An Open-label, Randomized Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneous Administration of Human Plasma-derived C1-esterase Inhibitor in the Prophylactic Treatment of Hereditary Angioedema
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 126
- 试验地点
- 33
- 主要终点
- Person-time Incidence Rates (Subject Based)
研究概览
简要总结
The aim of this study is to assess the long-term safety of C1-esterase inhibitor (C1-INH) in preventing hereditary angioedema (HAE) attacks when it is administered under the skin of subjects with HAE. The safety of participating subjects will be assessed for up to 54 weeks. The long-term efficacy of C1-INH will also be assessed. Each eligible subject will enter the treatment phase, wherein subjects will be randomized to treatment with either low- or medium-volume C1-INH. Subjects who have an insufficient treatment response during the study will be given an opportunity to undergo a dose increase. The study aims to enroll eligible subjects who completed study CSL830_3001 (NCT01912456). Subjects who did not participate in study CSL830_3001 may also participate, if eligible and if space permits. Subjects from the United States (US) who complete Treatment Period 2 will be allowed to participate in an Extension Period. During the Extension Period participating US subjects will continue to receive treatment with open-label CSL830 for up to an additional 88 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males or females aged 6 years or older.
- •A confirmed diagnosis of HAE type I or II.
- •HAE attacks over a consecutive 2-month period that required acute treatment, medical attention, or caused significant functional impairment.
- •For subjects who have used oral therapy for prophylaxis against HAE attacks within 3 months of first study visit: use of a stable regimen within 3 months of the first study visit.
排除标准
- •Incurable malignancies.
- •Any clinical condition that will interfere with the evaluation of C1-INH therapy.
- •Clinically significant history of poor response to C1-esterase therapy for the management of HAE.
- •Suspected or confirmed diagnosis of acquired HAE or HAE with normal C1-INH.
- •Inability to have HAE managed pharmacologically with on-demand treatment.
研究组 & 干预措施
C1-INH - low-volume dose
A low-volume dose of C1-INH will be administered subcutaneously twice a week for up to 52 weeks (up to 146 weeks extension period).
干预措施: C1-esterase inhibitor (Biological)
C1-INH - medium-volume dose
A medium-volume dose of C1-INH will be administered subcutaneously twice a week for up to 52 weeks (up to 146 weeks extension period).
干预措施: C1-esterase inhibitor (Biological)
结局指标
主要结局
Person-time Incidence Rates (Subject Based)
时间窗: Up to 146 weeks.
Subject-based Analysis for Person-Time Incidence Rate = (the total number of subjects who experienced the event during the respective treatment) / (the sum of the date each subject experienced the event - the subject's start date + 1 day) / (365.25 days). The analysis population for this endpoint was the Safety Population: the Safety Population comprised all subjects who provided informed consent / assent, who were randomized, and who received at least 1 dose or a partial dose of CSL830.
The Person-time Incidence Rates (Event Based)
时间窗: Up to 146 weeks.
Event-based Analysis for Person-Time Incidence Rate = (the total number of events documented during the respective treatment) / (the sum of each subject's end date - the subject's start date + 1 day) / (365.25 days). The analysis population for this endpoint was the Safety Population: The Safety Population comprised all subjects who provided informed consent / assent, who were randomized, and who received at least 1 dose or a partial dose of CSL830.
次要结局
- Percentage of Injections Followed by At Least One Solicited Adverse Event(Up to 146 weeks)
- Percentage of Subjects Who Experience a Time-normalized HAE Attack Frequency of <1 HAE Attack Per 4-Week Period(Up to 146 weeks)
- Percentage of Subjects Who Are Responders(Up to 146 weeks)
- Percentage of Subjects Who Have Solicited Adverse Events (AEs)(Up to 146 weeks)
- Percentage of Subjects Who Become Seropositive for Human Immunodeficiency Virus (HIV-1/-2), Hepatitis B Virus, or Hepatitis C Virus.(Up to 146 weeks)