A Study to Evaluate the Mode of Administration and Safety of EUR-1008 (APT-1008) in Infants 1 to 12 Months of Age
Condition: Cystic Fibrosis; Exocrine Pancreatic Insufficiency
MeSH terms: Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Fibrosis
Intervention: EUR-1008 (APT-1008); EUR-1008 (APT-1008)
Intervention description: EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) from open capsule, mixed with a small amount of apple juice, will be given orally daily using a syringe nurser at dose increments of 3,000 lipase units, for 10 days in either first treatment period or second treatment period. Total dose will not exceed 10,000 lipase units per kilogram (kg) of body weight per day.; EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) from open capsule, mixed with a small amount of apple sauce, will be given orally daily using a spoon at dose increments of 3,000 lipase units, for 10 days in either first treatment period or second treatment period. Total dose will not exceed 10,000 lipase units per kg of body weight per day.
Intervention MeSH terms: Pancrelipase; Pancreatin
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: None (Open Label)
Enrollment: 15.0
Sex: All
Minimum age: 1 Month
Maximum age: 12 Months
Healthy volunteers: No
Keywords: Exocrine Pancreatic Insufficiency; Infants with CF

Summary:

      A study to determine the safety, effectiveness, and acceptability of 2 methods of
      administration of EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) 3,000
      lipase units capsule, a pancreatic enzyme product (PEP), in infants with exocrine pancreatic
      insufficiency (EPI) due to cystic fibrosis (CF). This study is sponsored by Aptalis Pharma
      (formerly Eurand).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Participants with diagnosis of CF based on the following criteria: one clinical
             feature consistent with CF, and either a genotype with 2 identifiable mutations known
             to cause CF or a sweat chloride concentration that is greater than 60 milliequivalent
             per liter by quantitative pilocarpine iontophoresis

          -  Participants who have the need for a PEP defined as monoclonal fecal elastase less
             than 200 microgram per gram (mcg/g) stool

          -  Caregiver must be willing to switch participant from their previous PEP (if any) to
             Zenpep®

          -  Participants who have a height to weight ratio target at greater than tenth percentile

          -  Participants who are clinically stable with no evidence of concomitant illness or
             acute upper or lower respiratory tract infection during the 7-day interval prior to
             screening and preceding accession into this clinical study

        Exclusion Criteria:

          -  Participants who are less than 1 month old or are greater than 12 months old

          -  Participants with history of meconium ileus or small bowel atresia in the newborn
             period that required surgery

          -  Participants who are allergic to pork or other porcine PEPs

          -  Participants with any respiratory condition or other serious comorbidity (for example
             patent ductus arteriosus [PDA], or necrotizing enterocolitis [NEC]) that in the
             investigator's opinion would result in an intervention requiring hospitalization or
             intensive pulmonary or other treatment during the trial

          -  Participants with other comorbidities independent of CF that, in the investigator's
             opinion, would result in an inability to participate in the study or excess risk to
             the participant that is above the standard of care

          -  Participants with acute respiratory infection in the previous 14 days requiring
             antibiotics

          -  Participants who required change in antacid dose in the 7 days before screening

          -  Participants with administration of oral, intramuscular (IM), intravenous (IV)
             glucocorticoids in the 4 weeks prior to screening

          -  Participants with any condition that would, in the investigator's opinion, limit the
             participant's ability to complete the study

          -  Participants currently participating in or has participated in an investigational
             study, with the exception of observational studies, within 30 days of the screening
             visit