A Randomized, Placebo-controlled, Double Blind Phase II/III Study of the Safety and Efficacy of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 77
- 试验地点
- 1
- 主要终点
- Time to Beginning of Relief of Symptoms
研究概览
简要总结
Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that lead to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy, and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.
Funding Source - FDA OOPD
详细描述
A prospectively planned interim analysis will be performed on the double-blind data.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clear clinical and laboratory diagnosis of HAE
- •Plasma level of functional C1INH of less than 50% of normal
- •Acute abdominal, urogenital, peripheral, and/or oro-facial/pharyngeal/laryngeal HAE attack
排除标准
- •Acquired angioedema
- •Pregnancy or breastfeeding
- •Treatment with any investigational drug within prior 30 days
- •Body weight >120 kg
研究组 & 干预措施
100 IU/kg rhC1INH
100 IU/kg Recombinant human C1 inhibitor
干预措施: Recombinant Human C1 Inhibitor (Drug)
50 IU/kg rhC1INH
50 IU/kg Recombinant human C1 inhibitor
干预措施: Recombinant Human C1 Inhibitor (Drug)
Saline
干预措施: placebo (Drug)
结局指标
主要结局
Time to Beginning of Relief of Symptoms
时间窗: up to 48 hours after study drug administration
The time to beginning of relief of symptoms at the location that showed the first visual analogue scale ("VAS") score decrease of at least 20 mm from baseline score with persistence to the next timepoint, assessment timepoints were taken on pre-scheduled time-points after study drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to beginning of relief has been calculated as median time, by using the exact timepoints on which each assessment was performed.
次要结局
- Time to Minimal Symptoms(up to 48 hours after study drug administration)