Efficacy and Safety of ULTRASE MT20 in Improving the Coefficient of Fat Absorption (CFA%) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)
This protocol will enroll children aged 7 to 11 years old, suffering from Cystic Fibrosis and Pancreatic Insufficiency in order to demonstrate the safety of Ultrase MT20 as well as the efficacy of this product in the improvement of the fat absorption.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Efficacy and Safety of ULTRASE MT20 in Improving the Coefficient of Fat Absorption (CFA%) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)|
- Coefficient of fat absorption (CFA%) [ Time Frame: The CFA% will be assessed with a 72 hour stool collection ]
- Coefficient of Nitrogen absorption (CNA%) and Safety of Ultrase MT20 [ Time Frame: The CNA% will be assessed with a 72 hour stool collection and the safety will be assessed with laboratory testing, recording of all AEs throughout the study and recording of all SAEs until 30 days after the last visit. ]
|Study Start Date:||July 2007|
|Study Completion Date:||April 2008|
|Primary Completion Date:||March 2008 (Final data collection date for primary outcome measure)|
Experimental: Treatment phase
During the treatment phase, the patient will eat a high fat diet and will take ULTRASE MT20 at a specific daily dose to treat his pancreatic insufficiency.
Drug: ULTRASE MT20
A stabilized dose of ULTRASE MT20 in capsules will be dispensed with each meal and snack during all days of the treatment phase.
This study will include 3 phases: the screening phase, the washout phase and the treatment phase.
Screening Phase: this phase will last fifteen (15) days and all patients will take ULTRASE MT20 during this period. During the last 4 days, patents will be stabilized on a high fat diet and with Ultrase MT20.
Washout Phase: this phase will last 6 to 7 days. The patient will continue the high-fat diet but WILL REFRAIN from taking ULTRASE MT20 or any other enzymes. A 72-hour stool collection will be performed and all food consumed by the patient will be recorded to assess the Coefficient of Fat Absorption (CFA%) and the Coefficient of Nitrogen Absorption (CNA%).
Treatment Phase: this phase will last 7 to 11 days. The patient will continue the high-fat diet and will take the 'Stabilized Dose' of ULTRASE MT20 established during screening. Another 72-hour stool collection will be performed and all food consumed by the patient will be recorded to assess the Coefficient of Fat Absorption (CFA%) and the Coefficient of Nitrogen Absorption CNA%).
|United States, Michigan|
|University of Michigan Health System Cystic Fibrosis Center|
|Ann Arbor, Michigan, United States, 48109-0212|
|United States, Ohio|
|Cystic Fibrosis Center Rainbow Babies and Children's HospitalDivision|
|Cleveland, Ohio, United States, 44106|
|United States, Pennsylvania|
|Pennsylvania State University And the Milton S. Hershey Medical Center|
|Hershey, Pennsylvania, United States, 17033|
|Principal Investigator:||Michael Konstan, MD||Rainbow Baby- University hospital of Cleveland|
|Study Director:||Jean Spénard, Ph.D||Axcan Pharma Inc.|