C1 ESTERASE INHIBITOR AS TREATMENT AND PREVENTION IN HEREDITARY ANGIOEDEMA

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 C1INH基因杂合性缺失可导致遗传性血管性水肿(HAE)。这种疾病的特征是皮肤或粘膜出现非瘙痒性肿胀。这些肿胀一般持续2-3天，之后消退。如果喉部等部位受到影响，血管水肿的急性发作可能危及生命，而且在胃肠道发作期间往往与显著的发病率有关。因此，HAE发作需要迅速治疗，通常是在急诊室。给予C1INH-nf也可作为预防性治疗。在有治疗方法的国家，使用从正常捐赠者的混合血浆中提纯的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，旨在证明与安慰剂相比，预防性服用C1INH-nf的受试者较少发生血管性水肿症。这项研究的零假设是，HAE受试者在接受预防性C1INH-nf治疗期间与接受安慰剂治疗期间相比，他/她经历的血管性水肿症发作总数将为零。另一种假设是，与C1INH-nf治疗期间相比，受试者在安慰剂治疗期间将经历不同数量的血管水肿发作。

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 subjects treated with prophylactic C1INH-nf have fewer attacks of angioedema compared to placebo. The null hypothesis for this study is that the difference in the total number of attacks of angioedema an HAE subject experiences during the period when he/she is receiving prophylactic C1INH-nf treatments compared to the period when he/she is receiving placebo treatments will be zero. The alternative hypothesis will be that the subject will experience a different number of angioedema attacks during the placebo treatment period compared to the C1INH-nf treatment period.