An Efficacy and 2-Year Safety Study of Open-label Rosuvastatin in Children and Adolescents (Aged From 6 to Less Than 18 Years) With Familial Hypercholesterolaemia
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- AstraZeneca
- 入组人数
- 315
- 试验地点
- 1
- 主要终点
- Single Dose PK - Cmax
研究概览
简要总结
This study is being carried out to see if the study medication, rosuvastatin, is effective in treating familial hypercholesterolaemia in children and adolescents, and to determine the long term (over 2 years) safety, tolerability and efficacy of the study medication in these patients.
This study will also measure levels of drug in the blood and see how well it is tolerated. This is known as pharmacokinetic (PK) analysis.
At baseline only a small number of patients will participate in a single dose PK phase over 24 hours.
In order to see if this medication works, a control group of healthy siblings will help the researchers to compare certain results.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •children and adolescents (aged 6 to less than 18 years) with Familial Hypercholesterolaemia
- •Patients aged between 6 and less than 10 years of age must not be taking a statin medicine
排除标准
- •History of muscle or sensitivity reactions to any statin medicines
- •Current active liver disease or dysfunction (except a confirmed diagnosis of Gilbert's disease)
研究组 & 干预措施
1
干预措施: rosuvastatin calcium (Drug)
结局指标
主要结局
Single Dose PK - Cmax
时间窗: Serial blood samples over 24 hours.
Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing
Percent Change From Baseline in Height
时间窗: At Month 12 and Month 24
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
Percent Change From Baseline in LDL-C
时间窗: At Month 3, Month 12 and Month 24
Negative values represent a decrease and positive values represent an increase. In total, 198 patients were treated. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
Sexual Maturation by Tanner Staging at Baseline
时间窗: At Baseline
Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.
Sexual Maturation by Tanner Staging at Month 12
时间窗: At Baseline
Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.
Single Dose PK - Tmax
时间窗: Serial blood samples over 24 hours
Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing
Single Dose PK - AUC(0-24)
时间窗: Serial blood samples over 24 hours
Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing
Sexual Maturation by Tanner Staging at Month 24
时间窗: At Baseline
Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.
次要结局
- Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT)(At Month 12 and Month 24)
- Adverse Events(2-year study period)
- Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1(At Month 3, Month 12 and Month 24)
- Overal Treatment Adherence(2-year study period)
- Total Duration of Exposure(2-year study period)