TYPE 1 DIABETES GENETICS CONSORTIUM

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。 这是一项单中心、随机、开放标签、剂量范围的研究，旨在确定不同剂量/方案的Baxter/Arriva Raat雾化治疗对下呼吸道上皮衬里液(ELF)AAT酶水平、AAT人中性粒细胞弹性酶复合体浓度、炎症介质和细胞学的影响，并评估不同剂量/方案在诊断为AAT严重缺乏的受试者中的安全性和免疫原性。这项研究将至少招收15名合格的受试者。 研究中的药物将通过吸入给药。这项研究中使用的输送装置将是AeroEclipse？呼吸驱动雾化器(BAN；Truedell Medical International，Inc，Inc.，London，Ontario，Canada)，与Pari ProNeb？-Ultra压缩机相结合。在Baxter/Arriva Raat溶液的测试中，该雾化器的空气动力学质量中值直径(MMAD)从1.4微米到2.3微米不等。对于200毫克剂量(雾化器装药)，雾化器产量平均为47%。

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. This is a single-center, randomized, open-label, dose-ranging study to characterize the effects of different doses/regimens of aerosolized Baxter/Arriva rAAT therapy on lower respiratory epithelial lining fluid (ELF) AAT enzyme levels, AAT human neutrophil elastase complex concentration, inflammatory mediators, and cytology, and to evaluate safety and immunogenicity of different doses/regimens in subjects with a diagnosis of severe AAT deficiency. A minimum of 15 qualified subjects will be enrolled in the study. The study drug will be administered by inhalation. The delivery device to be used in this study will be the AeroEclipse¿- Breath-Actuated Nebulizer (BAN; Truedell Medical International, Inc, London, Ontario, Canada), in conjunction with the PARI PRONEB¿- ULTRA compressor. In tests with the Baxter/Arriva rAAT solution, the mass median aerodynamic diameter (MMAD) for this nebulizer ranged from 1.4 to 2.3 microns. For a 200 mg dose (nebulizer charge), the nebulizer output averaged 47%.ii