CLINICAL TRIAL: CORRECTION OF STEATORRHEA IN PATIENS WITH CYSTIC FIBROSIS

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on the market and that have been in use for a very long time originates from the FDA requirements published in a draft Guidance in April 2004.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