C1 ESTERASE INHIBITOR AS TREATMENT AND PREVENTION IN HEREDITARY ANGIOEDEMA

C1 酯酶抑制剂治疗和预防遗传性血管性水肿

• 批准号: 7953680
• 负责人: PAULA J BUSSE
• 金额: $ 7.54万
• 依托单位: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-03-01 至 2009-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7953680
• 关键词: Accident and Emergency department; Acute; Affect; Angioneurotic Edema; Clinical; Clinical Research; Complement 1 Inactivators; Computer Retrieval of Information on Scientific Projects Database; Country; Disease; Funding; Grant; Institution; Larynx; Life; Morbidity - disease rate; Mucous Membrane; Placebos; Plasma; Prevention; Prophylactic treatment; Pruritus; Research; Research Personnel; Resources; Site; Skin; Source; Swelling; United States National Institutes of Health; clinical practice; 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.

该副本是利用众多研究子项目之一 由NIH/NCRR资助的中心赠款提供的资源。子弹和 调查员（PI）可能已经从其他NIH来源获得了主要资金， 因此，可以在其他清晰的条目中表示。列出的机构是 对于中心，这不一定是调查员的机构。 C1酯酶抑制剂（C1INH）的杂合缺乏会导致临床疾病称为遗传性血管性水肿（HAE）。 该疾病的特征是皮肤或粘膜的非核肿胀发作。 这些肿胀总体上持续了2至3天，然后他们解决了。 如果像喉部这样的部位受到影响，并且在胃肠道攻击期间通常与明显的发病率有关，则血管性水肿的急性发作可能会威胁生命。 因此，HAE攻击通常需要及时治疗，通常是在急诊室中。 C1INH-NF的给药也可以作为预防治疗。 从正常捐助者的合并等离子体中纯化的C1INH产品已成为常规临床实践，以治疗可用疗法的国家的HAE攻击。 假设： 这是一项优越的试验，以证明用预防性C1INH-NF治疗的受试者与安慰剂相比的血管性水肿发作较少。 这项研究的无效假设是，与接受安慰剂治疗的时期相比零。 另一种假设是，与C1INH-NF治疗期相比，在安慰剂治疗期间，受试者将经历不同数量的血管性水肿攻击。