CLINICAL STUDY TO INVESTIGATE THE EFFICACY AND SAFETY OF PURIFIED C1 ESTERASE

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目及 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 背景：遗传性血管性水肿是一种罕见的遗传性疾病，症状为手臂、腿部、面部、咽喉和/或腹部肿胀。 假设：C1酯酶抑制剂有望减轻遗传性血管性水肿的症状，并阻止急性发作患者的进展。 具体目的和程序（总结）：在研究期间，您可能会接受4剂DX-88中的1剂。 它将通过输液超过10分钟。 所给的金额是基于你的体表面积，计算你的身高和体重。 实验设计（总结）：最初，将在大约60名患者中研究60次发作。美国和欧洲的50个临床中心。 这可能会增加到240次攻击。 每次发作最多给予2剂DX-88。 在本研究中，您可以接受多次发作的治疗。 意义（总结）：目前在美国和一些欧洲国家还没有批准用于阻止遗传性血管性水肿发作的药物。 现有的药物只能治疗症状。

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. Background: Hereditary angioedema is a rare inherited disease with symptoms of swelling of your arms, legs, face, throat, and/or abdomen. Hypothesis: An inhibitor of C1 esterase would be expected to reduce symptoms of hereditary angioedema and to stop the progress in patients having acute attacks. Specific Aims and Procedures (summary): During the study you may receive 1 of 4 doses of DX-88. It will be given through an infusion over 10 minutes. The amount given is based on your body surface area, calculating your height and weight. Experimental Design (summary): Initially, 60 attacks in up to 60 patients will be studied at approx. 50 clinical centers in the United States and Europe. This may be increased to up to 240 attacks. Up to 2 doses of DX-88 will be given per attack. You can be treated for multiple attacks in this study. Significance (summary): There are currently no drugs approved to stop an attack of hereditary angioedema in the US and some European countries. The drugs that are available treat symptoms only.