CORRECTION OF STEATORRHEA IN PATIENS WITH CYSTIC FIBROSIS

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。子项目和 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以在其他 CRISP 条目中表示。列出的机构是 对于中心来说，它不一定是研究者的机构。 这是一项多中心、III 期研究，旨在证明 Ultrase MT20 新配方对于纠正患有胰腺功能不全的囊性纤维化 (CF) 患者的脂肪泻的功效。本研究的主要目的是证明 AXCAN Pharma Inc. Ultrase MT20 安全有效。这项研究的最终目标是获得 FDA 的 NDA 批准，以继续营销 Ultrase MT 产品线。已经上市且已使用很长时间的产品需要提交新药申请（NDA），源于 FDA 在 2004 年 4 月发布的指南草案中的要求。新药申请流程是根据 FDA 的调查结果制定的，即目前在美国销售的酶产品之间的药物效力存在重大差异，这可能会严重影响这些药物的安全性和有效性。 为了对此进行评估，患者被送入 GCRC 接受 2 个疗程，每个疗程持续 6 至 7 天，以便进行安慰剂对照比较。 请列出您的研究目的和目标。

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 a multicenter, Phase III study, to demonstrate the efficacy of a new formulation of Ultrase MT20 for the correction of steatorrhea in cystic fibrosis (CF) patients suffering from pancreatic insufficiency. The main rationale for this study is to demonstrate that AXCAN Pharma Inc. Ultrase MT20 is safe and effective. The ultimate goal of this study is to get an NDA approval from the FDA for the continued marketing of the Ultrase MT products line. The need to submit an NDA for products that are already on the market and that have been in use for a very long time originates from the FDA requirements published in a draft Guidance in April 2004. The NDA process has been put in place based on the FDA findings about substantial variations in drug potency among enzyme products currently marketed in the US, which could significantly affect the safety and effectiveness of these drugs. In order to evaluate this, patients are admitted to the GCRC for 2 periods of treatment lasting 6 to 7 days each in order to do a placebo-controlled comparison. Please list the purpose and objectives of your research.