LEVP2005-1/Part B: A Double-blind, Placebo-Controlled, Clinical Study to Investigate the Efficacy and Safety of Purified C1 Esterase Inhibitor (Human) as Prophylactic Treatment to Prevent HAE Attacks
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 26
- 试验地点
- 16
- 主要终点
- Number of Hereditary Angioedema (HAE) Attacks During Each Prophylactic Therapy Period
研究概览
简要总结
The study objective was to determine the safety and efficacy of C1INH-nf for the prevention of acute HAE attacks.
详细描述
Subjects were given diary cards and instructed to document all HAE attacks on a daily basis. Subjects evaluated their symptoms over the previous 24 hours, noting the severity and duration of swelling at each of 5 locations (abdominal, genitourinary, facial, respiratory [including laryngeal], and/or extremity).
The study design also allowed for administration of open-label C1INH-nf (1,000 U of C1INH-nf administered IV [repeated after 60 minutes, if necessary] for treatment of laryngeal angioedema or if deemed necessary by the investigator; 1,000 U of C1INH-nf administered IV [single dose] prior to emergency surgical procedures).
A total of 26 subjects were enrolled in the study. One subject received open-label C1INH-nf but withdrew prior to randomization. Another subject was randomized but withdrew prior to receiving study drug. Twenty-four (24) subjects were randomized and treated with blinded study drug. In total, 25 subjects received at least 1 dose of study drug and were analyzed for safety; all 25 subjects were exposed to C1INH-nf and 23 subjects were exposed to placebo.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Prevention
- 盲法
- Double (Participant, Investigator)
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
- •Documented HAE
- •Normal C1q level
- •Relatively frequent angioedema attacks (at least 2 per month on average)
排除标准
- •Low C1q level
- •B-cell malignancy
- •Presence of anti-C1INH autoantibody
- •History of allergic reaction to C1INH or other blood products
- •Narcotic addiction
- •Current participation in any other investigational drug study or within the past 30 days
- •Participation in a C1 esterase inhibitor trial, or received blood or a blood product in the past 90 days
- •Pregnancy or lactation
- •Any clinically significant medical condition, such as renal failure, that in the opinion of the investigator would interfere with the subject's ability to participate in the study
研究组 & 干预措施
C1INH-nf First, then Placebo
1,000 Units (U) of C1INH-nf administered intravenously (IV) every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by matching placebo (saline) administered IV every 3 to 4 days for 12 weeks.
干预措施: C1 esterase inhibitor [human] (C1INH-nf) (Biological)
C1INH-nf First, then Placebo
1,000 Units (U) of C1INH-nf administered intravenously (IV) every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by matching placebo (saline) administered IV every 3 to 4 days for 12 weeks.
干预措施: Placebo (saline) (Drug)
Placebo First, then C1INH-nf
Matching placebo (saline) administered IV every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by 1,000 U of C1INH-nf administered IV every 3 to 4 days for 12 weeks.
干预措施: C1 esterase inhibitor [human] (C1INH-nf) (Biological)
Placebo First, then C1INH-nf
Matching placebo (saline) administered IV every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by 1,000 U of C1INH-nf administered IV every 3 to 4 days for 12 weeks.
干预措施: Placebo (saline) (Drug)
结局指标
主要结局
Number of Hereditary Angioedema (HAE) Attacks During Each Prophylactic Therapy Period
时间窗: 12 weeks
An HAE attack was defined as the subject-reported indication of swelling at any location following a report of no swelling on the previous day. Analyses include observed attack counts and normalized attack counts (i.e., the number of attacks observed during each therapy period, normalized for the number of days the subject participated in that period).
次要结局
- Number of Subject Withdrawals During Each Prophylactic Therapy Period(12 weeks)
- Average Severity of HAE Attacks During Each Prophylactic Therapy Period(12 weeks)
- Antigenic C1 Inhibitor (C1INH) Serum Levels(Pre-infusion to 1 hour post-infusion at Visit 1 and Weeks 4, 8, and 12)
- Average Duration of HAE Attacks During Each Prophylactic Therapy Period(12 weeks)
- Number of Open-label C1INH-nf Infusions Required During Each Prophylactic Therapy Period(12 weeks)
- Functional C1INH Serum Levels(Pre-infusion to 1 hour post-infusion at Visit 1 and Weeks 4, 8, and 12)