ORAL THERACLECTM-TOTAL IN CYSTIC FIBROSIS SUBJECTS WITH PANCREATIC INSUFFICIENCY

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。这是一项国际性、多中心、随机、双盲的TheraCLEC-Total和Creon的平行剂量范围口服TheraCLEC-Total研究，用于囊性纤维化(CF)伴胰腺外分泌功能不全(PI)患者。这项第二阶段研究将测试TheraCLEC-Total S活性成分(脂肪酶[主要终点]、蛋白酶和淀粉酶[次要终点])的安全性和有效性终点。将对TheraCLEC-Total的三种剂量进行研究，试图确定最低有效剂量，从而优化第三阶段研究药物剂量的选择。作为次要目标，TheraCLEC-Total将与活性比较器Creon？进行比较。在80名患者完成随机治疗后，将进行中期分析。

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 an international, multicenter, randomized, double blind for TheraCLEC-Total and single blind for Creon, parallel dose ranging study of oral TheraCLEC-Total in cystic fibrosis (CF) subjects with exocrine pancreatic insufficiency (PI). This Phase 2 study will test the safety of TheraCLEC¿-Total and efficacy endpoints for each of TheraCLEC-Total¿s active components (lipase [primary endpoint], protease and amylase [secondary endpoints]). Three doses of TheraCLEC-Total will be studied in an attempt to identify the lowest effective dose, thereby optimizing the choice of study drug dose in Phase 3. As a secondary objective, TheraCLEC-Total will be compared to the active comparator, Creon¿. An interim analysis will be performed after 80 patients complete the randomized Treatment Period.