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)

March 18, 2021 updated by: AbbVie

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)

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.

Study Overview

Status

Terminated

Conditions

Fibrosing Colonopathy

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Actual)

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

United States
- Alabama
  - Birmingham, Alabama, United States, 35233
    - Children's Hospital of Alabama /ID# 78920
- Alaska
  - Anchorage, Alaska, United States, 99508
    - Alaska Medical Center /ID# 79256
- Arizona
  - Phoenix, Arizona, United States, 85016-7710
    - Phoenix Children's Hospital /ID# 78906
  - Tucson, Arizona, United States, 85719-1478
    - University of Arizona Cancer Center - North Campus /ID# 78939
- Arkansas
  - Little Rock, Arkansas, United States, 72202
    - Arkansas Children's Hospital /ID# 79265
  - Little Rock, Arkansas, United States, 72205
    - University of Arkansas for Medical Sciences /ID# 76785
- California
  - La Jolla, California, United States, 92093
    - Ucsd /Id# 105039
  - Loma Linda, California, United States, 92354
    - Loma Linda University /ID# 79237
  - Long Beach, California, United States, 90806
    - Miller Childrens Hospital /ID# 78896
  - Los Angeles, California, United States, 90027
    - Kaiser Permanente Regional Met /ID# 79235
  - Los Angeles, California, United States, 90033
    - Usc /Id# 76793
  - Oakland, California, United States, 94609
    - Children's Hosp and Res Ctr /ID# 79295
  - Oakland, California, United States, 94611
    - Kaiser Permanente /ID# 78899
  - Sacramento, California, United States, 95817
    - UC Davis Comprehensive Cancer Center - Main /ID# 118935
  - San Francisco, California, United States, 94115
    - California Pacific Medical Ctr /ID# 76775
  - San Francisco, California, United States, 94143-2204
    - Univ California, San Francisco /ID# 78925
  - Stanford, California, United States, 94305
    - Stanford University /ID# 95156
- Colorado
  - Aurora, Colorado, United States, 80045
    - Univ of Colorado Cancer Center /ID# 79277
  - Denver, Colorado, United States, 80206
    - National Jewish Health /ID# 76784
- Connecticut
  - Hartford, Connecticut, United States, 06106
    - CT Childrens Medical Ctr, US /ID# 78923
  - New Haven, Connecticut, United States, 06510
    - Yale University /ID# 78940
- Delaware
  - Wilmington, Delaware, United States, 19803
    - Nemours/Alfred duPont Hospital for Children /ID# 78945
- District of Columbia
  - Washington, District of Columbia, United States, 20010
    - Children's National Medical Center /ID# 79373
- Florida
  - Altamonte Springs, Florida, United States, 32701
    - Central FL Pulmonary Altamonte /ID# 76776
  - Gainesville, Florida, United States, 32610
    - University of Florida - Archer /ID# 76790
  - Gainesville, Florida, United States, 32610
    - University of Florida - Archer /ID# 78928
  - Hollywood, Florida, United States, 33021-5402
    - Joe DiMaggio Children's Hospital at Memorial /ID# 79234
  - Jacksonville, Florida, United States, 32207
    - Nemour Child Clin FL, US /ID# 78898
  - Miami, Florida, United States, 33136
    - University of Miami, Miller School of Medicine /ID# 78927
  - Orlando, Florida, United States, 32801
    - Nemours Children's Clinic /ID# 78917
  - Orlando, Florida, United States, 32806
    - Arnold Palmer Hosp Children /ID# 96075
  - Saint Petersburg, Florida, United States, 33701
    - All Children's Hospital /ID# 79264
- Georgia
  - Atlanta, Georgia, United States, 30322
    - Emory University Hospital /ID# 79394
  - Atlanta, Georgia, United States, 30342-1605
    - Children's Healthcare of Atlanta - Ferry Rd /ID# 116275
  - Augusta, Georgia, United States, 30912
    - Georgia Regents University /ID# 79395
- Idaho
  - Boise, Idaho, United States, 83702
    - St Luke's Cystic Fibrosis Center of Idaho /ID# 79259
- Illinois
  - Chicago, Illinois, United States, 60611
    - Duplicate_Childrens Hospital of Chicago /ID# 79296
  - Chicago, Illinois, United States, 60637-1443
    - University of Chicago /ID# 78924
  - Maywood, Illinois, United States, 60153
    - Loyola University Medical Ctr /ID# 76780
  - Park Ridge, Illinois, United States, 60068
    - Advocate Lutheran Gen Children /ID# 76781
  - Peoria, Illinois, United States, 61603
    - OSF Saint Francis Medical Ctr /ID# 78905
- Indiana
  - Fort Wayne, Indiana, United States, 46804
