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
- Interventions
- Biological: ULTRESABiological: PANCREAZEBiological: CREONBiological: ZENPEPBiological: other non-sponsor pancreatic enzyme replacement therapyBiological: PERTZYEOther: No pancreatic enzyme replacement therapy
- 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
- Arm && Interventions
Group Intervention Description Cystic fibrosis (CF) patients in the CF Patient Registry ULTRESA Patients diagnosed with cystic fibrosis at participating sites who are providing data to the Cystic Fibrosis Patient Registry Cystic fibrosis (CF) patients in the CF Patient Registry PANCREAZE Patients diagnosed with cystic fibrosis at participating sites who are providing data to the Cystic Fibrosis Patient Registry Cystic fibrosis (CF) patients in the CF Patient Registry CREON Patients diagnosed with cystic fibrosis at participating sites who are providing data to the Cystic Fibrosis Patient Registry Cystic fibrosis (CF) patients in the CF Patient Registry other non-sponsor pancreatic enzyme replacement therapy Patients diagnosed with cystic fibrosis at participating sites who are providing data to the Cystic Fibrosis Patient Registry Cystic fibrosis (CF) patients in the CF Patient Registry ZENPEP Patients diagnosed with cystic fibrosis at participating sites who are providing data to the Cystic Fibrosis Patient Registry Cystic fibrosis (CF) patients in the CF Patient Registry PERTZYE Patients diagnosed with cystic fibrosis at participating sites who are providing data to the Cystic Fibrosis Patient Registry Cystic fibrosis (CF) patients in the CF Patient Registry No pancreatic enzyme replacement therapy Patients diagnosed with cystic fibrosis at participating sites who are providing data to the Cystic Fibrosis Patient Registry
- 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)
Alaska Medical Center /ID# 79256
🇺🇸Anchorage, Alaska, United States
Arkansas Children's Hospital /ID# 79265
🇺🇸Little Rock, Arkansas, United States
University of Arizona Cancer Center - North Campus /ID# 78939
🇺🇸Tucson, Arizona, United States
Loma Linda University /ID# 79237
🇺🇸Loma Linda, California, United States
University of Arkansas for Medical Sciences /ID# 76785
🇺🇸Little Rock, Arkansas, United States
Ucsd /Id# 105039
🇺🇸La Jolla, California, United States
Kaiser Permanente Regional Met /ID# 79235
🇺🇸Los Angeles, California, United States
Miller Childrens Hospital /ID# 78896
🇺🇸Long Beach, California, United States
Usc /Id# 76793
🇺🇸Los Angeles, California, United States
Children's Hosp and Res Ctr /ID# 79295
🇺🇸Oakland, California, United States
Kaiser Permanente /ID# 78899
🇺🇸Oakland, California, United States
Stanford University /ID# 95156
🇺🇸Stanford, California, United States
Tufts-New England Medical Ctr /ID# 79254
🇺🇸Boston, Massachusetts, United States
CT Childrens Medical Ctr, US /ID# 78923
🇺🇸Hartford, Connecticut, United States
Children's National Medical Center /ID# 79373
🇺🇸Washington, District of Columbia, United States
Nemours/Alfred duPont Hospital for Children /ID# 78945
🇺🇸Wilmington, Delaware, United States
Central FL Pulmonary Altamonte /ID# 76776
🇺🇸Altamonte Springs, Florida, United States
Joe DiMaggio Children's Hospital at Memorial /ID# 79234
🇺🇸Hollywood, Florida, United States
Nemour Child Clin FL, US /ID# 78898
🇺🇸Jacksonville, Florida, United States
All Children's Hospital /ID# 79264
🇺🇸Saint Petersburg, Florida, United States
Emory University Hospital /ID# 79394
🇺🇸Atlanta, Georgia, United States
Children's Healthcare of Atlanta - Ferry Rd /ID# 116275
🇺🇸Atlanta, Georgia, United States
St Luke's Cystic Fibrosis Center of Idaho /ID# 79259
🇺🇸Boise, Idaho, United States
Duplicate_Childrens Hospital of Chicago /ID# 79296
🇺🇸Chicago, Illinois, United States
