A Safety and Efficacy Study on ULTRASE MT20 in Patients With Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (PI)
Information source: Axcan Pharma
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Cystic Fibrosis; Exocrine Pancreatic Insufficiency
Intervention: ULTRASE MT20 (Drug)
Phase: Phase 3
Status: Completed
Sponsored by: Axcan Pharma Official(s) and/or principal investigator(s):
Michael W Konstan, MD, Principal Investigator, Affiliation: Rainbow Babies & Children's Hospital, Cleveland, Ohio
Jean Spénard, Ph.D., Study Director, Affiliation: Axcan Pharma Inc., Mont-St-Hilaire, Québec, Canada
Summary
Cystic Fibrosis (CF) is a genetic disease resulting from the inheritance of a defective
autosomal recessive gene. It often results in exocrine pancreatic insufficiency (PI) that
leads to non-digestion of fats and proteins and finally to malabsorption of these nutrients.
The patients need to take pancreatic enzymes to allow absorption of these nutriments and this
has contributed to a significant increase in the life span of CF patients.
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 patients with CF and PI.
Clinical Details
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).
Study design: Treatment, Randomized, Double-Blind, Placebo Control, Crossover Assignment, Safety/Efficacy Study
Primary outcome: Compare the coefficient of fat absorption (CFA) of ULTRASE MT20 to placebo. The CFA will be measured by assessing the dietary fat intake and excretion during a 72-hour period.
Secondary outcome: Compare the coefficient of protein absorption (CNA) of ULTRASE MT20 to placebo. The CNA will be measured as the CFA. The safety will be evaluated from the laboratory analyses, the adverse events and the overall signs and symptoms.
Eligibility
Minimum age: 7 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Key Inclusion Criteria:
- Confirmed diagnosis of Cystic Fibrosis
- Patients must have pancreatic insufficiency and must require pancreatic enzyme
supplementation
- Patients must be 7 years and older
- Patients must be on an optimal clinical dose of pancreatic enzymes and must be
clinically stable
- Patients must have an adequate nutritional status
Key Exclusion Criteria:
- Patients with acute pancreatitis or acute exacerbation of chronic pancreatic disease
- Patients with an acute pulmonary infection
- Patients with a history of bowel resection
- Patients suffering from any dysmotility disorders
- Patients with chronic or severe abdominal pain
- Patients known to have a significant medical disease that would compromise their
welfare or confound the study results
- Patients with a history of or a current diagnosis of clinically significant portal
hypertension
- Patients who have a condition known to increase fecal fat loss
- Patients with a current diagnosis or a history of complete DIOS
- Patients with poorly controlled diabetes
- Female patients who are pregnant or lactating
Locations and Contacts
DeVos Children's Hospital, Grand Rapids, Michigan 49503, United States
Rainbow Babies & Children's Hospital, Cleveland, Ohio 44106, United States
Pennsylvania State University, The Milton S. Hershey Medical Center, Hershey, Pennsylvania 17033, United States
University of Utah Health Sciences Center, Salt Lake City, Utah 84112, United States
Additional Information
Starting date: November 2006
Ending date: April 2007
Last updated: August 7, 2007
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