A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, 3-Period Crossover Study to Evaluate the Efficacy and Safety of Recombinant Human C1 Inhibitor in the Prophylaxis of Angioedema Attacks in Patients With Hereditary Angioedema (HAE)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 32
- 试验地点
- 19
- 主要终点
- Number of HAE Attacks
研究概览
简要总结
Primary Objective:
To evaluate the efficacy of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE
Secondary Objective:
To evaluate the safety and immunogenicity of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE
详细描述
Study Design:
This is a multi-center, randomized, double-blind, placebo-controlled, 3-period crossover study of rhC1INH in prophylaxis of angioedema attacks in patients with HAE.
Medical screening (clinical and laboratory parameters) will be performed and patient medical history specific to HAE attacks will be collected to assess eligibility. Each patient will receive three 4 week periods of treatment twice weekly.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 13 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients fulfilling the following criteria at Screening are eligible for participation in the study:
- •Age 13 years or older
- •Laboratory confirmed diagnosis of HAE
- •A history of frequent HAE attacks (at least 4 attacks per month across a minimum of 3 consecutive months).
- •Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception.
- •Provided written informed consent (and written assent for minors)
- •Willingness and ability to comply with all protocol procedures
排除标准
- •Patients who meet any of the following criteria at Screening are to be excluded from study participation:
- •Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
- •Diagnosis of acquired angioedema (AAE)
- •Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant
- •Treatment with any investigational drug in the past 30 days
- •Patients with any condition or treatment that, in the opinion of the Investigator, might interfere with the evaluation of study objectives
- •Patients currently treated with angiotensin-converting enzyme (ACE) inhibitors
研究组 & 干预措施
rhC1INH twice weekly
rhC1INH administered twice weekly
干预措施: Recombinant human C1 inhibitor (Biological)
rhC1INH once weekly
rhC1INH administered once weekly
干预措施: Recombinant human C1 inhibitor (Biological)
Placebo (Saline) twice weekly
Placebo (Saline) administered twice weekly
干预措施: Placebo (Other)
结局指标
主要结局
Number of HAE Attacks
时间窗: 28 days
Average number of HAE attacks normalized to a 28 day period
次要结局
- Number of Participants With Adverse Events(20 weeks)
- Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks(28 days)