CHANGE TRIAL

变更试用

• 批准号: 7718156
• 负责人: PAULA J BUSSE
• 金额: $ 10.1万
• 依托单位: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-03-01 至 2009-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7718156
• 关键词: Accident and Emergency department; Acute; Affect; Angioneurotic Edema; Clinical; Complement 1 Inactivators; Computer Retrieval of Information on Scientific Projects Database; Country; Disease; Funding; Grant; Institution; Larynx; Life; Morbidity - disease rate; Mucous Membrane; Numbers; Placebos; Plasma; Prophylactic treatment; Pruritus; Research; Research Personnel; Resources; Site; Skin; Source; Swelling; United States National Institutes of Health; day; experience; gastrointestinal; hereditary angioneurotic edema; prophylactic; treatment duration

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.

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。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 研究中心，而研究中心不一定是研究者所在的机构。 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，接受预防性C1 INH-nf治疗的受试者的血管性水肿发作较少。 本研究的零假设是HAE受试者在他/她接受预防性C1 INH-nf治疗期间经历的血管性水肿发作总数与他/她接受安慰剂治疗期间相比的差异为零。 备择假设是，与C1 INH-nf治疗期相比，受试者在安慰剂治疗期发生血管性水肿发作的次数不同。