Study of Pancreatic Enzyme Product in Pediatric Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency
Condition: Cystic Fibrosis; Exocrine Pancreatic Insufficiency
MeSH terms: Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Fibrosis
Intervention: EUR-1008 (APT-1008)
Intervention description: EUR-1008 (APT-1008) Microtabs contained in a capsule will be administered orally from Day 5 to Day 11 at an enzyme dose based on investigator's discretion, in dose stabilization period or the content of the capsule will be allowed to sprinkle on food, where necessary, followed by stabilized dose from Day 12 to Day 18 in treatment period, up to a maximum total dose of 10,000 lipase units per kilogram body weight per day (unit/kg/day).
Intervention MeSH terms: Pancrelipase
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 19.0
Sex: All
Maximum age: 7 Years
Healthy volunteers: No
Keywords: Cystic fibrosis; Exocrine Pancreatic Insufficiency; Zenpep-1009

Summary:

      This is an open-label study to evaluate the efficacy and safety of Aptalis' (formerly Eurand)
      pancreatic enzyme product (PEP) microtabs in pediatric participants under age 7 with cystic
      fibrosis (CF) and exocrine pancreatic insufficiency (EPI).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Participants less than 7 years of age

          -  Participants who have pancreatic insufficiency documented by a fecal elastase level
             less than 100 micrograms per gram (mcg/g), or if not documented, the fecal elastase
             test must be done at the screening visit

          -  Participants who have a need of de novo treatment with pancreatic enzymes or be able
             to be switched from an existing treatment

          -  Participants who have a body mass index greater than the twenty fifth percentile for
             children 2 years and older

          -  Participants with a weight for height index greater than the twenty fifth percentile
             for children less than 2 years of age

          -  Participants with diagnosis of CF based upon the following criteria:

               -  Have 2 clinical features consistent with CF and

               -  Have either a genotype with 2 identifiable mutations consistent with CF or a
                  sweat chloride concentration that is more than 60 milliequivalent per liter
                  (mEq/L) by quantitative pilocarpine iontophoresis

          -  Participants who are clinically stable with no evidence of acute upper or lower
             respiratory tract infection

        Exclusion Criteria:

          -  Participants with fibrosing colonopathy

          -  Participants allergic to pork or other porcine PEPs

          -  Participants with any respiratory condition that in the investigator's opinion would
             result in an intervention requiring hospitalization or intensive pulmonary treatment
             during the trial

          -  Participants with any acute systemic administration of an antibiotic for any reason in
             the previous 4 weeks; however, a low stable dose of an antibiotic (such as
             azithromycin 250 or 500 milligram [mg] up to 3 times per week) is allowed. Moreover,
             chronic treatment (that is, daily for at least 1 month) with an inhalatory antibiotic
             (for example, colistin, tobramycin, or ceftazidime) is allowed

          -  Participants who have hepatic insufficiency as defined by a history or presence of
             ascites, or a serum albumin level of less than 3.0 milligram per deciliter (mg/dL), or
             coagulopathy with an international normalized ratio that is greater than 1.7

          -  Participants with hyperuricemia or hyperuricosuria

          -  Participants participating in an investigational study of a drug, biologic, or device
             not currently approved for marketing within 30 days prior to screening visit

          -  Participants with history of or current screening evaluation of hyperglycemia as
             defined by an 8-hour fasting serum glucose equivalent to 126 mg/dL or more, or of
             cystic-fibrosis-related diabetes as determined according to the Cystic Fibrosis
             Foundation (CFF) Consensus Conference of January 1999 (Section IX Part II), that is:

               -  Fasting Blood Glucose (FBG) greater than126 mg/dl (7.0 milli mole [mM]) on two or
                  more occasions

               -  FBG greater than 126 mg/dl (7 .0 mM) plus casual (without regard to time of day
                  or last meal consumed) glucose level greater than200 mg/dl (11.1 mM)

               -  Casual (previously called random) glucose levels greater than 200 mg/dl (11.1 mM)
                  on two or more occasions with symptoms

          -  Participants with any solid organ transplant or surgery affecting the bowel

          -  Participants using an enzyme preparation in excess of 10,000 lipase units/kg/day

          -  Participants with an acute dose of any steroid in the previous 2 weeks; however, low
             chronic doses of a steroid (less 0.5 mg/kg every other day) will be allowed

          -  Participants with any condition that would, in the investigator's opinion, limit the
             patient's ability to complete the study

          -  Participants with history of or current screening determination of distal ileal
             obstruction syndrome (DIOS), or any clinical signs and symptoms suggestive of DIOS
             (that is, constipation, abdominal pain, anorexia, early satiety, recurrent vomiting
             and palpable fecal mass) on physical examination

          -  Participants who are unable to discontinue excluded concomitant medications over the
             course of the study