An Effectiveness, Safety, and Palatability Study of Pancrelipase Microtablets in Infants and Toddlers With Cystic Fibrosis and Fat Malabsorption
Primary Purpose
Cystic Fibrosis, Steatorrhea
Status
Completed
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Pancrelipase microtablets
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis focused on measuring Cystic fibrosis, Pancreatic insufficiency, Steatorrhea, Fat malabsorption, Pancrelipase
Eligibility 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
Sites / Locations
Outcomes
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 & weight. Mean daily calories, fat intake. Coefficient fat absorption
Full Information
NCT ID
NCT00217204
First Posted
September 13, 2005
Last Updated
May 17, 2011
Sponsor
McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT00217204
Brief Title
An Effectiveness, Safety, and Palatability Study of Pancrelipase Microtablets in Infants and Toddlers With Cystic Fibrosis and Fat Malabsorption
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 Type
Interventional
2. Study Status
Record Verification Date
April 2010
Overall Recruitment Status
Completed
Study Start Date
July 2005 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
February 2006 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.
4. Oversight
5. Study Description
Brief Summary
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
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis, Steatorrhea
Keywords
Cystic fibrosis, Pancreatic insufficiency, Steatorrhea, Fat malabsorption, Pancrelipase
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
Single
Allocation
Randomized
Enrollment
18 (Actual)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Pancrelipase microtablets
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
Clinical Global Impression Severity Subscale. Global Change Subscale. Weight gain/loss. Global Assessment effectiveness. Nitrogen excretion/24 hr. Mean dose of pancrease assessed/day & weight. Mean daily calories, fat intake. Coefficient fat absorption
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
30 Months
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
McNeil Consumer & Specialty Pharmaceuticals, a Division of Mc Neil-PPC, Inc. Clinical Trial
Organizational Affiliation
McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.
Official's Role
Study Director
12. IPD Sharing Statement
Links:
URL
http://filehosting.pharmacm.com/DownloadService.ashx?client=CTR_JNJ_6051&studyid=907&filename=CR002665_CSR.pdf
Description
An Effectiveness, Safety, and Palatability Study of Pancrelipase Microtablets in Infants and Toddlers With Cystic Fibrosis and Fat Malabsorption
Learn more about this trial
An Effectiveness, Safety, and Palatability Study of Pancrelipase Microtablets in Infants and Toddlers With Cystic Fibrosis and Fat Malabsorption
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