CLINICAL TRIAL: STAMP

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。 这项研究旨在评估Alpha-1 MP在患有Alpha1抗胰蛋白酶缺乏症的成人受试者中的安全性和耐受性，该研究将在北美和欧洲的大约12个中心进行。共有35名成年人将参加这项研究，至少有15名受试者，即那些以前从未接受过α1蛋白酶抑制剂增强治疗的受试者。参加研究的时间为每周治疗20周，筛查1周，并在最后一次治疗后7天进行电话随访最后一次访问将在最后一次治疗后4周进行。在治疗期间，入选的受试者将接受每周20次的Alpha-1 MP输注。由于Alpha1-Proteinase抑制剂的半衰期约为5天，因此在进入研究之前不需要Alpha1-Proteinase抑制剂的非治疗期，患者也不必中断每周的治疗来参与研究。血液样本将在输注前和输注后抽取，病毒筛查在基线、治疗开始后2个月(第8周)、治疗开始后4个月(第16周)、治疗结束(第20周)和治疗结束后1个月(第24周)进行。此外，在基线(第1周)和治疗结束后1个月(第24周)抽取血样进行免疫原性分析。如果在受试者S的血液样本中检测到对α1-蛋白水解酶抑制剂的免疫遗传反应，也将对该受试者S的血样进行α1-蛋白抑制物中和抗体检测。

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 study is designed to evaluate the safety and tolerability of Alpha-1 MP in adult subjects with Alpha1-antitrypsin deficiency and will be conducted in approximately 12 centers in North America and Europe. A total of 35 adults will be enrolled in the study with a minimum of 15 subjects, i.e., those subjects that have never received previous Alpha1-Proteinase Inhibitor augmentation therapy. The duration of study participation is 20 weeks of weekly treatment, 1 week for screening, and follow-up phone call, which will occur 7 days following the last treatment A final visit will occur 4 weeks following the last treatment. During the treatment period, enrolled subjects will receive 20 weekly infusions of Alpha-1 MP. Since the half-life of Alpha1-Proteinase Inhibitor is approximately 5 days, an Alpha1-Proteinase Inhibitor treatment-free period will not be required prior to study entry and patients will not have to interrupt their weekly treatments to participate in the study. Blood samples will be drawn pre- and post-infusion with viral screening conducted at baseline, 2 months following treatment initiation (Week 8), 4 months following treatment initiation (Week16) end of treatment (Week 20), and 1 month following the end of treatment (Week 24). Additionally, blood samples are drawn for the performance of immunogenicity assays at baseline (Week 1) and 1 month following the end of treatment period (Week 24). If any immunogenetic response to Alpha1-Proteinase Inhibitor is detected in a subject?s blood sample, an Alpha1-Proteinase Inhibitor neutralizing antibody assay will also be performed on that subject?s sample.