Efficacy and Safety of Ultrase® MT20 in Improving the Coefficient of Fat Absorption (CFA) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)
Condition: Cystic Fibrosis; Pancreatic Insufficiency
MeSH terms: Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Fibrosis
Intervention: Ultrase® MT20
Intervention description: Ultrase® MT20 capsules will be administered orally with each meal during Day 1 to 15 in screening phase at a dose based on investigator's discretion. During Day 12 to 15, participants will receive high-fat diet and Ultrase® MT20 dose will be adjusted depending on symptoms of steatorrhea. This will be followed by a washout phase of 6 to 7 days, in which participants will receive only high-fat diet; then stabilized dose of Ultrase® MT20 capsule (as identified during screening phase) will be administered orally for 7 to 11 days during treatment phase. The stabilized dose should not to exceed 2500 lipase units per kilogram body weight per meal (lipase units/kg/meal).
Intervention MeSH terms: Pancrelipase; Pancreatin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 9.0
Sex: All
Minimum age: 7 Years
Maximum age: 11 Years
Healthy volunteers: No
Keywords: Cystic Fibrosis; Pancreatic insufficiency; Steatorrhea; Children 7 to 11 years; Pancreatic enzyme

Summary:

      This is a Phase III, multicenter, open-label study, that will evaluate the improvement of
      nutrient absorption when participants will receive Ultrase® MT20. This study is sponsored by
      Aptalis (formerly Axcan). This study is performed in children from 7 to 11 years old.
    

Eligibility criteria:

        Inclusion Criteria:

          -  After a full explanation of the study, participants or their legally authorized
             representative must have signed the informed consent or assent form along with a
             parental form

          -  Participants must have a confirmed diagnosis of CF based on one or more clinical
             features consistent with the CF phenotype, and one of the following:

               -  A genotype with two identifiable mutations consistent with CF

               -  A sweat chloride test greater that 60 millimole per liter (mmol/L) by
                  quantitative pilocarpine iontophoresis

          -  Participants must have PI as demonstrated by a fecal elastase-1 (FE-1) concentration
             less than 100 microgram per gram (mcg/g) of stools (ScheBo test) and requiring
             pancreatic enzyme supplementation

          -  Participants must be clinically stable according to the physician's judgment by:

               -  Medical and medication history

               -  Baseline physical examination including vital signs and laboratory analyses

          -  Participants must be 7 to 11 years of age

          -  Participants must have an adequate nutritional status based on body mass index (BMI)
             greater than or equal to fifth percentile

          -  Participants must be on an optimal clinical dose of pancreatic enzymes (Ultrase® MT12
             , MT18 or MT20 or other pancreatic enzyme preparations) prior to entry in the study
             and must tolerate this medication in the opinion of the investigator

          -  Participants must be able to be able to swallow capsules and eat a high-fat diet
             calculated as 2 gram fat per kilogram (g fat/kg) of body weight per day

          -  Participants must be, in the opinion of the investigator, able and willing to complete
             the study

          -  Female participants should be premenarcheal. Otherwise, a female participant of
             childbearing potential (WOCBP) must not be pregnant and must have practiced an
             acceptable method of contraception for at least 1 month prior to the study entry

        Exclusion Criteria:

          -  Participants with a known contraindication, sensitivity or hypersensitivity to
             Ultrase® MT20 or any porcine protein

          -  Participants with a known allergy to the food, drug and cosmetic (FD&C) Blue No. 2 dye
             indicator (stool marker)

          -  Participants who are not willing to stop the prohibited medications or products at
             study entry and throughout the study

          -  Participants who use narcotics chronically and bowel stimulants and/or laxatives on a
             regular basis

          -  Participants with acute pancreatitis or acute exacerbation of chronic pancreatic
             disease

          -  Participants with an acute pulmonary infection

          -  Participants with a history of bowel resection

          -  Participants suffering from any dysmotility disorders

          -  Participants with chronic or severe abdominal pain

          -  Participants receiving enteral tube feeding and not willing to stop during the course
             of the study

          -  Participants with a significant medical disease that would compromise their welfare or
             confound the study results

          -  Participants with a history of or a current diagnosis of clinically significant portal
             hypertension

          -  Participants who have a condition known to increase fecal fat loss including celiac's
             disease, biliary cancer, biliary stricture, cholelithiasis, Crohn's disease,
             ulcerative colitis, pancreas cancer, radiation enteritis, tropical sprue, Whipple's
             disease, lactose intolerance, pseudomembranous colitis

          -  Participants with a current diagnosis or a history of complete distal intestinal
             obstruction syndrome (DIOS) in the past 6 months; or, participants who had 2 or more
             episodes of DIOS in the past year

          -  Participants with poorly controlled diabetes according to the investigator's opinion

          -  Female participants who are pregnant or lactating

          -  Participants who received an investigational drug within 30 days prior to entry into
             the study