Treatment of Hereditary Angioedema Prodrome with Recombinant C1-esterase Inhibitor (Ruconest)
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 5
- 试验地点
- 2
- 主要终点
- The primary endpoint was the difference in response rates in preventing HAE attacks between Conestat Alfa®-treated vs. untreated HAE prodrome events.
研究概览
简要总结
To assess the efficacy of recombinant human C1-esterase inhibitor in the management of HAE prodrome for preventing the progression from prodrome to an acute angioedema attacks. Subjects will either receive Ruconest after the first 2 prodromes or during the last 2 prodromes. 5 clinic visits will occur within 24 hours of a prodrome. Subjects will complete prodrome severity and angioedema attack diaries
详细描述
After screening, subjects will be followed in the study for four consecutive prodromes. For prodromes designed to be treated with Ruconest, the drug will be administered within 12 hours of prodromal onset and prior to the onset of objective selling. Ruconset will be self-administered at home or by a healthcare professional at the recommended dose of 50 IU/kg body weight with maximum dose of 4200 IU as a slow IV injection over 5 minutes Subjects will be randomized into two arms after enrollment. Subjects in Arm A receives Ruconest after 1st and 2nd prodrome and no Ruconest after 3rd and 4th prodromes. Subjects in Arm B receive no Ruconest after 1st and 2nd prodrome and Ruconest after 3rd and 4th prodromes.
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
- None
入排标准
- 年龄范围
- 14 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Prior diagnosis of HAE Type 1 and 2,
- •One or more HAE attacks per month,
- •History of 4 prodromes that proceed to angioedema attacks
排除标准
- •History of thrombosis or arterial/venous thromboembolic attacks
- •History of atherosclerosis, morbid obesity, immobility
- •History of allergy to rabbits or products from rabbits
- •History of life-threatening immediate allergic reactions to C1 esterase inhibitor preparations
研究组 & 干预措施
Ruconest
open-label Ruconest
干预措施: Ruconest (Biological)
结局指标
主要结局
The primary endpoint was the difference in response rates in preventing HAE attacks between Conestat Alfa®-treated vs. untreated HAE prodrome events.
时间窗: up to 12 months from start of study
A mixed model for repeated measures was used to determine the statistical significance of Conestat Alfa®'s clinical efficacy for treating the HAE prodrome versus the acute attack.
次要结局
未报告次要终点
研究者
Jonathan A. Bernstein, MD
PI
Bernstein Clinical Research Center