EUR-1009-M

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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. Ewand S.p. A has designed an open-label, multiple-dose, single-treatment, multicenter, clinical study in younger patients with CF and Exocrine Pancreatic Insufficiency (EPI) (age younger than 7 years) to determine the efficacy and safety of Eurand PEP (Pancreatic Eszyme Product) microtabs in young CF patients with pancreatic insufficiency. The study population of subjects will be enrolled among approximately 10 sites in the United States. Patients will receive approximately 2,000 units/kg/meal (not to exceed 10,000 lipase units/kg/day). These doses are within the recommended range for CF patients with EPI. Although Eurand PEP Microtabs are available in microtablets containing 5,000 lipase units/capsule and minitablets containing 10,000, 15,000, or 20,000 lipase units/capsule, patients will receive Eurand PEP Microtabs only. This study design involves a 4-day screening period, a 7-day dose-stabilization period, and a 7-day treatment period. The primary efficacy endpoint is the percentage of "responders" that is, patients in whom sign and symptoms and steatorrhea are not present after administration of Eurand PEP Microtabs. The percentage and 95% confidence interval will be presented. The fecal fat content change from baseline at Days 11 and 18 will be tested using the Wilcoxon signed-rank test. The incidence of clinical symptoms of EPI following oral administration of Eurand PEP Microtabs will be summarized. The patient's condition after treatment with Eurand PEP Microtabs will be listed and summarized based on the physician's and parent's judgment. The results of this study will provide important and overdue information regarding both the safety and efficacy of this product in young children with CF and EPI.