STAMP

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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.