 70 mg/dL (<1.81 mmol/L) or High CV Risk Participants Achieving Calculated LDL-C < 100 mg/dL (<2.59 mmol/L) at Week 24 - On-Treatment Analysis(Up to Week 52)
- Percent Change From Baseline in HDL-C at Week 24 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Calculated LDL-C at Week 12 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Non-HDL-C at Week 12 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Lipoprotein (a) at Week 24 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Total Cholesterol (Total-C) at Week 24 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Apolipoprotein A-1 (Apo A-1) at Week 24 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in HDL-C at Week 12 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Calculated LDL-C at Week 24 - On-Treatment Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Apolipoprotein B (Apo B) at Week 24 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Apo B at Week 24 - On-Treatment Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Non-HDL-C at Week 24 - On-Treatment Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Calculated LDL-C at Week 52 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Lipoprotein (a) at Week 12 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Week 24 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Apo B at Week 12 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Total-C at Week 12 - ITT Analysis(From Baseline to Week 52)
- Percentage of Very High Cardiovascular (CV) Risk Participants Achieving Calculated LDL-C < 70 mg/dL (<1.81 mmol/L) or High CV Risk Participants Achieving Calculated LDL-C < 100 mg/dL (<2.59 mmol/L) at Week 24 - ITT Analysis(Up to Week 52)
- Percent Change From Baseline in Fasting Triglycerides at Week 24 - ITT Analysis(From Baseline to Week 52)
- Percent Change From Baseline in Fasting Triglycerides at Week 12 - ITT Analysis(From Baseline to Week 52)
- Percentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - ITT Analysis(Up to Week 52)
- Percent Change From Baseline in Apo A-1 at Week 12 - ITT Analysis(From Baseline to Week 52)
- Percentage of Participants Achieving Calculated LDL-C <70 mg/dL (1.81 mmol/L) at Week 24 - On-Treatment Analysis(Up to Week 52)