A Three Year, Prospective, Open-label, Study To Evaluate Clinical Efficacy, Safety And Tolerability Of Atorvastatin In Children And Adolescents With Heterozygous Familial Hypercholesterolemia
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 272
- 试验地点
- 40
- 主要终点
- Low Density Lipoprotein Cholesterol (LDL-C; Millimoles Per Liter [mMol/L]) During the Study
研究概览
简要总结
The purpose of this study is to characterize three year descriptive growth and development (ie, height, weight, body mass index, Tanner Stage) and efficacy of cholesterol reduction in pediatric subjects with Heterozygous Familial Hypercholesterolemia receiving atorvastatin treatment.
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
- 6 Years 至 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Heterozygous familial hypercholesterolemia, ages 6-15, LDL greater than 4 mmol/l
排除标准
- •Active liver disease or hepatic dysfunction, or persistent elevations of serum transaminases exceeding three times the upper limit of normal (ULN).
- •Female of childbearing potential who is not using adequate contraceptive measures or any female who is pregnant or breastfeeding. Any female who becomes pregnant during study participation will be immediately discontinued from treatment and counseled appropriately about the in utero exposure.
- •Known hypersensitivities to HMG-CoA reductase inhibitors
研究组 & 干预措施
Atorvastatin
All subjects will be treated with atorvastatin
干预措施: atorvastatin (Drug)
结局指标
主要结局
Low Density Lipoprotein Cholesterol (LDL-C; Millimoles Per Liter [mMol/L]) During the Study
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination [ET])
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Percent Change From Baseline in LDL-C
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or ET)
Assessments were performed in the fasting state (minimum 10-hour fast).
Percent Change From Baseline in Total Cholesterol
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast).
Apoliprotein B (Apo B; g/L) During the Study
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Age (Years) During the Study: Females
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of age during the study. Change from baseline was also determined.
High-Density Lipoprotein Cholesterol (HDL-C; mMol/L) During the Study
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Total Cholesterol (mMol/L) During the Study
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Percent Change From Baseline in Trigylcerides
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast).
Trigylcerides (mMol/L) During the Study
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Percent Change From Baseline in Apo B
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast).
Number of Participants With Shift From Baseline in Tanner_Stage by Timepoint and Baseline Tanner_Stage
时间窗: Baseline, Months 6, 12, 18, 24, 30, and 36/ET
Tanner_Stage was assessed based on 2 components by gender, pubic hair and breasts for females and pubic hair and genitalia for males. If these values of components were not same, then the Tanner_Stage had the higher value of 2 components for each gender by visit.
Weight (kg) During the Study: Females
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of weight changes during the study. Change from baseline was also determined.
Percent Change From Baseline in Weight: Females
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of weight changes during the study.
BMI (kg/m^2) During the Study: Females
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of BMI changes during the study. Change from baseline was also determined.
Age (Years) During the Study: Males
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of age during the study. Change from baseline was also determined.
Percent Change From Baseline in HDL-C
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast).
Very Low-Density Lipoprotein (VLDL; mMol/L) During the Study
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Apoliprotein A-1 (Apo A-1; Grams Per Liter [g/L]) During the Study
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast). Change from baseline was also determined.
Percent Change From Baseline in Apo A-1
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast).
Height (Centimeters [cm]) During the Study: Males
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of height changes during the study. Change from baseline was also determined.
Percent Change From Baseline in Height: Females
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of height changes during the study.
Weight (Kilograms [kg]) During the Study: Males
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of weight changes during the study. Change from baseline was also determined.
Percent Change From Baseline in Weight: Males
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of weight changes during the study.
Percent Change From Baseline in Age: Males
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of age during the study.
Percent Change From Baseline in FMD
时间窗: Months 6, 12, 18, 24, 30 and 36/ET
Percent (%) FMD was calculated as (hyperemic diameter minus resting diameter) divided by the resting diameter multiplied by 100.
Percent Change From Baseline in VLDL
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Assessments were performed in the fasting state (minimum 10-hour fast).
Percent Change From Baseline in Height: Males
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of height changes during the study.
Height (cm) During the Study: Females
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of height changes during the study. Change from baseline was also determined.
Body Mass Index (BMI in kg Per Square Meter [kg/m^2]) During the Study: Males
时间窗: Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of BMI changes during the study. Change from baseline was also determined.
Percent Change From Baseline in BMI: Males
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of BMI changes during the study.
Percent Change From Baseline in BMI: Females
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of BMI changes during the study.
Percent Change From Baseline in Age: Females
时间窗: Months 1, 2, 3, 6, 12, 18, 24, 30 and 36/ET
Investigator assessment of age during the study.
Flow-Mediated Dilatation (FMD) During the Study
时间窗: Baseline, Months 6, 12, 18, 24, 30 and 36/ET
Percent (%) FMD was calculated as (hyperemic diameter minus resting diameter) divided by the resting diameter multiplied by 100. Change from baseline was also determined.
次要结局
- Percentage of Participants With Overall Expected Maturation and Development Consistent With Expectations as Assessed by the Investigator(Baseline, Months 1, 2, 3, 6, 12, 18, 24, 30 and 36 (or early termination))
- Percentage of Participants by Study Drug Compliance Category(Months 1, 2, 3, 6, 12, 18, 24, 30, and 36 (or early termination))