SIMPLICITY: Studying Impacts on Malabsorption With Liprotamase in Cystic Fibrosis

A Phase 3, Open-Label Study Evaluating the Efficacy and Safety of Liprotamase in Subjects With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency

Liprotamase consists of 3 soluble, non-porcine digestive enzymes, lipase, protease, and amylase, combined in a fixed ratio. Liprotamase is stable in the stomach and can be formulated without enteric coating for administration either as a capsule or as a dosing solution dissolved in water or juice. The purpose of the present study is to provide efficacy and safety data for a new, soluble formulation of liprotamase, Liprotamase Powder for Oral Solution, in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).

Inclusion Criteria:

• For Part A: males or females ≥7 years of age
• For Part B: males or females 28 days to <7 years
• Diagnosis of cystic fibrosis based on presentation, genotype and/or sweat chloride
• Low fecal elastase
• Fair-to-good nutritional status

Exclusion Criteria:

• History or diagnosis of fibrosing colonopathy
• Distal intestinal obstruction syndrome in 6 months prior to screening
• Receiving enteral tube feedings
• Chronic diarrheal illness unrelated to pancreatic insufficiency
• Liver abnormalities, or liver or lung transplant, or significant bowel resection