University of Chicago /ID# 78924
🇺🇸Chicago, Illinois, United States
Loyola University Medical Ctr /ID# 76780
🇺🇸Maywood, Illinois, United States
Georgia Regents University /ID# 79395
🇺🇸Augusta, Georgia, United States
Advocate Lutheran Gen Children /ID# 76781
🇺🇸Park Ridge, Illinois, United States
OSF Saint Francis Medical Ctr /ID# 78905
🇺🇸Peoria, Illinois, United States
Lutheran Hospital of Indiana /ID# 79238
🇺🇸Fort Wayne, Indiana, United States
University of Iowa Hospitals and Clinics /ID# 78921
🇺🇸Iowa City, Iowa, United States
University of Kansas Health Sy /ID# 78929
🇺🇸Kansas City, Kansas, United States
Via Christi Specialty Clinics /ID# 78946
🇺🇸Wichita, Kansas, United States
Louisiana State Univ HSC /ID# 78943
🇺🇸Shreveport, Louisiana, United States
MMP Women's Health /ID# 78894
🇺🇸Portland, Maine, United States
Johns Hopkins University /ID# 78893
🇺🇸Baltimore, Maryland, United States
Boston Childrens Hospital /ID# 79276
🇺🇸Boston, Massachusetts, United States
Massachusetts General Hospital /ID# 78895
🇺🇸Boston, Massachusetts, United States
Baystate Medical Center /ID# 103437
🇺🇸Springfield, Massachusetts, United States
UMass Memorial Medical Center /ID# 78954
🇺🇸Worcester, Massachusetts, United States
Michigan State University /ID# 78950
🇺🇸East Lansing, Michigan, United States
Helen DeVos Childrens Hospital /ID# 79397
🇺🇸Grand Rapids, Michigan, United States
Univ of Mississippi Med Ctr,US /ID# 78934
🇺🇸Jackson, Mississippi, United States
University of Missouri /ID# 109755
🇺🇸Columbia, Missouri, United States
Cardinal Glennon Children's Ho /ID# 79275
🇺🇸Saint Louis, Missouri, United States
Dartmouth-Hitchcock Medical Center /ID# 78957
🇺🇸Lebanon, New Hampshire, United States
Children's Hospital St Louis /ID# 78910
🇺🇸Saint Louis, Missouri, United States
St. Barnabas Medical Center /ID# 79258
🇺🇸Livingston, New Jersey, United States
Monmouth Medical Center /ID# 78901
🇺🇸Long Branch, New Jersey, United States
Rutgers Robert Wood Johnson /ID# 119978
🇺🇸New Brunswick, New Jersey, United States
Goryeb Chidlren's Hospital /ID# 76782
🇺🇸Morristown, New Jersey, United States
Albany Medical College /ID# 76774
🇺🇸Albany, New York, United States
Cystic Fibrosis Therapeutics /ID# 78941
🇺🇸Buffalo, New York, United States
North Shore University Hospital /ID# 76779
🇺🇸New Hyde Park, New York, United States
Univ Rochester Med Ctr /ID# 92713
🇺🇸Rochester, New York, United States
Mount Sinai Beth Israel /ID# 76773
🇺🇸New York, New York, United States
Columbia Univ Medical Center /ID# 79376
🇺🇸New York, New York, United States
SUNY Upstate Medical University - Downtown /ID# 78912
🇺🇸Syracuse, New York, United States
New York Medical College /ID# 78956
🇺🇸Valhalla, New York, United States
Akron Childrens Hospital /ID# 79263
🇺🇸Akron, Ohio, United States
Mid Dakota Clinic, PC /ID# 78958
🇺🇸Bismarck, North Dakota, United States
Case Western Reserve Univ /ID# 78904
🇺🇸Cleveland, Ohio, United States
Nationwide Childrens Hospital /ID# 78897
🇺🇸Columbus, Ohio, United States
University of Miami, Miller School of Medicine /ID# 78927
🇺🇸Miami, Florida, United States
The Childrens Medical Center /ID# 78915
🇺🇸Dayton, Ohio, United States
ProMedica Toledo Hospital /ID# 78913
🇺🇸Toledo, Ohio, United States
Geisinger Medical Center /ID# 79233
🇺🇸Danville, Pennsylvania, United States
Penn State Child Hosp.Hersh,PA /ID# 79398
🇺🇸Hershey, Pennsylvania, United States
Indiana University /ID# 121395
🇺🇸Indianapolis, Indiana, United States
Children's Hosp Philadelphia /ID# 79280
🇺🇸Philadelphia, Pennsylvania, United States
Indiana University /ID# 78903
🇺🇸Indianapolis, Indiana, United States
Baylor College of Medicine /ID# 79273
🇺🇸Houston, Texas, United States
Drexel Univ College of Med /ID# 76777
🇺🇸Philadelphia, Pennsylvania, United States
Seattle Children's Hospital /ID# 78902
