SUBCUTANEOUS FORMULATION OF ICATIBANT FOR THE TREATMENT OF HEREDITARY ANGIOEDEMA

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。 遗传性血管水肿是一种罕见的遗传性疾病，患者的皮肤、肠壁、面部和生殖器、手、脚和身体以及舌头和喉咙都会出现肿胀(血管水肿)。攻击可能在任何时候发生，但已知与感染、牙科工作的创伤和月经周期有关。一些袭击事件的原因不明。当咽喉肿胀时，就有窒息的危险。 现在已经知道，肿胀至少在一定程度上是由于一种名为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，该药物可阻断体内细胞上缓激肽及其受体的作用。 在美国，没有FDA批准的药物来治疗遗传性血管性水肿症的急性发作。我们知道，我们可以通过用男性激素治疗许多患者来减少他们的发作频率。许多患者，如孕妇、年轻女性和老年男性，由于副作用而不能服用男性激素。这项研究的目的是了解在体内阻断缓激肽是否是治疗急性发作的安全和有益的方法。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症患者。最终，从这项研究中获得的信息可以用来批准icatibant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.