Use of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)
Condition: Cystic Fibrosis; Pancreatic Insufficiency
MeSH terms: Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Fibrosis
Intervention: 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.
Intervention MeSH terms: Pancrelipase; Pancreatin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 49.0
Sex: All
Minimum age: 2 Years
Maximum age: 6 Years
Healthy volunteers: No
Keywords: Steatorrhea; Malabsorption of fat; Pancreatic enzymes; Abdominal pain; Greasy stools

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).
    

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