Official Title: “A Phase II, Randomized, Investigator-Blinded, Parallel-Group, Pilot Study Evaluating the Safety, Palatability and Effectiveness of Four Doses of Pancrelipase Microtablets in the Treatment of Infants and Toddlers With Cystic Fibrosis-Related Pancreatic Insufficiency and Fat Malabsorption”

The purpose of this study is to evaluate the effectiveness and safety of PANCREASE MT (pancrelipase microtablets) to improve steatorrhea (excessive excretion of fat in feces) in infants and toddlers with cystic fibrosis who have pancreatic insufficiency, and to assess whether the consistency of the microtablets is acceptable for swallowing in infants and toddlers

Study Type: Interventional

Study Design: Treatment, Randomized, Single Blind, Parallel Assignment, Safety/Efficacy Study

The objective of this randomized, Investigator-blinded, parallel group, multicenter, pilot study is to evaluate the preliminary safety, palatability and effectiveness of pancrelipase microtablets to improve fat absorption. The hypothesis is that PANCREASE MT will provide effective, safe and palatable pancreatic enzyme supplementation to be used for the treatment of fat malabsorption in a cohort of infants with cystic fibrosis-related pancreatic insufficiency. On Day 1 of the study, parents will be instructed to administer 500 units lipase/kg/meal for a full five days. Stool will be collected and analyzed during the last 72 hours of this baseline period. On Day 6 of the study, subjects will be randomly assigned to one of four treatment groups. Parents will be instructed to administer the appropriate dose for a full five days and stool will be collected and analyzed during the last 72 hours of this randomized treatment period.

Patients will receive PANCREASE MT 500 units lipase/kg/meal by mouth for a maximum of five doses per day for the first 120 hours. Patients will receive PANCREASE MT 500, 1000, 1500 or 2000 units lipase/kg/meal by mouth, for a maximum of five meals per day for the next 120 hours.

Intervention(s) in this Clinical Trial

• Drug: pancrelipase

Primary Measures

• Change in coefficient of fecal fat absorption from baseline to end of study period. Palatability. Percent carbon dioxide expired by 13C-mixed triglyceride breath test measuring exogenous lipase activity.

Secondary Measures

• Clinical Global Impression Severity Subscale
• Global Change Subscale. Weight gain/loss. Global Assessment effectiveness. Nitrogen excretion/24 hr. Mean dose of pancrease assessed/day and weight. Mean daily calories, fat intake. Coefficient fat absorption.

Inclusion Criteria:

• Diagnosis of cystic fibrosis
• Excessive discharge of fat in feces
• Stable patient requiring pancreatic enzyme therapy

Exclusion Criteria:

• No stable antibiotic therapy for small bowel overgrowth
• No hypersensitivity to pork products
• No use of prokinetics eg, metoclopramide or cisapride within the last 30 days
• No nasogastric feeding tube feeding
• No use of steroids
• No use of concomitant H2 blockers or proton pump inhibitors as concomitant therapy

Gender Eligibility for this Clinical Trial: Both

Minimum Age for this Clinical Trial: 6 Months

Maximum Age for this Clinical Trial: 30 Months

Are Healthy Volunteers Accepted for this Clinical Trial?: No

Lead Sponsor: McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.

Overall Clinical Trial Officials and Contacts

McNeil Consumer & Specialty Pharmaceuticals Clinical Trial Study Director McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.  

Information obtained from ClinicalTrials.gov on October 06, 2008

Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT00217204

Study ID Number: CR002665

ClinicalTrials.gov Identifier: NCT00217204

Health Authority: United States: Institutional Review Board

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For FDA Safety Alerts and Recalls refer to the following link:www.fda.gov/MEDWATCH/safety.htm