Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency (PEI) Due to Cystic Fibrosis (CF)

A Double-blind, Randomized, Multi-center, Placebo-controlled, Cross-over Study to Assess the Efficacy and Safety of Pancrelipase Delayed Release 12,000 Unit Capsules in Subjects Aged 7 - 11 With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis

This study will assess the effect of pancrelipase delayed release 12,000 unit capsules on fat and nitrogen absorption in subjects 7 - 11 with pancreatic exocrine insufficiency due to Cystic Fibrosis.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis; Pancreatic Exocrine Insufficiency
• Intervention / Treatment: Drug: Placebo Comparator; Drug: Pancrelipase Delayed Release

• Study Type: Interventional
• Enrollment (Actual): 17
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Iowa
    • Iowa City, Iowa, United States
      • Site 2
  • Kentucky
    • Louisville, Kentucky, United States
      • Site 5
  • Massachusetts
    • Boston, Massachusetts, United States
      • Site 9
  • Michigan
    • Ann Arbor, Michigan, United States
      • Site 6
  • Minnesota
    • Minneapolis, Minnesota, United States
      • Site 4
  • New Mexico
    • Albuquerque, New Mexico, United States
      • Site 8
  • Ohio
    • Cincinnati, Ohio, United States
      • Site 1
  • Oklahoma
    • Oklahoma City, Oklahoma, United States
      • Site 10
    • Oklahoma City, Oklahoma, United States
      • Site 7
  • Pennsylvania
    • Hershey, Pennsylvania, United States
      • Site 3

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 years to 11 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Confirmed CF diagnosis by two positive chloride sweat tests or gene analysis
• Confirmed PEI by historical Coefficient of fat Absorption < 70% without supplementation or current or historical fecal elastase < 50µg/stool (within the last 12 months)
• Currently receiving treatment with a commercially available pancreatic enzyme product on a stable dose for more than 3 months
• Clinically stable condition without evidence of acute respiratory disease or any other acute condition
• Stable body weight and agrees to abstain from sexual activity

Exclusion Criteria:

• Ileus or acute abdomen
• History of fibrosing colonopathy, celiac disease, gastrectomy, Crohn´s disease and small bowel surgery other than minor resection due to meconium ileus without resulting in malabsorption syndrome
• History of distal ileal obstruction syndrome within 6 months of enrollment
• Use of an immunosuppressive drug
• Any type of malignancy involving the digestive tract in the last 5 years
• Known infection with HIV

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: A	Drug: Pancrelipase Delayed Release; 12,000 unit Capsules, dosed individually based on fat intake.
Placebo Comparator: B	Drug: Placebo Comparator; Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Coefficient of Fat Absorption (%)	This coefficient is calculated from fat intake and fat excretion : 100*[fat intake-fat excretion]/fat intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response.	5 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Coefficient of Nitrogen Absorption (%)	This coefficient is calculated from nitrogen intake and nitrogen excretion : 100*[nitrogen intake-nitrogen excretion]/nitrogen intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response.	5 days
Total Fat Excretion (Grams)	Total amount of fat excreted during the stool collection period. Stools were collected on 3 days during the 5 days treatment period. Lower values indicate a better response.	5 days
Total Stool Weight (Grams)	Total weight of the stools collected during the stool collection period. Stools were collected on 3 days during the 5 days treatment period. Lower values indicate a better response.	5 days
Stool Frequency	Stool frequency is the average of the daily number of stools recorded during the treatment period. Lower values indicate a better response.	5 days
Percentage of Days With no Flatulence.	The percentage of days with no flatulence is calculated from the diary during the treatment period: 100*(number of days with no flatulence/number of days recorded in diary). Higher values indicate a better response.	5 days
Percentage of Days With Formed/Normal Stools.	The percentage of days with formed/normal stools is calculated from the diary during the treatment period: 100*(number of days with formed/normal stools/number of days with any stool). Higher values indicate a better response.	5 days
Percentage of Days With no Abdominal Pain.	The percentage of days with no abdominal pain is calculated from the diary during the treatment period: 100*(number of days with no abdominal pain / number of days recorded in diary). Higher values indicate a better response.	5 days

Collaborators and Investigators

• Sponsor: Solvay Pharmaceuticals

Publications and helpful links

General Publications

• Graff GR, Maguiness K, McNamara J, Morton R, Boyd D, Beckmann K, Bennett D. Efficacy and tolerability of a new formulation of pancrelipase delayed-release capsules in children aged 7 to 11 years with exocrine pancreatic insufficiency and cystic fibrosis: a multicenter, randomized, double-blind, placebo-controlled, two-period crossover, superiority study. Clin Ther. 2010 Jan;32(1):89-103. doi: 10.1016/j.clinthera.2010.01.012.
• Caras S, Boyd D, Zipfel L, Sander-Struckmeier S. Evaluation of stool collections to measure efficacy of PERT in subjects with exocrine pancreatic insufficiency. J Pediatr Gastroenterol Nutr. 2011 Dec;53(6):634-40. doi: 10.1097/MPG.0b013e3182281c38.

Study record dates

Study Major Dates

• Study Start: June 1, 2008
• Primary Completion (Actual): December 1, 2008
• Study Completion (Actual): December 1, 2008

Study Registration Dates

• First Submitted: June 3, 2008
• First Submitted That Met QC Criteria: June 4, 2008
• First Posted (Estimate): June 5, 2008

Study Record Updates

• Last Update Posted (Estimate): June 2, 2010
• Last Update Submitted That Met QC Criteria: May 26, 2010
• Last Verified: February 1, 2010

More Information

Terms related to this study

• Keywords: Cystic Fibrosis; Pancreatic Exocrine Insufficiency
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Gastrointestinal Agents; Pancrelipase; Pancreatin
• Other Study ID Numbers: S245.3.127