Use of Ultrase MT12 in Young Cystic Fibrosis Children - Full Text View

Sponsor:	Axcan Pharma
Information provided by:	Axcan Pharma
ClinicalTrials.gov Identifier:	NCT00880100

Purpose

Multicenter, explorative phase IIIb study designed to assess the efficacy of Ultrase MT12 in the control of steatorrhea and clinical signs and symptoms of malabsorption in cystic fibrosis children with pancreatic insufficiency.

Condition	Intervention	Phase
Cystic Fibrosis Pancreatic Insufficiency	Drug: Pancrelipase	Phase III

Study Type:	Interventional
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 MT12 in the Control of Steatorrhea in Cystic Fibrosis (CF) and Pancreatic Insufficient (PI) Children Aged 2 to 6 Years Old

Resource links provided by NLM:

Genetics Home Reference related topics: cystic fibrosis

MedlinePlus related topics: Cystic Fibrosis Malabsorption Syndromes

Drug Information available for: Pancrelipase Ultrase

U.S. FDA Resources

Further study details as provided by Axcan Pharma:

Primary Outcome Measures:

Proportion of subjects with control of steatorrhea under Ultrase MT12. [ Time Frame: Day 5 to Day 9 of Baseline Phase and Day 15 to Day 19 of Treatment Phase ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Stool frequency,consistency and characteristics under Ultrase MT12. Abdominal complaints under Ultrase MT12. Tolerability of Ultrase MT12. [ Time Frame: At the end of Baseline Phase and the end of Treatment Phase. Tolerability will be assessed throughout the study. ] [ Designated as safety issue: Yes ]

Enrollment:	54
Study Start Date:	April 2009
Study Completion Date:	November 2009
Primary Completion Date:	November 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Usual pancreatic enzymes The study subjects will remain on their usual pancreatic enzymes therapy during a portion of the study and switch to Ultrase MT12 during the second portion of the study.	Drug: Pancrelipase Pancrelipase capsules with each meal and snack. The lipase dose will be lower than 2500 UI lipase/kg of body weight per meal or snack.

Eligibility

Ages Eligible for Study:	2 Years to 6 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Male or female aged 2 to 6 years inclusively.
Diagnosis of CF based on one or more typical clinical features of CF OR a sibling with CF OR a positive newborn screening, plus and at least one of the two followings:
- Sweat chloride test ≥60mmol/L on two separate occasions.
- Two identifiable CF-causing mutations.
Presence of PI as demonstrated by FE-1 test < 100µg/g of stools and treating currently with pancreatic enzyme supplementation.
Able to eat a high-fat diet and having a current adequate nutritional status based on the BMI.

Exclusion Criteria:

Subject currently receiving or received an Ultrase MT product in the last 30 days.
Contraindication/hypersensitivity to Ultrase or to any porcine protein.
Presence of a medical condition known to increase fecal fat loss or that could compromise study results or the study subject safety.
History of complete DIOS in the past 6 months OR 2 or more episodes of incomplete DIOS in the past year.
Use of medications that can interfere with fat excretion or frequency of bowel movements.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00880100

Locations

United States, Colorado
The Children's Hospital
Aurora, Colorado, United States, 80045
United States, Michigan
University of Michigan Health System Cystic Fibrosis Center
Ann Arbor, Michigan, United States, 48109-0212
Helen DeVos Children's Hospital-Spectrum Health Research Department
Grand Rapids, Michigan, United States, 40503
United States, New York
SUNY Upstate Medical University
Syracuse, New York, United States, 13203
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Ohio
Rainbow Babies and Children's Hospital - Cystic Fibrosis Center
Cleveland, Ohio, United States, 44106
Children's Medical Center of Dayton
Dayton, Ohio, United States, 45404
United States, Oklahoma
Respiratory Diseases of Children and Adolescents
Oklahoma City, Oklahoma, United States, 73112
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States, 73104
United States, Pennsylvania
Pennsylvania State University and the Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15213
United States, South Dakota
Sanford Children's Specialty Clinic
Sioux Falls, South Dakota, United States, 57117-5039
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84108
United States, Virginia
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
United States, Wisconsin
UW Hospital and Clinics
Madison, Wisconsin, United States, 53792

Sponsors and Collaborators

Axcan Pharma

Investigators

Study Chair:

Michael W Konstan, MD

Rainbow Babies and Children's Hospital -Cystic Fibrosis Center, Cleveland, OH

More Information

No publications provided

Responsible Party:	Josée Grondin, Clinical Research Scientist, Axcan Pharma Inc.
ClinicalTrials.gov Identifier:	NCT00880100 History of Changes
Other Study ID Numbers:	UMT12CF08-01
Study First Received:	April 9, 2009
Last Updated:	May 12, 2010
Health Authority:	United States: Food and Drug Administration

Keywords provided by Axcan Pharma:

Steatorrhea
Malabsorption of fat
Pancreatic enzymes
Abdominal pain
Greasy stools

Additional relevant MeSH terms:

Cystic Fibrosis
Fibrosis
Exocrine Pancreatic Insufficiency
Steatorrhea
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases

Pathologic Processes
Malabsorption Syndromes
Intestinal Diseases
Gastrointestinal Diseases
Metabolic Diseases
Pancrelipase
Gastrointestinal Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on February 09, 2012