An Open-label, Cross-over, Dose-ranging Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of the Subcutaneous Administration of a Human Plasma-derived C1-esterase Inhibitor in Subjects With Hereditary Angioedema
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 18
- 试验地点
- 9
- 主要终点
- Modeled C1-esterase Inhibitor Functional Activity Trough Level
研究概览
简要总结
The aim of the study is to assess what happens to C1-esterase inhibitor that is administered under the skin of subjects with hereditary angioedema. Three different dosing regimens of C1-esterase inhibitor will be assessed. Each subject will be assigned to receive 2 of the 3 dosing regimens, each for 4 weeks. The activity and concentration of C1-esterase inhibitor in the blood will be measured during each 4-week period. The study will also examine how well C1-esterase inhibitor administered under the skin is tolerated by the subjects.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Crossover
- 主要目的
- Prevention
- 盲法
- None
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
- •Males or females aged 18 years or older.
- •Laboratory-confirmed hereditary angioedema type I or II.
- •Less than two hereditary angioedema attacks per month in the last three months.
- •Body weight of 50.0 kg to 110.0 kg.
排除标准
- •Receiving prophylactic C1-esterase inhibitor therapy.
- •Received C1-esterase inhibitor, ecallantide, icatibant or any blood products for the prevention or treatment of hereditary angioedema within 7 days before the screening visit.
- •Intends to use recombinant C1-esterase inhibitor or fresh frozen plasma for the acute treatment of hereditary angioedema during the study.
- •Received androgen therapy (e.g., danazol, oxandrolone, stanozolol, testosterone) within 30 days before the screening visit.
- •Female subjects who started taking or changed dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progesterone-containing products) within 3 months prior to the screening visit.
- •Known or suspected hypersensitivity to the study product, or to any excipients of the study product.
- •Pregnancy or lactation.
结局指标
主要结局
Modeled C1-esterase Inhibitor Functional Activity Trough Level
时间窗: at the fourth week of each dosing regimen
Mean trough C1-esterase inhibitor functional activity of the low, medium and high subcutaneous dose regimens, based on modeling and simulation
次要结局
- As-observed C1-esterase Inhibitor Functional Activity Trough Level(during the last week of 4-week dose regimen)
- C1-esterase Inhibitor Concentration Trough Level(during the last week of 4-week dose regimen)
- C4 Concentration Trough Level(during the last week of 4-week dose regimen)
- Change From Baseline in C1-esterase Inhibitor Functional Activity(Baseline and during the last week of 4-week dose regimen)
- Change From Baseline in C1-esterase Inhibitor Concentration(Baseline and during the last week of 4-week dose regimen)
- Change From Baseline in C4 Concentration(Baseline and during the last week of 4-week dose regimen)