STDY OF EFFICACY&SAFETY OF RCMBNNT C1 INHBTR 4 TRTMNT OF ACUTEATCKS OF ANGIOEDEM

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。 遗传性血管性水肿是一种遗传性疾病，患者容易受到面部、手、脚、臀部、生殖器和肠道皮肤下肿胀的攻击。如果攻击影响到舌头或喉咙，就有窒息的风险。发作可能是由创伤引起的，如受伤或牙科工作，并可能伴随月经期。一些袭击事件的原因不明。遗传性血管性水肿的患者不能制造一种名为C1抑制物(C1INH)的蛋白质。 在大多数患者中，可以通过低剂量的男性荷尔蒙(雄激素)治疗来预防发作。一些患者，特别是年轻女性，不能使用雄激素，因为它们会导致粉刺、面部毛发生长和体重增加。怀孕期间不能使用雄激素。雄激素治疗刺激身体产生C1抑制物。 在美国，急性发作的治疗包括使用麻醉药止痛和补液等支持性治疗。对于一些患者来说，输注新鲜冰冻血浆是有益的。在欧洲，C1抑制剂在商业上可以买到，是治疗遗传性血管水肿患者急性发作的标准护理。本研究的目的是研究在影响肠道、生殖器和/或面部和喉部的急性发作期间，静脉输注C1抑制剂对遗传性血管水肿患者的安全性和临床效果。我们的目标是了解哪些剂量有效，以及药物起效的速度有多快。这些结果将与接受非活性静脉输液(安慰剂)的患者观察到的结果进行比较。这项将在美国15-20个中心进行的研究的长期目标是为向美国食品和药物管理局申请批准C1抑制剂作为美国遗传性血管水肿的治疗药物提供信息

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. Hereditary angioedema is a genetic disease in which patients are susceptible to attacks of swelling under the skin of the face, hands, feet, buttocks, genitalia and intestines. If the attacks affect the tongue or throat, there is a risk of suffocation. The attacks may be brought on by trauma such an injury or dental work and may accompany a menstrual period. Some attacks have no known cause. Patients with hereditary angioedema are not able to make a protein called C1 inhibitor (C1 INH). In most patients the attacks can be prevented by treatment with low doses of male hormones (androgens). Some patients, especially young women, are not able to use androgens because they can cause acne, growth of facial hair and weight gain. Androgens cannot be used during pregnancy. Androgen treatment stimulates the body to make C1 inhibitor. In the United States, treatment of acute attacks involves use of narcotics for pain and supportive treatments such as replacement of fluids. For some patients, infusions of fresh frozen blood plasma are beneficial. In Europe, the C1 inhibitor is commercially available and is the standard of care for treatment of acute attacks in patients with hereditary angioedema. The purpose of this research study is to study the safety and clinical effects of IV infusions of C1 inhibitor in patients with hereditary angioedema during acute attacks that affect the intestines, genitalia and/or face and throat. The goals are to learn which doses are effective and how fast the drug works. These results will be compared with those observed in patients who get an inactive IV infusion (placebo). The long term goal of the study which will be done in 15-20 centers in the Country is to get information for an application to the U.S. Food and Drug Administration for approval of C1 inhibitor as a treatment for hereditary angioedema in the United States