Safety and Efficacy Study of ULTRASE® MT20 in Participants With Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (PI)
Condition: Cystic Fibrosis; Exocrine Pancreatic Insufficiency
MeSH terms: Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Fibrosis
Intervention: Ultrase® MT20; Placebo
Intervention description: Ultrase® MT 20 capsules containing enteric-coated minitablets orally daily at a dose stabilized during the first stabilization period (4 days), as per investigator's discretion, for 6 to 7 days in either first intervention period or second intervention period.; Placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in either first intervention period or second intervention period.
Intervention MeSH terms: Pancrelipase; Pancreatin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 36.0
Sex: All
Minimum age: 7 Years
Healthy volunteers: No
Keywords: Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Ultrase® MT20

Summary:

      The purpose of this study is to assess the safety and efficacy of Ultrase® MT20 compared to
      placebo for the correction of fat and protein malabsorption in participants with cystic
      fibrosis (CF) and exocrine pancreatic insufficiency (EPI). This study is sponsored by Aptalis
      Pharma (formerly Axcan).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Participants or their legally authorized representative must understand the nature of
             the study and sign an informed consent or assent form along with a parental form

          -  Participants must have a confirmed diagnosis of CF based on 1 or more clinical
             features consistent with the CF phenotype, and one of the following:

               -  A genotype with 2 identifiable mutations consistent with CF

               -  A sweat chloride test greater than 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 must require
             pancreatic enzyme supplementation

          -  Participants must be clinically stable as evidenced by medical and medication history,
             baseline physical examination including vital signs and laboratory analyses

          -  Participants must be 7 years and older

          -  Participants must have an adequate nutritional status based on the following body mass
             index (BMI):

               -  Participants 7 to 20 years old must have a BMI greater than or equal to fifth
                  percentile

               -  Female participants greater than 20 years old must have a BMI greater than or
                  equal to 16

               -  Male participants greater 20 years old must have a BMI greater than or equal to
                  16.5

          -  Participants must be on an optimal clinical dose of pancreatic enzymes (Ultrase® MT18
             or MT20 or other pancreatic enzymes preparations including Ultrase® MT12) prior to
             entry in the study, and must tolerate this medication in the opinion of the
             investigator

          -  Participants must be able to swallow capsules and must be able to eat a high fat diet
             calculated as 2 gram (± 15%) fat per kilogram body weight per day

          -  Participants must be, in the opinion of the investigator, able and willing to complete
             this study

          -  Female participants must be premenarcheal, surgically sterile or postmenopausal for at
             least 12 consecutive months. Otherwise, the women of childbearing potential (WOCBP)
             must not be pregnant and must have practiced an acceptable method of contraception for
             at least one month prior to the study entry

        Exclusion Criteria:

          -  Participants with a known contraindication, sensitivity or hypersensitivity to Ultrase
             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 not willing to stop the prohibited medications or products at study entry
             and throughout the study

          -  Participants who are using narcotics

          -  Participants who are using 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 known to have 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, 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 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