Open Label Safety Study of DCI-1020 in Pediatric Cystic Fibrosis (CF) Patients

Hypothesis:DCI 1020 capsules are safe and effective in treating exocrine pancreatic insufficiency in CF patients <= 2 years of age.

The results of this study are intended to be submitted to the FDA as part of the NDA package for marketing approval of PANCRECARB (DCI 1020).

A large body of data supporting the safety and efficacy of PANCRECARB® (pancrelipase) Capsules is available in patients above age 2. This study is being performed to gather the data to demonstrate the safety and efficacy of DCI1020 in pediatric CF patients (≤ 2 years) with exocrine pancreatic insufficiency. This study also takes into consideration an "age appropriate" dosing form. Specifically, the enzymes will be administered orally by opening the capsules and emptying the microspheres into a small amount of applesauce as an example of a slightly acidic soft food such as jelly, jello, etc. for feeding.

The results of this study are intended to be submitted to the FDA as part of the NDA package for marketing approval of PANCRECARB® DCI 1020(pancrelipase).

• Digestive System Diseases
• Pancreatic Disease
• Cystic Fibrosis
• Exocrine Pancreatic Insufficiency

Inclusion Criteria:

• Male or female age ≤ 2 years of age
• Confirmed diagnosis of CF based on the following criteria:
• One or more clinical features consistent with the CF phenotype, AND
• Positive sweat chloride ≥ 60 mEq/liter (by pilocarpine iontophoresis), OR
• Genotype with two identifiable mutations consistent with CF
• Adequate nutritional status
• Pancreatic insufficiency documented by spot fecal elastase-1 (FE 1) greater or equal to 100 micrograms/g stool
• Clinically stable with no evidence of an acute medical condition
• Parent/Guardian able to understand and sign a written informed consent and comply with the requirements of the study

Exclusion Criteria:

• History of fibrosing colonopathy
• History of being refractory to pancreatic enzyme replacement therapy
• Solid organ transplant
• History of intra-abdominal surgery
• A current diagnosis or a history of distal intestinal obstruction syndrome (DIOS) in the past six (6) months, or 2 or more episodes of DIOS in the past twelve (12) months
• Conditions known to increase fecal fat loss including: inflammatory bowel disease , celiac disease, Crohn's disease, tropical Sprue, Whipple's disease
• A known contraindication, sensitivity or hypersensitivity to porcine pancreatic enzymes
• Active liver disease with liver enzymes (alanine aminotransferase (ALT/SGPT), aspartate aminotransferase (AST/SGOT) or bilirubin ≥ 3 times the upper limit of normal
• Acute pancreatitis or acute exacerbation of chronic pancreatitis
• Antibiotic use as follows:
• Acute treatment with any systemic (oral or IV) antibiotics two (2) weeks prior to screening
• Treatment with erythromycin and unwilling to discontinue the treatment two (2) weeks prior to the screening. (azithromycin is allowed)
• Change in chronic treatment with systemic (oral and IV) antibiotics during the trial

NOTE:

Study patient may remain on a chronic regimen of systemic (oral or IV) antibiotics (with exception of erythromycin), if he/she started the antibiotics at least 2 weeks prior to study screening, was at his/her usual bowel pattern at the time of screening, and does not stop or change these antibiotics during the study.

• Receiving enteral tube feeding during the study
• Breast feeding during the study (expressed breast milk may be used, but not feeding at the breast)
• Expected inability to cooperate with or be non-adherent to required study procedures
• Use of narcotics
• Poorly controlled diabetes
• Participation in an investigational study of a drug, biologic, or device not currently approved for marketing, within 30 days of screening visit
• A medical condition which the investigator deems significant enough to interfere with the ability of the study patient to participate in the trial or interfering with assessment of effects of enzyme therapy on fat absorption