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
- Children’s Medical Center (Dallas, Plano, Southlake)
- Clements University Hospital
- UT Southwestern Ambulatory Services
Prospective, observational, population-based cohort study conducted in patients with diagnosis of CF enrolled in Port
CF and treated at sites with iRB approval for the study. Routinely collected data (i.e., standard Port CF data collection
form) will be used to determine exposure to specific PeRT, and potential risk factors for the study outcome. Patients
with suspected fibrosing colonopathy during the study period will be approached by their clinical care team to obtain
informed consent for collection of additional data (i.e., outside the standard Port CF data collection form) to augment
surveillance and confirmation of fibrosing colonopathy diagnosis via an independent adjudication Panel.
The inclusion criteria for enrollment in the Base Study Population are:
1. Enrolled in Port CF (eIRB study number STU_102010-1214) .
2. Diagnosis of CF.
3. Receiving medical care from a CFF-accredited care center providing data to Port CF with
IRB approval for this study