Recombinant Human C1 Inhibitor for Treatment of Heredit*

产品说明： 全世界每30，000人中约有1人患有HAE。 这种疾病的特征是由于功能性C1抑制剂的先天性缺乏导致补体和接触系统的不受控制的激活而引起的复发性血管性水肿，并且如果水肿在口-咽-喉区域足够严重，则可能导致死亡。 在美国，除了支持性治疗外，没有其他特定的批准治疗。 该试验将研究三组，每组13名患者。 一组将接受100单位/kg的rhC 1-Inh，第二组将接受50单位/kg的rhC 1-Inh，第三组接受安慰剂。 患者必须在腹部、面部或泌尿生殖道血管性水肿发作后5小时内就诊，并且如果适合研究，则随机接受治疗。 将使用VAS随访患者对治疗的反应。 将监测C4和C1-Inh功能的血液检查。 该试验的目的是评估这种rhC 1-Inh是否能减少急性HAE发作的持续时间和严重程度。 治疗将在急诊室或医院输液中心进行，允许患者在12或24小时出院。 随访将持续6个月。 除了确定治疗疗效外，本研究的其他目的还包括评价rhC 1-Inh的安全性和耐受性以及药理学行为。

DESCRIPTION: Approximately 1 in 30,000 people worldwide suffer from HAE. This disorder is characterized by recurrent angioedema caused by uncontrolled activation of the complement and contact systems due to a congenital deficiency of functional C1 inhibitor and may lead to death if the edema is severe enough in the oro-pharyngeal-laryngeal region. There is no specific approved treatment in the United States other than supportive care. This trial will study three groups consisting of 13 patients each. One group will receive (rhC1-Inh) at 100 units per kg, a second will receive rhC1-Inh at 50 units per kg, and a third placebo. Patients must be seen within 5 hours of the onset of abdominal, facial or urogenital attacks of angioedema and, if appropriate for the study, randomized for treatment. Patient responses to treatment will be followed using a VAS. Blood tests of C4 and C1-Inh function will be monitored. The goal of the trial is to assess if this rhC1-Inh reduces the duration and severity of acute HAE attacks. The treatment will be administered in an emergency room or hospital infusion center and the patients will be allowed to be discharged at 12 or 24 hours. Follow up will continue for six months. In addition to establishing therapeutic efficacy, other objectives of the study include evaluations of safety and tolerability and pharmacological behavior of the rhC1-Inh.