🇺🇸Seattle, Washington, United States
St. Christopher's Hospital /ID# 78908
🇺🇸Philadelphia, Pennsylvania, United States
Child Hosp of Pittsburgh,PA /ID# 79281
🇺🇸Pittsburgh, Pennsylvania, United States
Rhode Island Hospital /ID# 79274
🇺🇸Providence, Rhode Island, United States
Sanford Research/USD /ID# 78909
🇺🇸Sioux Falls, South Dakota, United States
LeBonheur Children's Med Ctr /ID# 78938
🇺🇸Memphis, Tennessee, United States
UT Southwestern Medical Center /ID# 78914
🇺🇸Dallas, Texas, United States
Cook Children's Med. Center /ID# 78949
🇺🇸Fort Worth, Texas, United States
The Univ Texas HSC at Tyler /ID# 79261
🇺🇸Tyler, Texas, United States
University of Vermont Medical Center /ID# 78933
🇺🇸Burlington, Vermont, United States
University of Virginia /ID# 78942
🇺🇸Charlottesville, Virginia, United States
Child Hosp of the King's Dtr's /ID# 79293
🇺🇸Norfolk, Virginia, United States
Arthritis Northwest, PLLC /ID# 79257
🇺🇸Spokane, Washington, United States
West Virginia Univ School Med /ID# 78953
🇺🇸Morgantown, West Virginia, United States
Tulane Univ /ID# 78916
🇺🇸New Orleans, Louisiana, United States
Cincinnati Children's Hospital /ID# 79375
🇺🇸Cincinnati, Ohio, United States
University of Cincinnati /ID# 76789
🇺🇸Cincinnati, Ohio, United States
University of Minnesota /ID# 76791
🇺🇸Minneapolis, Minnesota, United States
Duke University Medical Center /ID# 79393
🇺🇸Durham, North Carolina, United States
Univ Texas HSC San Antonio /ID# 79374
🇺🇸San Antonio, Texas, United States
Phoenix Children's Hospital /ID# 78906
🇺🇸Phoenix, Arizona, United States
California Pacific Medical Ctr /ID# 76775
🇺🇸San Francisco, California, United States
Univ California, San Francisco /ID# 78925
🇺🇸San Francisco, California, United States
Children's Hospital of MI /ID# 79278
🇺🇸Detroit, Michigan, United States
Univ Oklahoma HSC /ID# 79279
🇺🇸Oklahoma City, Oklahoma, United States
Vanderbilt Univ Med Ctr /ID# 78947
🇺🇸Nashville, Tennessee, United States
University of Utah /ID# 76794
🇺🇸Salt Lake City, Utah, United States
Children's Hospital of Alabama /ID# 78920
🇺🇸Birmingham, Alabama, United States
National Jewish Health /ID# 76784
🇺🇸Denver, Colorado, United States
Kaiser Permanente, NW /ID# 79236
🇺🇸Portland, Oregon, United States
Oregon Health and Science University /ID# 78900
🇺🇸Portland, Oregon, United States
UC Davis Comprehensive Cancer Center - Main /ID# 118935
🇺🇸Sacramento, California, United States
Univ of Colorado Cancer Center /ID# 79277
🇺🇸Aurora, Colorado, United States
Univ Nebraska Med Ctr /ID# 78932
🇺🇸Omaha, Nebraska, United States
Froedtert & the Medical College of Wisconsin /ID# 76778
🇺🇸Milwaukee, Wisconsin, United States
Children's Hospital Wisconsin - Milwaukee Campus /ID# 79294
🇺🇸Milwaukee, Wisconsin, United States
Nemours Children's Clinic /ID# 78917
🇺🇸Orlando, Florida, United States
Arnold Palmer Hosp Children /ID# 96075
🇺🇸Orlando, Florida, United States
University of Louisville /ID# 78926
🇺🇸Louisville, Kentucky, United States
University of Michigan Hospitals /ID# 99035
🇺🇸Ann Arbor, Michigan, United States
Yale University /ID# 78940
🇺🇸New Haven, Connecticut, United States
The Children's Mercy Hospital /ID# 78948
🇺🇸Kansas City, Missouri, United States
Univ NC Chapel Hill /ID# 78937
🇺🇸Chapel Hill, North Carolina, United States
Medical University of South Carolina /ID# 79262
🇺🇸Charleston, South Carolina, United States
University of Florida - Archer /ID# 78928
🇺🇸Gainesville, Florida, United States
Specially for Children /ID# 79213
🇺🇸Austin, Texas, United States
Children's Hospital of Richmon /ID# 78955
🇺🇸Richmond, Virginia, United States
University of Florida - Archer /ID# 76790
🇺🇸Gainesville, Florida, United States
Kentucky Clinic /ID# 78922
🇺🇸Lexington, Kentucky, United States
Univ of Wisconsin Hosp/Clinics /ID# 78944
🇺🇸Madison, Wisconsin, United States