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

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。所列机构为中心机构，不一定为研究者机构。C1酯酶抑制剂（C1 INH）的杂合缺陷导致称为遗传性血管性水肿（HAE）的临床疾病。这种疾病的特征是皮肤或粘膜的非瘙痒性皮炎发作。这些症状通常持续2至3天，然后消退。血管性水肿的急性发作可能危及生命，如果像喉的网站受到影响，并往往与胃肠道发作期间的显着发病率。因此，HAE发作需要及时治疗，通常在急诊室。C1 INH-nf给药也可能作为预防性治疗。从正常供体的合并血浆中纯化的C1 INH产品的给药已成为治疗HAE发作的常规临床实践，在治疗可用的国家。 假设：这是一项优效性试验，旨在证明与安慰剂相比，C1 INH-nf治疗的受试者至定义症状缓解的中位时间较短。本研究的零假设是，在接受C1 INH-nf或安慰剂治疗的HAE受试者中，至合格血管性水肿发作的定义症状开始缓解的时间差异为零。备择假设为C1 INH-nf治疗受试者的至缓解时间与安慰剂治疗受试者不同。

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.