SUBCUTANEOUS FORMULATION OF ICATIBANT FOR THE TREATMENT OF HEREDITARY ANGIOEDEMA

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。遗传性血管性水肿是一种罕见的遗传性疾病，患者在皮肤下、肠壁、面部和生殖器、手、脚、身体以及舌头和喉咙出现肿胀（血管性水肿）。发作可能随时发生，但已知与感染、牙科工作创伤和月经周期有关。有些攻击没有已知的原因。当喉咙肿胀时，就有窒息的危险。现在我们知道，这种肿胀至少部分是由于一种叫做缓激肽的化学物质的释放。这项研究的目的是研究一种名为Icatibant的药物的安全性和有效性，这种药物可以阻断缓激肽及其受体对体内细胞的作用。在美国，没有FDA批准的药物用于治疗遗传性血管性水肿的急性发作。我们知道，我们可以通过使用男性激素来减少许多患者的发作频率。许多患者，如孕妇、年轻女性和老年男性，由于副作用不能服用男性激素。这项研究的目的是了解在急性发作时，阻断体内的缓激肽是否是一种安全有益的治疗方法。这项研究将在美国约15个中心进行，涉及约56名遗传性血管性水肿患者。最终，从该研究中获得的信息可用于批准伊卡替班用于治疗遗传性血管性水肿患者。

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 an uncommon genetic disease in which patients have attacks of swelling (angioedema) under the skin, in the wall of the intestine, of the face and genitalia, of the hands, feet, and body and of the tongue and throat. The attacks may occur at any time, but they are known to be associated with infections, the trauma of dental work and with the menstrual cycle. Some attacks have no known cause. When the swelling occurs in the throat there is a risk of suffocation. It is now known that the swelling is due, at least in part, to release of a chemical known as bradykinin. The purpose of this research study is to study the safety and effectiveness of a drug known as Icatibant that blocks the action of the bradykinin with its receptor on cells in the body. In the United States, there are no FDA approved drugs for treatment of the acute attacks of hereditary angioedema. We know that we can reduce the frequency of attacks in many patients by treating them with male hormones. Many patients, such as pregnant women, young women and older men cannot take male hormones because of the side effects. The purpose of the research is to learn if blocking of bradykinin in the body is a safe and beneficial way to treat acute attacks when they occur. The study will be done in about 15 centers in the United States and will involve about 56 patients with hereditary angioedema. Eventually, the information gained from the study could be used to get approval of Icatibant for treatment of patients with hereditary angioedema.