Study of Pancreatic Enzyme Product in Pediatric Patients With Cystic Fibrosis and Exocrine Pancreatic Insufficiency

Inclusion Criteria:

1. Are less than 7 years of age
2. Have pancreatic insufficiency documented by a fecal elastase level less than 100 microg/g, or if not documented, the fecal elastase test must be done at the Screening visit
3. Have a need of de novo treatment with pancreatic enzymes or be able to be switched from an existing treatment
4. Have a body mass index greater than the 25th percentile for children 2 years and older
5. Have a weight for height index greater than the 25th percentile for children less than 2 years of age52

6. Have a diagnosis of CF based upon the following criteria:

   • Have 2 clinical features consistent with CF
   • Have either a genotype with 2 identifiable mutations consistent with CF or a sweat chloride concentration that is more than 60 mEq/L by quantitative pilocarpine iontophoresis
7. Are clinically stable with no evidence of acute upper or lower respiratory tract infection

Exclusion Criteria:

1. Fibrosing colonopathy
2. Allergy to pork or other porcine PEPs
3. Any respiratory condition that in the investigator's opinion would result in an intervention requiring hospitalization or intensive pulmonary treatment during the trial
4. 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 mg up to 3 times per week) is allowed. Moreover, chronic treatment (i.e., daily for at least 1 month) with an inhalatory antibiotic (e.g., colistin, tobramycin, or ceftazidime) is allowed.
5. Hepatic insufficiency as defined by a history or presence of ascites, or a serum albumin level of less than 3.0 mg/dL, or coagulopathy with an international normalized ratio that is greater than 1.7.
6. Hyperuricemia or hyperuricosuria
7. Participating in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to Screening visit.
8. A history of or current screening evaluation of hyperglycemia
9. Any solid organ transplant or surgery affecting the bowel. Patients with a history of appendectomy and inguinal (non incarcerated) hernioplasty or meconium ileus without the need for bowel resection, may be enrolled. GI tube fed patients, in absence of dumping syndrome, are also eligible.
10. Using an enzyme preparation in excess of 10,000 lipase units/kg/day
11. An acute dose of any steroid in the previous 2 weeks; however, low chronic doses of a steroid (< 0.5 mg/kg every other day) will be allowed
12. Any condition that would, in the investigator's opinion, limit the patient's ability to complete the study
13. A history of or current screening determination of distal ileal obstruction syndrome (DIOS), or any clinical signs and symptoms suggestive of DIOS (i.e., constipation, abdominal pain, anorexia, early satiety, recurrent vomiting, and palpable fecal mass) on physical examination
14. Inability to discontinue excluded concomitant medications over the course of the study