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：
7604439
负责人：
CHARLES HARVEY KIRKPATRICK
金额：
$ 0.17万
依托单位：
UNIVERSITY OF COLORADO DENVER
依托单位国家：
美国
项目类别：
财政年份：
2007
资助国家：
美国
起止时间：
2007-07-01 至 2008-03-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/7604439
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代表。列出的机构是对于中心，这不一定是调查员的机构。遗传性血管性水肿是一种遗传疾病，患者容易在面部，手，脚，臀部，生殖器和肠子的皮肤下肿胀发作。如果攻击影响舌头或喉咙，则有窒息的风险。袭击可能是由于受伤或牙科工作的创伤带来的，并且可能伴随着月经时期。有些攻击没有已知原因。遗传性血管性水肿的患者无法制造称为C1抑制剂（C1 INH）的蛋白质。在大多数患者中，可以通过低剂量的雄性激素（雄激素）治疗来预防攻击。一些患者，尤其是年轻女性，无法使用雄激素，因为它们会引起痤疮，面部毛发的生长和体重增加。怀孕期间不能使用雄激素。雄激素治疗刺激人体制造C1抑制剂。在美国，急性攻击的治疗涉及使用麻醉品用于疼痛和支持治疗，例如替代液体。对于某些患者而言，新鲜冷冻血浆的输注是有益的。在欧洲，C1抑制剂可商购，是治疗遗传性血管性血管性患者急性攻击的护理标准。这项研究的目的是研究C1抑制剂在影响肠道，生殖器和/或脸部和喉咙的急性攻击期间C1抑制剂IV输注的安全性和临床作用。目标是学习哪种剂量有效以及药物的工作速度。这些结果将与在静脉注射不活跃（安慰剂）的患者中观察到的结果进行比较。该研究的长期目标将在该国15-20个中心完成，以获取向美国食品和药物管理局申请批准C1抑制剂作为遗传性血管性水肿的治疗的信息