An Observational Patient Registry to Evaluate the Real-world Safety of Ruconest® (C1 Esterase Inhibitor [Recombinant]) for the Treatment of Hereditary Angioedema
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 152
- 试验地点
- 1
- 主要终点
- Safety analysis will consist of AEs reported per patient for up to 30 days after each single or repeated dose of Ruconest.
研究概览
简要总结
This is a prospective, real-world, observational patient registry for patients with HAE who are receiving treatment with Ruconest for HAE.
详细描述
See below.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Patient provides informed consent as documented on the Institutional Review Board (IRB) approved informed consent document (ICF). For patients aged between 13 and 17 years old, the method of consent with or without assent will be determined by the IRB.
- •Patient is male or female and at least 13 years of age at the time of providing consent / assent.
- •Patient has been prescribed Ruconest for HAE.
排除标准
- •Patient is receiving HAE therapy as part of a clinical trial.
研究组 & 干预措施
Hereditary Angioedema
Patients with Hereditary Angioedema who are receiving treatment with Ruconest (rhC1INH).
干预措施: rhC1INH (Drug)
结局指标
主要结局
Safety analysis will consist of AEs reported per patient for up to 30 days after each single or repeated dose of Ruconest.
时间窗: 3 years
In addition, extent of exposure to Ruconest and concomitant medications taken for HAE will be summarized. Concomitant medications will be limited to those specifically indicated for treatment AEs or treatment of symptoms of HAE, for example C1-inhibitors, epinephrine, IV fluids, etc. AEs will be summarized as incidence per 10,000 person-days where each patient counts only once for multiple events of the same System Organ Class and preferred terms within the 30-day evaluation period. The person-time duration for each AE for each patient is the time period (in days) between the dose of Ruconest and AE onset.
次要结局
- AEs occurring during pregnancy or lactation and for breastfed infants will be summarized separately using the same methods as described in the primary outcome measure.(3 years)