Long-term Study in US Cystic Fibrosis Patients Receiving Digestive Enzyme Supplements to Assess Narrowing of the Large Intestine Causing Adverse Intestinal Symptoms (Fibrosing Colonopathy)

Study Overview
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Brief Summary
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This is a long-term study in cystic fibrosis patients who are participating in the Cystic Fibrosis Patient Registry to assess the occurrence and risk factors for a rare bowel disorder called fibrosing colonopathy (narrowing of the large intestine). Patients will be followed at their regular clinical care visits over a 10-year period and approached if they develop symptoms of fibrosing colonopathy for collection and use of further detailed information.

Detailed Description
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This is a prospective, observational, population-based cohort study in US cystic fibrosis patients participating in the Cystic Fibrosis Patient Registry in order to assess the incidence of and risk factors for fibrosing colonopathy. Cystic fibrosis (CF) patients participating in the registry from participating sites, as well as new CF patients enrolled in the registry at these sites over a 2-year period, will serve as the base study population (estimated to include 24,500-25,000 cystic fibrosis patients between the first patient encounter documented in the registry from any participating site and the 31st of July 2014). Cystic fibrosis patients in the base study population will be followed at their regular clinical care visits and, any patients presenting over a 10-year period with signs and symptoms of suspected fibrosing colonopathy, based on a prospective definition, will be approached to obtain a study-specific informed consent for collection of additional data outside the standard registry data collection form in order to augment surveillance. Data routinely collected via the standard CF registry will be used to determine exposure to any specific pancreatic enzyme replacement therapy and to assess potential risk factors for the outcome of confirmed fibrosing colonopathy. An independent adjudication panel will be utilized to validate the diagnosis of fibrosing colonopathy based on a prospective case definition as well as decision rules.

Official Title
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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 CF-FC Study)

Conditions
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Fibrosing Colonopathy

Intervention / Treatment
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* Biological: ULTRESA
* Biological: PANCREAZE
* Biological: CREON
* Biological: ZENPEP
* Biological: other non-sponsor pancreatic enzyme replacement therapy
* Biological: PERTZYE
* Other: No pancreatic enzyme replacement therapy


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The inclusion criteria for enrollment in the Base Study Population Diagnosed with cystic fibrosis, Enrolled in the Cystic Fibrosis Patient Registry Receiving medical care at a Cystic Fibrosis Foundation-accredited care center providing data to the Cystic Fibrosis Patient Registry Exclusion Criteria: None

Ages Eligible for Study
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Minimum Age: 0 Months
Maximum Age: 99 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Cystic fibrosis (CF) patients in the CF Patient Registry<br>Patients diagnosed with cystic fibrosis at participating sites who are providing data to the Cystic Fibrosis Patient Registry | Biological: ULTRESA<br>* pancreatic enzyme replacement therapy<br>* Other names: pancrelipase;Biological: PANCREAZE<br>* pancreatic enzyme replacement therapy<br>* Other names: pancrelipase;Biological: CREON<br>* pancreatic enzyme replacement therapy<br>* Other names: pancrelipase;Biological: ZENPEP<br>* pancreatic enzyme replacement therapy<br>* Other names: pancrelipase;Biological: other non-sponsor pancreatic enzyme replacement therapy<br>* pancreatic enzyme replacement therapy<br>* Other names: pancrelipase;Biological: PERTZYE<br>* pancreatic enzyme replacement therapy<br>* Other names: pancrelipase;Other: No pancreatic enzyme replacement therapy<br>* Cystic fibrosis patients in the cystic fibrosis registry not receiving any pancreatic enzyme replacement therapy (approximately 10%)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving ZENPEP | Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria. | up to 10 years | 
| Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving ULTRESA | Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria. | up to 10 years | 
| Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving PERTZYE. | Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria. | up to 10 years | 
| Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving CREON | Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria. | up to 10 years | 
| Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving PANCREAZE | Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria. | up to 10 years | 
| Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients not receiving pancreatic enzyme therapy. | Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria. | up to 10 years | 
| Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving non-sponsor pancreatic enzyme replacement therapy | Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria. | up to 10 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Odds ratio (95% confidence interval) to quantify the association between possible risk factors and confirmed fibrosing colonopathy | Potential risk factors include demographics, medical history, and concurrent medications, including daily dosage and length of specific pancreatic enzyme replacement therapy. | up to 10 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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prospective, observational, pancreatic insufficiency, pancreatic enzyme replacement therapy, fibrosing colonopathy, patients with cystic fibrosis