An Effectiveness, Safety, and Palatability Study of Pancrelipase Microtablets in Infants and Toddlers With Cystic Fibrosis and Fat Malabsorption

Condition(s) targeted: Steatorrhea; Cystic Fibrosis

Intervention: pancrelipase (Drug)

Phase: Phase 2

Status: Completed

Sponsored by: McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.

Official(s) and/or principal investigator(s):
McNeil Consumer & Specialty Pharmaceuticals Clinical Trial, Study Director, Affiliation: McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.

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

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

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

Primary outcome: 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 outcome:

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.

Detailed description: 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.

Minimum age: 6 Months. Maximum age: 30 Months. Gender(s): Both.

Criteria:

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

For FDA Approved Product labeling, refer to the following link:http://www.accessdata.fda.gov/scripts/cder/drugsatfda/

Additional information is provided at the following link;http://dailymed.nlm.nih.gov/dailymed/about.cfm

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

Starting date: July 2005
Ending date: February 2006
Last updated: March 6, 2008