Efficacy and Safety of ULTRASE MT20 in Improving the Coefficient of Fat Absorption (CFA%) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)
This study has been completed.
Sponsor:
Axcan Pharma
Information provided by:
Axcan Pharma
ClinicalTrials.gov Identifier:
NCT00513682
First received: August 7, 2007
Last updated: February 1, 2010
Last verified: February 2010
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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 3 |
| 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 MT20 in Improving the Coefficient of Fat Absorption (CFA%) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI) |
Resource links provided by NLM:
Genetics Home Reference related topics:
cystic fibrosis
MedlinePlus related topics:
Cystic Fibrosis
U.S. FDA Resources
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 |
|---|---|
|
Experimental: Treatment phase
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
No publications provided
| ClinicalTrials.gov Identifier: | NCT00513682 History of Changes |
| Other Study ID Numbers: | UMT20CF07-01 |
| Study First Received: | August 7, 2007 |
| Last Updated: | February 1, 2010 |
| 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 |
Additional relevant MeSH terms:
|
Cystic Fibrosis Fibrosis Exocrine Pancreatic Insufficiency Pancreatic Diseases Digestive System Diseases Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn |
Infant, Newborn, Diseases Pathologic Processes Pancreatin Pancrelipase Gastrointestinal Agents Therapeutic Uses Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 22, 2013