Efficacy and Safety of ULTRASE MT20 in Improving the Coefficient of Fat Absorption (CFA%) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI) - Full Text View

Sponsored by:	Axcan Pharma
Information provided by:	Axcan Pharma
ClinicalTrials.gov Identifier:	NCT00513682

Purpose

This protocol will enroll children aged 7 to 11 years old, suffering from Cystic Fibrosis and Pancreatic Insufficiency in order to demonstrate the safety of Ultrase MT20 as well as the efficacy of this product in the improvement of the fat absorption.

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

Genetics Home Reference related topics: cystic fibrosis

MedlinePlus related topics: Cystic Fibrosis

Drug Information available for: Pancrelipase Ultrase

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Crossover Assignment, Safety/Efficacy Study
Official Title:	Efficacy and Safety of ULTRASE MT20 in Improving the Coefficient of Fat Absorption (CFA%) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)

Further study details as provided by Axcan Pharma:

Primary Outcome Measures:

Coefficient of fat absorption (CFA%) [ Time Frame: The CFA% will be assessed with a 72 hour stool collection ]

Secondary Outcome Measures:

Coefficient of Nitrogen absorption (CNA%) and Safety of Ultrase MT20 [ Time Frame: The CNA% will be assessed with a 72 hour stool collection and the safety will be assessed with laboratory testing, recording of all AEs throughout the study and recording of all SAEs until 30 days after the last visit. ]

Enrollment:	9
Study Start Date:	July 2007
Study Completion Date:	April 2008
Primary Completion Date:	March 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Treatment phase: Experimental During the treatment phase, the patient will eat a high fat diet and will take ULTRASE MT20 at a specific daily dose to treat his pancreatic insufficiency.	Drug: ULTRASE MT20 A stabilized dose of ULTRASE MT20 in capsules will be dispensed with each meal and snack during all days of the treatment phase.

Detailed Description:

This study will include 3 phases: the screening phase, the washout phase and the treatment phase.

Screening Phase: this phase will last fifteen (15) days and all patients will take ULTRASE MT20 during this period. During the last 4 days, patents will be stabilized on a high fat diet and with Ultrase MT20.

Washout Phase: this phase will last 6 to 7 days. The patient will continue the high-fat diet but WILL REFRAIN from taking ULTRASE MT20 or any other enzymes. A 72-hour stool collection will be performed and all food consumed by the patient will be recorded to assess the Coefficient of Fat Absorption (CFA%) and the Coefficient of Nitrogen Absorption (CNA%).

Treatment Phase: this phase will last 7 to 11 days. The patient will continue the high-fat diet and will take the 'Stabilized Dose' of ULTRASE MT20 established during screening. Another 72-hour stool collection will be performed and all food consumed by the patient will be recorded to assess the Coefficient of Fat Absorption (CFA%) and the Coefficient of Nitrogen Absorption CNA%).

Eligibility

Ages Eligible for Study:	7 Years to 11 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Signature of an informed consent or assent form along with a parental form.
Patients must have a confirmed diagnosis of Cystic Fibrosis.
Patients must have Pancreatic Insufficiency and must require pancreatic enzyme supplementation.
Patients must be 7 to 11 years of age.
Patients must have an adequate nutritional status.
Patients must be on an optimal clinical dose of pancreatic enzymes prior to entry in the study, and must tolerate this medication in the opinion of the investigator.
Patients must be able to eat a high-fat diet.
Female patients should be premenarcheal. Otherwise, a female patient of childbearing potential (WOCBP) must not be pregnant and must have practiced an acceptable method of contraception for at least one month prior to the study entry.

Exclusion Criteria:

Patients with a known contraindication, sensitivity or hypersensitivity to ULTRASE or any porcine protein.
Patients with a known allergy to the FD&C Blue No. 2 dye indicator (stool marker).
Patients who use narcotics chronically and bowel stimulants and/or laxatives on a regular basis.
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 receiving enteral tube feeding and not willing to stop during the course of the study.
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 in the past six (6) months; or, patients who had two (2) or more episodes of DIOS in the past year.
Patients with poorly controlled diabetes according to the investigator's opinion
Female patients who are pregnant or lactating.
Patients who received an Investigational drug within 30 days prior to entry into the study.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00513682

Locations

United States, Michigan
University of Michigan Health System Cystic Fibrosis Center
Ann Arbor, Michigan, United States, 48109-0212
United States, Ohio
Cystic Fibrosis Center Rainbow Babies and Children's HospitalDivision
Cleveland, Ohio, United States, 44106
United States, Pennsylvania
Pennsylvania State University And the Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033

Sponsors and Collaborators

Axcan Pharma

Investigators

Principal Investigator:	Michael Konstan, MD	Rainbow Baby- University hospital of Cleveland
Study Director:	Jean Spénard, Ph.D	Axcan Pharma Inc.

More Information

Study ID Numbers:	UMT20CF07-01
Study First Received:	August 7, 2007
Last Updated:	April 9, 2008
ClinicalTrials.gov Identifier:	NCT00513682
Health Authority:	United States: Food and Drug Administration

Keywords provided by Axcan Pharma:

Cystic Fibrosis
Pancreatic insufficiency
Steatorrhea
Children 7 to 11 years
pancreatic enzyme

Study placed in the following topic categories:

Digestive System Diseases
Genetic Diseases, Inborn
Respiratory Tract Diseases
Cystic Fibrosis
Fibrosis
Lung Diseases

Infant, Newborn, Diseases
Pancreatic Diseases
Pancrelipase
Cystic fibrosis
Steatorrhea
Exocrine Pancreatic Insufficiency

Additional relevant MeSH terms:

Pathologic Processes
Therapeutic Uses
Gastrointestinal Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 15, 2009