EFFICACY AND SAFETY OF PURIFIED C1 ESTERASE INHIBITOR FOR TREATMENT OF HAE

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。C1酯酶抑制剂（C1INH）的杂合性缺乏可导致遗传性血管性水肿（HAE）的临床疾病。这种疾病的特点是皮肤或粘膜出现非瘙痒性肿胀。这些肿胀一般会持续2到3天，之后就会消退。如果喉部等部位受到影响，血管性水肿的急性发作可能危及生命，并且在胃肠道发作时通常伴有显著的发病率。因此，HAE发作需要及时治疗，通常是在急诊室。给予C1INH-nf也可作为预防性治疗。在有这种疗法的国家，使用从正常供体汇集血浆中纯化的C1INH产品已成为治疗HAE发作的常规临床实践。假设：这是一项优势试验，证明与安慰剂相比，接受C1INH-nf治疗的受试者有更短的中位时间来缓解定义症状。本研究的零假设是，在接受C1INH-nf或安慰剂治疗的HAE患者中，血管性水肿发作的定义症状开始缓解的时间差异为零。另一种假设是，与安慰剂相比，接受C1INH-nf治疗的受试者缓解的时间不同。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Heterozygous deficiency of C1 esterase inhibitor (C1INH) leads to the clinical disease known as Hereditary Angioedema (HAE). This disease is characterized by attacks of non-itching swellings of the skin or mucosa. These swellings in general last for 2 to 3 days after which they resolve. Acute attacks of angioedema may be life-threatening if sites like the larynx are affected, and are often associated with significant morbidity during gastrointestinal attacks. Hence, HAE attacks require prompt treatment, often in an emergency room. Administration of C1INH-nf might also be warranted as prophylactic treatment. Administration of C1INH products purified from pooled plasma from normal donors has become routine clinical practice to treat attacks of HAE in countries where the therapy is available. Hypothesis: This is a superiority trial to demonstrate that C1INH-nf treated subjects have a shorter median time to relief of the defining symptoms compared to placebo. The null hypothesis for this study is that the difference in the time to beginning of relief for the defining symptom of an eligible attack of angioedema in HAE subjects treated with either C1INH-nf or placebo will be zero. The alternative hypothesis will be that the time to relief is different in subjects treated with C1INH-nf compared to placebo.