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

Sponsored by:	McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.
Information provided by:	McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.
ClinicalTrials.gov Identifier:	NCT00217204

Purpose

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

Condition	Intervention	Phase
Steatorrhea Cystic Fibrosis	Drug: pancrelipase	Phase II

Genetics Home Reference related topics: cystic fibrosis

MedlinePlus related topics: Cystic Fibrosis

Drug Information available for: Pancrelipase Ultrase

U.S. FDA Resources

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

Further study details as provided by McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.:

Primary Outcome 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 Outcome 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.

Estimated Enrollment:	20
Study Start Date:	July 2005
Study Completion Date:	February 2006

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.

Eligibility

Ages Eligible for Study:	6 Months to 30 Months
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00217204

Sponsors and Collaborators

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

Investigators

Study Director:

McNeil Consumer & Specialty Pharmaceuticals Clinical Trial

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

More Information

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

This link exits the ClinicalTrials.gov site

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

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

Study ID Numbers:	CR002665
Study First Received:	September 13, 2005
Last Updated:	March 6, 2008
ClinicalTrials.gov Identifier:	NCT00217204
Health Authority:	United States: Institutional Review Board

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

Fat malabsorption
Pancreatic insufficiency
Cystic fibrosis
Steatorrhea
Pancrelipase

Study placed in the following topic categories:

Metabolic Diseases
Fibrosis
Gastrointestinal Diseases
Intestinal Diseases
Pancrelipase
Digestive System Diseases
Cystic Fibrosis
Respiratory Tract Diseases
Genetic Diseases, Inborn

Lung Diseases
Malabsorption Syndromes
Pancreatic Diseases
Infant, Newborn, Diseases
Metabolic disorder
Cystic fibrosis
Exocrine Pancreatic Insufficiency
Steatorrhea

Additional relevant MeSH terms:

Pathologic Processes
Therapeutic Uses
Gastrointestinal Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 16, 2009