Use of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Aptalis Pharma
ClinicalTrials.gov Identifier:
NCT00880100
First received: April 9, 2009
Last updated: February 17, 2015
Last verified: February 2015
  Purpose

Multicenter, explorative, phase IIIb, open-label study to assess the efficacy and safety of Ultrase® MT12, in the control of steatorrhea and clinical signs and symptoms of malabsorption in CF children with pancreatic insufficiency (PI). This study is sponsored by Aptalis Pharma (formerly Axcan).


Condition Intervention Phase
Cystic Fibrosis
Pancreatic Insufficiency
Drug: Ultrase® MT12
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group 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:


Further study details as provided by Aptalis Pharma:

Primary Outcome Measures:
  • Percentage of Patients With Control of Steatorrhea [ Time Frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) ] [ Designated as safety issue: No ]
    Control of steatorrhea was defined as a less than 30 percent (%) of fat in stools as measured by nuclear magnetic resonance (NMR) spectroscopy in all stool samples which are collected at baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and during the 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12.


Secondary Outcome Measures:
  • Percentage of Patients With Normal Stool Frequency [ Time Frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) ] [ Designated as safety issue: No ]
    Normal stool frequency was defined as having less than 4 bowel movements per day in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the Treatment Phase during which the PEP was replaced with Ultrase MT12.

  • Percentage of Stools With Normal Consistency [ Time Frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) ] [ Designated as safety issue: No ]
    Normal consistency of stool was defined as hard and formed or soft and formed consistency. Abnormal consistency was defined as loose and unformed stool or liquid stools and diarrhea. Percentage of stools with normal consistency of each patient was calculated from normal consistency of stools by the patient per day. Mean percentage of stools with normal consistency in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized.

  • Percentage of Stools With Abnormal Characteristics [ Time Frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) ] [ Designated as safety issue: No ]
    Stools of abnormal characteristics were defined as bulky/large, foul-smelling and/or oily stools. Mean percentage of stools with abnormal characteristics in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized.

  • Mean Number of Days Without Abdominal Complaints [ Time Frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) ] [ Designated as safety issue: No ]
    Abdominal complaints were defined as the reporting of abdominal pain and/or unusual and excessive flatulence/gas production. Mean number of days without abdominal complaints in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized.


Other Outcome Measures:
  • Total Weight of Stools [ Time Frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) ] [ Designated as safety issue: No ]
    The total weight of stools in grams (g) is the total weight obtained during the stool collection period regardless of the number of stools that had been collected during this same collection period. Mean total weight of stools in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized.

  • Percentage of Days With Abdominal Pain and Excessive Flatulence [ Time Frame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) ] [ Designated as safety issue: No ]
    Mean percentage of days with abdominal complaints during baseline phase (BP) and the 5-day collection period of the treatment phase for total patients was summarized. Abdominal complaints were defined as the reporting of abdominal pain and/or unusual and excessive flatulence/gas production. Mean number of days abdominal pain (AP) and excessive flatulence (EF) in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized.


Enrollment: 49
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: Ultrase® MT12 Drug: Ultrase® MT12
Ultrase® MT12 capsules will be given orally daily based on investigator's discretion to a maximum dose of 2,500 lipase units per kilogram (kg) body weight per meal or snack for 19 to 24 days during the treatment phase. Total maximum dose not to exceed 10,000 lipase units/kg/day.

Detailed Description:

This is a multicenter, explorative, phase IIIb, open-label study in patients with CF and PI. The study consists of a screening visit (visit 1), followed by a baseline phase of 9 days (plus a 5-day window if necessary) during which the regular pancreatic enzyme will be maintained and 10 stool samples will be collected over 5 days, for baseline evaluation of steatorrhea. Afterward, a treatment phase of 19 days (plus a 5-day window if necessary) with Ultrase® MT12 will follow (the usual pancreatic enzyme will be replaced by Ultrase® MT12). Over the last 5 days of the treatment phase, 10 additional stool samples will be collected, for evaluation of steatorrhea.

  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 patients aged 2 to 6 years inclusively
  • Patients with current diagnosis of CF based on one or more typical clinical features of CF or a sibling with CF or a positive newborn screening and at least either with sweat chloride test greater than or equal to 60 millimoles/liter (mmol/L) by quantitative pilocarpine iontophoresis on two separate occasions or two identifiable CF-causing mutations
  • Patients with presence of PI as demonstrated by fecal elastase (FE-1) less than 100 microgram/gram (mcg/g) of stools (performed by ScheBo test) and requiring pancreatic enzyme supplementation
  • Patients who are able to eat a high-fat diet calculated at a value between 2g to 4g fat/kg of body weight per day during the whole study and having a current adequate nutritional status based on the body mass index (BMI) greater than or equal to fifth percentile
  • Patients receiving current treatment of PI with pancreatic enzymes
  • The parent or legal guardian signed informed consent form (ICF) and is mentally able to understand and comply with the study procedures

Exclusion Criteria:

  • Patients currently receiving or received an Ultrase® MT product (MT12, MT18, MT20)for PI in the last 30 days
  • Patients having known contraindication, sensitivity or hypersensitivity to Ultrase® or to any porcine protein
  • Patients with presence of a medical condition known to increase fecal fat loss or that could compromise study results or the study patient safety
  • Patients with current diagnosis or history of complete distal intestinal obstruction syndrome (DIOS) in the past 6 months or who had 2 or more episodes of incomplete DIOS in the past year
  • Patients with use of any prohibited medication or product at study entry and during the course of the study
  • Patients with chronic use of narcotics
  • Patients with use of bowel stimulants and/or laxatives more than once a week
  • Patients with presence of acute pancreatitis or exacerbation of chronic pancreatic disease
  • Patients with presence of an acute infection that needed to be treated with oral or intravenous (IV) broad-spectrum antibiotics
  • Patients having history of significant bowel resection; small bowel resection for meconium ileus at birth and appendectomy were accepted. Patients with Presence of dysmotility disorders
  • Patients with presence of chronic or severe abdominal pain
  • Patients unable to comply with diet requirement
  • Patients receiving enteral tube feeding overnight at study entry or who will need to receive enteral tube feeding overnight during the course of the study
  • Patients with history of or a current diagnosis of clinically significant portal hypertension
  • Patients with presence of poorly controlled diabetes according to the Investigator's clinical judgment
  • Patients having any condition or pre-study laboratory abnormality or history of any illness which, in the opinion of the Investigator, might have put the patient at risk, prevented the patient from completing the study, or otherwise affect the outcome of the study
  • Patient with use of any investigational drug within 30 days prior to the date of signature of the ICF
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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
Aptalis Pharma
Investigators
Study Director: Aptalis Medical Information Aptalis Pharma
  More Information

No publications provided

Responsible Party: Aptalis Pharma
ClinicalTrials.gov Identifier: NCT00880100     History of Changes
Other Study ID Numbers: UMT12CF08-01
Study First Received: April 9, 2009
Results First Received: March 5, 2014
Last Updated: February 17, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Aptalis Pharma:
Steatorrhea
Malabsorption of fat
Pancreatic enzymes
Abdominal pain
Greasy stools

Additional relevant MeSH terms:
Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Fibrosis
Digestive System Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lung Diseases
Pancreatic Diseases
Pathologic Processes
Respiratory Tract Diseases
Pancreatin
Pancrelipase
Gastrointestinal Agents
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on April 23, 2015