AUGMENTATION THERAPY--CONGENITAL ALPHA1 PROTEINASE INHIB

这个多中心的D类研究已经完成。该研究旨在评估一种新药ATC 97-01，旨在增强α -1抗胰蛋白酶活性，这种重要的血浆酶具有遗传性缺陷。将新药与现有制剂（促生素）进行比较。患者被随机分配到新药或比较药物中，为期10周，之后所有受试者接受新药治疗，总研究时间为两年。在过去的十年中，对于遗传性α -1抗胰蛋白酶缺乏症患者的治疗有了显著的改善。已经证明，巴氏灭菌的混合人类产品可以将血浆α -1抗胰蛋白酶水平提高到生物学显著水平。除了这一生化证据外，两项大型研究表明该疗法具有临床疗效。欧洲的一项研究表明，增强治疗影响肺功能的重要参数，最近，美国的一项研究表明，接受增强治疗的遗传性α -1抗胰蛋白酶缺乏症患者的生存优势。由于巴氏灭菌的人类酶的供应不足以满足患者的需求，因此开发了一种新产品，本研究正在测试该产品。新产品也是由汇集的人体血浆制成，使用溶剂洗涤剂处理方法制备，与现有制剂相比，大大提高了病毒安全性。

This multi-center, category D study has been completed. The study was designed to evaluate a new drug, ATC 97-01, designed to augment alpha-1 antitrypsin activity in patients with an inherited deficiency in this important plasma enzyme. The new drug was compared with an existing formulation (Prolastin). Patients were randomly allocated to the new drug or the comparator drug for ten weeks, after which all subjects received the new drug for a total study duration of two years. Over the last ten years, there have been significant improvements in the therapy of patients with inherited deficiencies of alpha-1 antitrypsin. It has been demonstrated that pasteurized, pooled human products can increase plasma levels of alpha-1 antitrypsin to biologically significant levels. In addition to this biochemical evidence, two large studies have suggested that the therapy has clinical efficacy. A European study demonstrated that augmentation therapy affected important parameters of lung function and, more recently, a study done in the United States demonstrated a survival advantage for patients receiving augmentation therapy for their inherited deficiency of alpha-1 antitrypsin. Because the supply of pasteurized, pool human enzyme has not been sufficient to meet patient demands, a new product was developed, and this study is testing that product. The new product, also made from pooled human plasma, is prepared using a solvent detergent treatment method, which greatly enhances viral safety over existing preparations.