Clinical trial NCT00880100: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Pancreatic Insufficiency
Keywords: Steatorrhea
Malabsorption of fat
Pancreatic enzymes
Abdominal pain
Greasy stools
Lead sponsor: Forest Laboratories
Sponsor class: INDUSTRY
First posted: 2009-04-13
Enrollment: 49
Sex: ALL
Minimum age (years): 2
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Percentage of Patients With Control of Steatorrhea
timeFrame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Secondary outcomes:
measure: Percentage of Patients With Normal Stool Frequency
timeFrame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
measure: Percentage of Stools With Normal Consistency
timeFrame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
measure: Percentage of Stools With Abnormal Characteristics
timeFrame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
measure: Mean Number of Days Without Abdominal Complaints
timeFrame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Other outcomes:
measure: Total Weight of Stools
timeFrame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
measure: Percentage of Days With Abdominal Pain and Excessive Flatulence
timeFrame: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Locations:
city: Aurora
country: United States
facility: The Children's Hospital
state: Colorado
city: Ann Arbor
country: United States
facility: University of Michigan Health System Cystic Fibrosis Center
state: Michigan
city: Grand Rapids
country: United States
facility: Helen DeVos Children's Hospital-Spectrum Health Research Department
state: Michigan
city: Syracuse
country: United States
facility: SUNY Upstate Medical University
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: Rainbow Babies and Children's Hospital - Cystic Fibrosis Center
state: Ohio
city: Dayton
country: United States
facility: Children's Medical Center of Dayton
state: Ohio
city: Oklahoma City
country: United States
facility: University of Oklahoma Health Sciences Center
state: Oklahoma
city: Oklahoma City
country: United States
facility: Respiratory Diseases of Children and Adolescents
state: Oklahoma
city: Hershey
country: United States
facility: Pennsylvania State University and the Milton S. Hershey Medical Center
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
city: Sioux Falls
country: United States
facility: Sanford Children's Specialty Clinic
state: South Dakota
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Madison
country: United States
facility: UW Hospital and Clinics
state: Wisconsin
Study officials:
affiliation: Forest Laboratories
name: Aptalis Medical Information
role: STUDY_DIRECTOR
References: