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

功效研究

基本信息

批准号：
7719489
负责人：
CHARLES HARVEY KIRKPATRICK
金额：
$ 0.02万
依托单位：
UNIVERSITY OF COLORADO DENVER
依托单位国家：
美国
项目类别：
财政年份：
2008
资助国家：
美国
起止时间：
2008-04-01 至 2008-05-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/7719489
关键词：
Acne Acute Affect Androgens Asphyxia Buttocks Caring Complement component C1s Computer Retrieval of Information on Scientific Projects Database Country Dental Dose Europe Face Freezing Funding Genitalia Goals Grant Growth Hair Hand Hereditary Disease Hormones Infusion procedures Injury Institution Intestines Intravenous infusion procedures Learning Liquid substance Menstruation Narcotics Pain Patients Pharmaceutical Preparations Pharyngeal structure Placebos Plasma Pregnancy Proteins Purpose Research Research Personnel Resources Risk Safety Source Standards of Weights and Measures Swelling Tongue Trauma United States United States Food and Drug Administration United States National Institutes of Health Weight Gain Woman Work clinical effect face skin foot hereditary angioneurotic edema inhibitor/antagonist male prevent research study

项目摘要

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

该子项目是利用该技术的众多研究子项目之一资源由 NIH/NCRR 资助的中心拨款提供。子项目及研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金，因此可以在其他 CRISP 条目中表示。列出的机构是对于中心来说，它不一定是研究者的机构。遗传性血管性水肿是一种遗传性疾病，患者面部、手、脚、臀部、生殖器和肠道的皮肤下容易出现肿胀。如果攻击影响到舌头或喉咙，则有窒息的风险。这些发作可能是由外伤（例如受伤或牙科手术）引起的，并且可能伴随月经期。有些攻击的原因不明。患有遗传性血管性水肿的患者无法产生一种称为 C1 抑制剂 (C1 INH) 的蛋白质。对于大多数患者来说，可以通过低剂量的雄性激素（雄激素）治疗来预防发作。一些患者，尤其是年轻女性，无法使用雄激素，因为雄激素会导致痤疮、面部毛发生长和体重增加。怀孕期间不能使用雄激素。雄激素治疗会刺激身体产生 C1 抑制剂。在美国，急性发作的治疗包括使用麻醉剂止痛和支持治疗，例如补充液体。对于某些患者来说，输注新鲜冰冻血浆是有益的。在欧洲，C1 抑制剂已上市，并且是治疗遗传性血管水肿患者急性发作的标准治疗方法。本研究的目的是研究影响肠道、生殖器和/或面部和喉咙的遗传性血管性水肿急性发作期间静脉输注 C1 抑制剂的安全性和临床效果。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注（安慰剂）的患者中观察到的结果进行比较。该研究将在国内 15-20 个中心进行，其长期目标是获取向美国食品和药物管理局申请批准 C1 抑制剂用于治疗遗传性血管性水肿的信息。