A Safety and Efficacy Study on ULTRASE MT20 in Patients With Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (PI)

Cystic Fibrosis (CF) is a genetic disease resulting from the inheritance of a defective autosomal recessive gene. It often results in exocrine pancreatic insufficiency (PI) that leads to non-digestion of fats and proteins and finally to malabsorption of these nutrients. The patients need to take pancreatic enzymes to allow absorption of these nutriments and this has contributed to a significant increase in the life span of CF patients.

The purpose of this study is to assess the safety and efficacy of ULTRASE MT20 compared to placebo for the correction of fat and protein malabsorption in patients with CF and PI.

• Cystic Fibrosis
• Exocrine Pancreatic Insufficiency

Key Inclusion Criteria:

• Confirmed diagnosis of Cystic Fibrosis
• Patients must have pancreatic insufficiency and must require pancreatic enzyme supplementation
• Patients must be 7 years and older
• Patients must be on an optimal clinical dose of pancreatic enzymes and must be clinically stable
• Patients must have an adequate nutritional status

Key Exclusion Criteria:

• Patients with acute pancreatitis or acute exacerbation of chronic pancreatic disease
• Patients with an acute pulmonary infection
• Patients with a history of bowel resection
• Patients suffering from any dysmotility disorders
• Patients with chronic or severe abdominal pain
• Patients known to have a significant medical disease that would compromise their welfare or confound the study results
• Patients with a history of or a current diagnosis of clinically significant portal hypertension
• Patients who have a condition known to increase fecal fat loss
• Patients with a current diagnosis or a history of complete DIOS
• Patients with poorly controlled diabetes
• Female patients who are pregnant or lactating