A Long-Term Prospective Observational Safety Study of the Incidence of and Risk Factors for Fibrosing Colonopathy

Confidential Clinical Trial A Long-Term Prospective Observational Safety Study of the Incidence of and Risk Factors for Fibrosing Colonopathy in US Patients with Cystic Fibrosis Treated with Pancreatic Enzyme Replacement Therapy: A Harmonized Protocol Across Sponsors The US Food and Drug Administration has mandated post-marketing requirements for the use of pancreatic enzyme replacement therapy (PERT) for the treatment of exocrine pancreatic insufficiency, including an up to 10-year, observational study to prospectively evaluate the incidence of fibrosing colonopathy in patients with cystic fibrosis (CF) and to assess potential risk factors for the event. This observational study will be conducted in US patients with a diagnosis of CF, treated in clinical practice and enrolled in Port CF, a patient registry which is supported by the US CFF. Primary Objectives: The primary objective of this study is to quantify over a 10-year period the incidence of fibrosing colonopathy in US patients with CF treated with pancreatic enzyme replacement therapy (PERT). Secondary Objectives: The secondary objective of this study is to quantify the association between potential risk factors and the development of fibrosing colonopathy in patients with CF.