A Safety and Efficacy Study on ULTRASE MT20 in Patients With Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (PI) - Full Text View

Sponsor:	Axcan Pharma
Information provided by:	Axcan Pharma
ClinicalTrials.gov Identifier:	NCT00408317

Purpose

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.

Condition	Intervention	Phase
Cystic Fibrosis Exocrine Pancreatic Insufficiency	Drug: ULTRASE MT20	Phase III

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double-Blind, Placebo Control, Crossover Assignment, Safety/Efficacy Study
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).

Resource links provided by NLM:

Genetics Home Reference related topics: cystic fibrosis

MedlinePlus related topics: Cystic Fibrosis

Drug Information available for: Pancrelipase Ultrase

U.S. FDA Resources

Further study details as provided by Axcan Pharma:

Primary Outcome Measures:

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 Measures:

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.

Enrollment:	37
Study Start Date:	November 2006
Study Completion Date:	April 2007

Eligibility

Ages Eligible for Study:	7 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00408317

Locations

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

Sponsors and Collaborators

Axcan Pharma

Investigators

Principal Investigator:	Michael W Konstan, MD	Rainbow Babies & Children's Hospital, Cleveland, Ohio
Study Director:	Jean Spénard, Ph.D.	Axcan Pharma Inc., Mont-St-Hilaire, Québec, Canada

More Information

No publications provided

Study ID Numbers:	UMT20CF05-01
Study First Received:	December 4, 2006
Last Updated:	August 7, 2007
ClinicalTrials.gov Identifier:	NCT00408317 History of Changes
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Fibrosis
Gastrointestinal Agents
Pancrelipase
Pharmacologic Actions
Digestive System Diseases
Pathologic Processes
Cystic Fibrosis

Respiratory Tract Diseases
Genetic Diseases, Inborn
Lung Diseases
Therapeutic Uses
Pancreatic Diseases
Infant, Newborn, Diseases
Exocrine Pancreatic Insufficiency

ClinicalTrials.gov processed this record on February 08, 2010