Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
Primary Purpose
Cystic Fibrosis, Pancreatic Exocrine Insufficiency
Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Pancrelipase Delayed Release
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis focused on measuring Cystic Fibrosis, Pancreatic Exocrine Insufficiency
Eligibility Criteria
Inclusion Criteria:
- Confirmed Cystic Fibrosis (CF) diagnosis by two positive chloride sweat tests or gene analysis
- Current or historical human fecal elastase < 50µg/gstool
- Weight greater than 3.75 kg
- Age 1 month to 6 years
- 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
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
Sites / Locations
- Site 11
- Site 5
- Site 9
- Site 6
- Site 12
- Site 4
- Site 13
- Site 8
- Site 1
- Site 10
- Site 7
- Site 3
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
A
Arm Description
Outcomes
Primary Outcome Measures
Number of Patients With at Least One Treatment Emergent Adverse Event (TEAE)
Treatment Emergent Adverse Events are defined as adverse events started at or after the first administration of study drug and include those events started prior to the first administration but which worsened after the first intake.
Secondary Outcome Measures
Stool Fat (% Fat)
The stool fat content was calculated as percent fat of dry solid weight per bowel movement. Stool fat per patient was derived as mean over three bowel movements sampled (i.e. one sample per day).
Fat Intake (g)
The mean daily fat intake was determined as the average of daily fat intake over a 3-day period.
Total Calorie Intake (kcal)
The total calorie intake was determined as the average of total calorie of daily food intake intake over a 3-day period.
Full Information
NCT ID
NCT00775528
First Posted
October 17, 2008
Last Updated
October 25, 2010
Sponsor
Solvay Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT00775528
Brief Title
Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
Official Title
An Open Label, Multi-center, Study to Assess the Safety and Tolerability of Pancrelipase Delayed Release Capsules in Infants and Children Less Than 7 Years of Age With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
Study Type
Interventional
2. Study Status
Record Verification Date
October 2010
Overall Recruitment Status
Completed
Study Start Date
April 2009 (undefined)
Primary Completion Date
June 2009 (Actual)
Study Completion Date
June 2009 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
Solvay Pharmaceuticals
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This study will assess the safety and tolerability of pancrelipase delayed release capsules in subjects up to 6 years of age with Pancreatic Exocrine Insufficiency (PEI) due to Cystic Fibrosis.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis, Pancreatic Exocrine Insufficiency
Keywords
Cystic Fibrosis, Pancreatic Exocrine Insufficiency
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
19 (Actual)
8. Arms, Groups, and Interventions
Arm Title
A
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Pancrelipase Delayed Release
Intervention Description
3,000, 6,000 and 12,000 unit Lipase Capsules
Primary Outcome Measure Information:
Title
Number of Patients With at Least One Treatment Emergent Adverse Event (TEAE)
Description
Treatment Emergent Adverse Events are defined as adverse events started at or after the first administration of study drug and include those events started prior to the first administration but which worsened after the first intake.
Time Frame
10 Days
Secondary Outcome Measure Information:
Title
Stool Fat (% Fat)
Description
The stool fat content was calculated as percent fat of dry solid weight per bowel movement. Stool fat per patient was derived as mean over three bowel movements sampled (i.e. one sample per day).
Time Frame
Last 3 days in a 10-day treatment period
Title
Fat Intake (g)
Description
The mean daily fat intake was determined as the average of daily fat intake over a 3-day period.
Time Frame
Last 3 days in a 10-day treatment period
Title
Total Calorie Intake (kcal)
Description
The total calorie intake was determined as the average of total calorie of daily food intake intake over a 3-day period.
Time Frame
Last 3 days in a 10-day treatment period
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Month
Maximum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Confirmed Cystic Fibrosis (CF) diagnosis by two positive chloride sweat tests or gene analysis
Current or historical human fecal elastase < 50µg/gstool
Weight greater than 3.75 kg
Age 1 month to 6 years
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
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Global Clinical Director Solvay
Organizational Affiliation
Solvay Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Site 11
City
Boise
State/Province
Idaho
Country
United States
Facility Name
Site 5
City
Louisville
State/Province
Kentucky
Country
United States
Facility Name
Site 9
City
Boston
State/Province
Massachusetts
Country
United States
Facility Name
Site 6
City
Ann Arbor
State/Province
Michigan
Country
United States
Facility Name
Site 12
City
Detroit
State/Province
Michigan
Country
United States
Facility Name
Site 4
City
Minneapolis
State/Province
Minnesota
Country
United States
Facility Name
Site 13
City
Long Branch
State/Province
New Jersey
Country
United States
Facility Name
Site 8
City
Albuquerque
State/Province
New Mexico
Country
United States
Facility Name
Site 1
City
Cincinnati
State/Province
Ohio
Country
United States
Facility Name
Site 10
City
Oklahoma City
State/Province
Oklahoma
Country
United States
Facility Name
Site 7
City
Oklahoma City
State/Province
Oklahoma
Country
United States
Facility Name
Site 3
City
Hershey
State/Province
Pennsylvania
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
20441244
Citation
Graff GR, McNamara J, Royall J, Caras S, Forssmann K. Safety and tolerability of a new formulation of pancrelipase delayed-release capsules (CREON) in children under seven years of age with exocrine pancreatic insufficiency due to cystic fibrosis: an open-label, multicentre, single-treatment-arm study. Clin Drug Investig. 2010;30(6):351-64. doi: 10.2165/11533390-000000000-00000.
Results Reference
background
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Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
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