CORRECTION OF STEATORRHEA IN PATIENS WITH CYSTIC FIBROSIS

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 这是一项多中心的第三阶段研究，旨在证明一种新的Ultrase MT20配方对纠正患有胰腺功能不全的囊性纤维化(CF)患者的脂肪泻的疗效。这项研究的主要理由是证明Axcan Pharma Inc.Ultrase MT20是安全有效的。这项研究的最终目标是获得FDA的NDA批准，继续营销Ultrase MT产品线。需要为已经上市和使用了很长时间的产品提交保密协议，这源于2004年4月在指南草案中公布的FDA要求。NDA程序是基于FDA的调查结果，即目前在美国销售的酶产品之间的药物效力存在显著差异，这可能会显著影响这些药物的安全性和有效性。 为了评估这一点，患者进入GCRC接受两个疗程的治疗，每个疗程持续6至7天，以便进行安慰剂对照比较。请列出您研究的目的和目标。

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on the market and that have been in use for a very long time originates from the FDA requirements published in a draft Guidance in April 2004.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.