Back to resultsUse of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)
Primary Purpose
Cystic Fibrosis, Pancreatic Insufficiency
Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Ultrase® MT12
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis focused on measuring Steatorrhea, Malabsorption of fat, Pancreatic enzymes, Abdominal pain, Greasy stools
Eligibility 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
Sites / Locations
- The Children's Hospital
- University of Michigan Health System Cystic Fibrosis Center
- Helen DeVos Children's Hospital-Spectrum Health Research Department
- SUNY Upstate Medical University
- Duke University Medical Center
- Rainbow Babies and Children's Hospital - Cystic Fibrosis Center
- Children's Medical Center of Dayton
- University of Oklahoma Health Sciences Center
- Respiratory Diseases of Children and Adolescents
- Pennsylvania State University and the Milton S. Hershey Medical Center
- Children's Hospital of Pittsburgh of UPMC
- Sanford Children's Specialty Clinic
- University of Utah
- Virginia Commonwealth University
- UW Hospital and Clinics
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Ultrase® MT12
Arm Description
Outcomes
Primary Outcome Measures
Percentage of Patients With Control of Steatorrhea
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
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
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
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
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00880100
Brief Title
Use of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)
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
Study Type
Interventional
2. Study Status
Record Verification Date
February 2017
Overall Recruitment Status
Completed
Study Start Date
April 2009 (undefined)
Primary Completion Date
November 2009 (Actual)
Study Completion Date
November 2009 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Forest Laboratories
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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).
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis, Pancreatic Insufficiency
Keywords
Steatorrhea, Malabsorption of fat, Pancreatic enzymes, Abdominal pain, Greasy stools
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
49 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Ultrase® MT12
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Ultrase® MT12
Intervention Description
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.
Primary Outcome Measure Information:
Title
Percentage of Patients With Control of Steatorrhea
Description
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.
Time Frame
A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Secondary Outcome Measure Information:
Title
Percentage of Patients With Normal Stool Frequency
Description
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.
Time Frame
A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Title
Percentage of Stools With Normal Consistency
Description
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.
Time Frame
A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Title
Percentage of Stools With Abnormal Characteristics
Description
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.
Time Frame
A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Title
Mean Number of Days Without Abdominal Complaints
Description
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.
Time Frame
A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Other Pre-specified Outcome Measures:
Title
Total Weight of Stools
Description
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.
Time Frame
A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Title
Percentage of Days With Abdominal Pain and Excessive Flatulence
Description
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.
Time Frame
A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Aptalis Medical Information
Organizational Affiliation
Forest Laboratories
Official's Role
Study Director
Facility Information:
Facility Name
The Children's Hospital
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Michigan Health System Cystic Fibrosis Center
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109-0212
Country
United States
Facility Name
Helen DeVos Children's Hospital-Spectrum Health Research Department
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
40503
Country
United States
Facility Name
SUNY Upstate Medical University
City
Syracuse
State/Province
New York
ZIP/Postal Code
13203
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Rainbow Babies and Children's Hospital - Cystic Fibrosis Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Children's Medical Center of Dayton
City
Dayton
State/Province
Ohio
ZIP/Postal Code
45404
Country
United States
Facility Name
University of Oklahoma Health Sciences Center
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Respiratory Diseases of Children and Adolescents
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73112
Country
United States
Facility Name
Pennsylvania State University and the Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Children's Hospital of Pittsburgh of UPMC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Sanford Children's Specialty Clinic
City
Sioux Falls
State/Province
South Dakota
ZIP/Postal Code
57117-5039
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
Virginia Commonwealth University
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Facility Name
UW Hospital and Clinics
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Use of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)
We'll reach out to this number within 24 hrs