Safety and Efficacy Study of ULTRASE® MT20 in Participants With Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (PI)
The purpose of this study is to assess the safety and efficacy of Ultrase® MT20 compared to placebo for the correction of fat and protein malabsorption in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI). This study is sponsored by Aptalis Pharma (formerly Axcan).
Exocrine Pancreatic Insufficiency
Drug: Ultrase® MT20
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
|Official Title:||A Multicenter, Randomized, Double-Blind, Crossover Study to Compare the Safety and Efficacy of Ultrase® MT20 to Placebo for the Correction of Steatorrhea in Patients With Cystic Fibrosis (CF)|
- Percent Coefficient of Fat Absorption (CFA) [ Time Frame: Day 3 to Day 7 in first intervention period and second intervention period ] [ Designated as safety issue: No ]Percent (%) CFA was calculated as ([fat intake - fat excretion]/fat intake)*100, determined by the stools collected during the 72-hour period which could extend to 96 hours during both intervention periods. Mean CFA percent was calculated for 72-hour/96-hour period during Day 3 to Day 7 in the first and second intervention periods.
- Percent Coefficient of Nitrogen Absorption (CNA) [ Time Frame: Day 3 to Day 7 in first intervention period and second intervention period ] [ Designated as safety issue: No ]Percent (%) CNA was calculated as [(nitrogen intake-nitrogen excretion)/nitrogen intake]*100, determined by the stools collected during the 72-hour period which could extend to 96 hours during both intervention periods. Nitrogen intake was calculated as protein intake/6.25. Mean percent CNA was calculated for 72-hour/96-hour period during Day 3 to Day 7 in the first and second intervention periods.
- Number of Bowel Movements [ Time Frame: Day 3 on first intervention period and second intervention period ] [ Designated as safety issue: No ]Number of bowel movements of each participant was calculated from frequency of stools by the participant per day. Mean daily number of bowel movements on Day 3 for the first treatment period and second treatment period was summarized.
- Percentage of Stool Categorized by Consistency [ Time Frame: Day 4 on first intervention period and second intervention period ] [ Designated as safety issue: No ]Stool consistency was categorized as hard, formed/normal, soft or watery stool. Percentage of stools of a specific consistency of each participant was calculated as the number of stools with a specific consistency relative to the total number of stools during the collection period. Mean percentage of stool with specific consistency on Day 4 for the first treatment period and second treatment period period for total participants was summarized.
|Study Start Date:||November 2006|
|Study Completion Date:||April 2007|
|Primary Completion Date:||April 2007 (Final data collection date for primary outcome measure)|
|Experimental: Ultrase® MT20||
Drug: Ultrase® MT20
Ultrase® MT 20 capsules containing enteric-coated minitablets orally daily at a dose stabilized during the first stabilization period (4 days), as per investigator's discretion, for 6 to 7 days in either first intervention period or second intervention period.
Other Name: Pancreatic enzyme product
|Placebo Comparator: Placebo||
Placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in either first intervention period or second intervention period.
This is a Phase III, multicenter, randomized, double-blind, two-period cross-over, placebo-controlled study designed to compare the efficacy and safety of Ultrase® MT20 to placebo in participants with CF and pancreatic insufficiency. The study consists of a screening period (up to 11 days) and two treatment periods (6-7 days). During screening period participants will be treated with open-label Ultrase® MT18 or MT20. Each treatment period will be preceded by a stabilization period (4 days) and the two treatment periods are separated by a break period (3-6 days). A safety follow-up visit will be performed 7-10 days after discharge from the last treatment period .
|United States, Michigan|
|DeVos Children's Hospital|
|Grand Rapids, Michigan, United States, 49503|
|United States, Ohio|
|Rainbow Babies & Children's Hospital|
|Cleveland, Ohio, United States, 44106|
|United States, Pennsylvania|
|Pennsylvania State University, The Milton S. Hershey Medical Center|
|Hershey, Pennsylvania, United States, 17033|
|United States, Utah|
|University of Utah Health Sciences Center|
|Salt Lake City, Utah, United States, 84112|
|Study Director:||Aptalis Medical Information||Aptalis Pharma|