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)
- Conditions
- Fibrosing Colonopathy
- Registration Number
- NCT01652157
- Lead Sponsor
- AbbVie
- Brief Summary
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
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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 17
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
- None
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving PERTZYE. up to 10 years Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria.
Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving CREON up to 10 years Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria.
Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving PANCREAZE up to 10 years Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria.
Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving non-sponsor pancreatic enzyme replacement therapy up to 10 years Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria.
Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving ZENPEP up to 10 years Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria.
Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients not receiving pancreatic enzyme therapy. up to 10 years Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria.
Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving ULTRESA up to 10 years Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria.
- Secondary Outcome Measures
Name Time Method Odds ratio (95% confidence interval) to quantify the association between possible risk factors and confirmed fibrosing colonopathy up to 10 years Potential risk factors include demographics, medical history, and concurrent medications, including daily dosage and length of specific pancreatic enzyme replacement therapy.
Trial Locations
- Locations (123)
Children's Hospital of Alabama /ID# 78920
🇺🇸Birmingham, Alabama, United States
Alaska Medical Center /ID# 79256
🇺🇸Anchorage, Alaska, United States
Phoenix Children's Hospital /ID# 78906
🇺🇸Phoenix, Arizona, United States
University of Arizona Cancer Center - North Campus /ID# 78939
🇺🇸Tucson, Arizona, United States
Arkansas Children's Hospital /ID# 79265
🇺🇸Little Rock, Arkansas, United States
University of Arkansas for Medical Sciences /ID# 76785
🇺🇸Little Rock, Arkansas, United States
Ucsd /Id# 105039
🇺🇸La Jolla, California, United States
Loma Linda University /ID# 79237
🇺🇸Loma Linda, California, United States
Miller Childrens Hospital /ID# 78896
🇺🇸Long Beach, California, United States
Kaiser Permanente Regional Met /ID# 79235
🇺🇸Los Angeles, California, United States
Scroll for more (113 remaining)Children's Hospital of Alabama /ID# 78920🇺🇸Birmingham, Alabama, United States