A Randomized, Placebo-controlled, Double-blind Phase III Study of the Efficacy and Safety of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 75
- 试验地点
- 2
- 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 leads to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy and pharmacodynamics/ pharmacokinetics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.
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
- 16 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
- •Baseline plasma level of functional C1INH of less than 50% of normal
- •Evidence for exacerbation or development of a severe abdominal, oro-facial/ pharyngeal/ laryngeal, genito-urinary and/or peripheral HAE attack
排除标准
- •Acquired angioedema
- •Pregnancy or breastfeeding
- •Participation in another clinical study within prior 3 months
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)
Saline
Saline solution
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 has been assessed by using a patient-reported visual analogue scale ("VAS") ranging from 0 mm (no symptoms at all) to 100 mm (extremely disabling). Time to beginning of relief of symptoms at the location that showed first "VAS" score decrease of at least 20 mm from baseline score (t= 0 min) to the next assessment time-point). Assessment time-points were taken on pre-scheduled time-points after drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to beginning of relief has been calculated as median time, by using the exact time-points on which each assessment was performed.
次要结局
- Time to Minimal Symptoms(up to 48 hours after study drug administration)