Evaluation of the Safety and Efficacy of Pancrecarb® MS-16 in Cystic Fibrosis
The primary objective of this study is to determine if PANCRECARB® MS-16 (pancrelipase) is safe and effective in reducing steatorrhea (as measured by 72-hour stool fat determinations) in children and adults with cystic fibrosis and pancreatic insufficiency.
Drug: PANCRECARB® (pancrelipase)
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Randomized, Double-Blind, Placebo-Controlled, Multi-Center, Crossover Study to Evaluate the Effectiveness and Safety of PANCRECARB® MS-16 (Pancrelipase) in Reducing Steatorrhea in Children and Adults With Cystic Fibrosis|
- percent coefficient of fat absorption (% CFA) [ Time Frame: calculated from the 72-hour stool collection and dietary records ] [ Designated as safety issue: No ]
- percent coefficient of nitrogen absorption (% CNA) [ Time Frame: calculated from the 72-hour stool collections and dietary records ] [ Designated as safety issue: No ]
- change in stool frequency and stool weight [ Time Frame: recorded over the 72-hour stool collection period ] [ Designated as safety issue: No ]
|Study Start Date:||January 2007|
|Study Completion Date:||September 2007|
|Primary Completion Date:||September 2007 (Final data collection date for primary outcome measure)|
Active Comparator: 1
Pancrecarb(R) MS-16 Capsules
Drug: PANCRECARB® (pancrelipase)
Other Name: MS-16
|Placebo Comparator: 2||
Other Name: placebo
Pancreatic insufficiency (PI) is a common pathologic condition that occurs in approximately 90% of patients with cystic fibrosis (CF). Pancreatic insufficiency is characterized by both pancreatic enzyme and bicarbonate insufficiencies. Consequently, maldigestion occurs and a variety of essential nutrients are lost through the stools, especially fat and fat soluble vitamins. As a result, patients often experience growth failure and malnutrition. Effective correction of maldigestion is critical to the survival and well-being of these patients.
Several strengths of PANCRECARB® (pancrelipase) (i.e., MS4, MS8, MS16) have been available and used by patients with CF for more than a decade. The digestive enzymes in PANCRECARB® (pancrelipase) act locally in the gastrointestinal tract. The active enzymes hydrolyze fats into glycerol and fatty acids, proteins into peptides and amino acids, and starches into dextrins and maltose. PANCRECARB® (pancrelipase) has the potential to promote increased lipase activity with efficient fat digestion. Efficient fat digestion is important in CF because it may lead to improved nutritional and pulmonary status and ultimately to improved quality of life and enhanced survival.
PANCRECARB® MS-8 (pancrelipase) has been compared to enteric-coated pancreatic enzymes without bicarbonate for its efficacy in reducing steatorrhea in patients with CF. Differences in fat excretion, when subjects received PANCRECARB® MS-8 (pancrelipase) versus enteric-coated enzymes without bicarbonate, were compared using linear modeling. Mean fat excretion decreased significantly in subjects who received PANCRECARB® MS-8 (pancrelipase) compared to enteric-coated enzymes without bicarbonate.
This study has been designed in accordance with FDA 2006 guideline on exocrine pancreatic insufficiency drug products. Assuming that the results of this study demonstrate that therapy with PANCRECARB® MS-16 (pancrelipase) results in clinically and statistically significant improvement in fat absorption relative to placebo in subjects with CF and pancreatic insufficiency, the study results will be part of a submission for marketing approval of PANCRECARB® (pancrelipase).
The study consists of two treatment periods with 72-hour stool collections separated by a washout period. Study subjects will be required to consume a diet high in fat content.
|United States, Ohio|
|Rainbow Babies and Children's Hospital|
|Cleveland, Ohio, United States, 44106|
|Principal Investigator:||Michael W Konstan, MD||University Hospitals of Cleveland|