CORRECTION OF STEATORRHEA IN PATIENS WITH CYSTIC FIBROSIS

该子项目是利用 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH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 中心，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 MT 20新制剂在患有胰腺功能不全的囊性纤维化（CF）患者中纠正脂肪堆积的疗效。本研究的主要依据是证明AXCAN Pharma Inc. Ultrase MT 20安全有效。本研究的最终目标是获得FDA的NDA批准，以继续销售Ultrase MT产品线。对于已经上市并已使用很长时间的产品，需要提交NDA，这源于FDA在2004年4月指南草案中发布的要求。NDA过程是根据FDA关于目前在美国销售的酶产品之间药物效力的实质性变化的发现而实施的，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进行2个治疗期，每个治疗期持续6至7天，以进行安慰剂对照比较。 请列出你的研究目的和目标。

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on the market and that have been in use for a very long time originates from the FDA requirements published in a draft Guidance in April 2004.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