STAMP

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。子项目和 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。 本研究设计为一项多中心、开放标签、为期20周的试验，旨在评价Alpha-1 MP在患有Alpha 1-抗胰蛋白酶缺乏症的成人受试者中的安全性和耐受性。 本研究将在北美和欧洲的约12个中心进行。本研究将入组共计35例成人受试者，其中至少15例非研究受试者，即，既往从未接受过α-1-蛋白酶抑制剂强化治疗的受试者。参与研究的预期持续时间为20周每周一次治疗，加上最多1周的筛选，加上末次治疗后7天的电话随访，以及末次治疗后4周的最终访视。在治疗期间，入组的研究受试者将接受20次每周一次的α-1 MP输注。由于α-1-蛋白酶抑制剂的半衰期约为5天，因此入组研究前不需要α-1-蛋白酶抑制剂停药期，患者无需中断每周一次的治疗来参加研究。将在输注前和输注后采集血样，并在基线、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）进行病毒筛查。此外，将在基线（第1周）和治疗期结束后1个月（第24周）采集血液样本进行免疫原性试验。如果在受试者的血液样本中检测到对α-1-蛋白酶抑制剂的任何免疫遗传学应答，则还将对该受试者的样本进行α-1-蛋白酶抑制剂中和抗体测定。整个临床研究的预期持续时间为51周。

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 as a multi-center, open-label, 20-week trial to evaluate the safety and tolerability of Alpha-1 MP in adult subjects with Alpha1-antitrypsin deficiency. This study will be conducted in approximately 12 centers in North America and Europe. A total of 35 adult subjects will be enrolled in the study with a minimum of 15 nanve subjects, i.e., those subjects that have never received previous Alpha-1-Proteinase Inhibitor augmentation therapy. The expected duration of study participation is 20 weeks of weekly treatment, plus up to 1 week for screening, plus a follow-up phone call which will occur 7 days following the last treatment, and a final visit which will occur 4 weeks following the last treatment. During the treatment period, enrolled study subjects will receive 20 weekly infusions of Alpha-1 MP. Since the half-life of Alpha-1-Proteinase Inhibitor is approximately 5 days, an Alpha-1-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 (Week 16), end of treatment (Week 20), and 1 month following the end of treatment (Week 24). In addition, blood samples will be 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 Alpha-1-Proteinase Inhibitor is detected in a subject's blood sample, an Alpha-1-Proteinase Inhibitor neutralizing antibody assay will also be performed on that subject's sample. The expected duration of the entire clinical study is 51 weeks.