    - Lutheran Hospital of Indiana /ID# 79238
  - Indianapolis, Indiana, United States, 46202
    - Indiana University /ID# 121395
  - Indianapolis, Indiana, United States, 46202
    - Indiana University /ID# 78903
- Iowa
  - Iowa City, Iowa, United States, 52242
    - University of Iowa Hospitals and Clinics /ID# 78921
- Kansas
  - Kansas City, Kansas, United States, 66160-8500
    - University of Kansas Health Sy /ID# 78929
  - Wichita, Kansas, United States, 67214
    - Via Christi Specialty Clinics /ID# 78946
- Kentucky
  - Lexington, Kentucky, United States, 40506
    - Kentucky Clinic /ID# 78922
  - Louisville, Kentucky, United States, 40202
    - University of Louisville /ID# 78926
- Louisiana
  - New Orleans, Louisiana, United States, 70112
    - Tulane Univ /ID# 78916
  - Shreveport, Louisiana, United States, 71130
    - Louisiana State Univ HSC /ID# 78943
- Maine
  - Portland, Maine, United States, 04102
    - MMP Women's Health /ID# 78894
- Maryland
  - Baltimore, Maryland, United States, 21287
    - Johns Hopkins University /ID# 78893
- Massachusetts
  - Boston, Massachusetts, United States, 02111
    - Tufts-New England Medical Ctr /ID# 79254
  - Boston, Massachusetts, United States, 02114
    - Massachusetts General Hospital /ID# 78895
  - Boston, Massachusetts, United States, 02115
    - Boston Childrens Hospital /ID# 79276
  - Springfield, Massachusetts, United States, 01199
    - Baystate Medical Center /ID# 103437
  - Worcester, Massachusetts, United States, 01655
    - UMass Memorial Medical Center /ID# 78954
- Michigan
  - Ann Arbor, Michigan, United States, 48109
    - University of Michigan Hospitals /ID# 99035
  - Detroit, Michigan, United States, 48201
    - Children's Hospital of MI /ID# 79278
  - East Lansing, Michigan, United States, 48824
    - Michigan State University /ID# 78950
  - Grand Rapids, Michigan, United States, 49503
    - Helen DeVos Childrens Hospital /ID# 79397
- Minnesota
  - Minneapolis, Minnesota, United States, 55455-0356
    - University of Minnesota /ID# 76791
- Mississippi
  - Jackson, Mississippi, United States, 39216-4643
    - Univ of Mississippi Med Ctr,US /ID# 78934
- Missouri
  - Columbia, Missouri, United States, 65212
    - University of Missouri /ID# 109755
  - Kansas City, Missouri, United States, 64108
    - The Children's Mercy Hospital /ID# 78948
  - Saint Louis, Missouri, United States, 63104
    - Cardinal Glennon Children's Ho /ID# 79275
  - Saint Louis, Missouri, United States, 63110
    - Children's Hospital St Louis /ID# 78910
- Nebraska
  - Omaha, Nebraska, United States, 68198
    - Univ Nebraska Med Ctr /ID# 78932
- New Hampshire
  - Lebanon, New Hampshire, United States, 03756
    - Dartmouth-Hitchcock Medical Center /ID# 78957
- New Jersey
  - Livingston, New Jersey, United States, 07039
    - St. Barnabas Medical Center /ID# 79258
  - Long Branch, New Jersey, United States, 07740
    - Monmouth Medical Center /ID# 78901
  - Morristown, New Jersey, United States, 07960
    - Goryeb Chidlren's Hospital /ID# 76782
  - New Brunswick, New Jersey, United States, 08901
    - Rutgers Robert Wood Johnson /ID# 119978
- New York
  - Albany, New York, United States, 12208
    - Albany Medical College /ID# 76774
  - Buffalo, New York, United States, 14222
    - Cystic Fibrosis Therapeutics /ID# 78941
  - New Hyde Park, New York, United States, 11040
    - North Shore University Hospital /ID# 76779
  - New York, New York, United States, 10003
    - Mount Sinai Beth Israel /ID# 76773
  - New York, New York, United States, 10032-3725
    - Columbia Univ Medical Center /ID# 79376
  - Rochester, New York, United States, 14642
    - Univ Rochester Med Ctr /ID# 92713
  - Syracuse, New York, United States, 13210
    - SUNY Upstate Medical University - Downtown /ID# 78912
  - Valhalla, New York, United States, 10595
    - New York Medical College /ID# 78956
- North Carolina
  - Chapel Hill, North Carolina, United States, 27514-4220
    - Univ NC Chapel Hill /ID# 78937
  - Durham, North Carolina, United States, 27710-3000
    - Duke University Medical Center /ID# 79393
- North Dakota
  - Bismarck, North Dakota, United States, 58504
    - Mid Dakota Clinic, PC /ID# 78958
- Ohio
  - Akron, Ohio, United States, 44308
    - Akron Childrens Hospital /ID# 79263
  - Cincinnati, Ohio, United States, 45229
    - Cincinnati Children's Hospital /ID# 79375
  - Cincinnati, Ohio, United States, 45267-0585
    - University of Cincinnati /ID# 76789
  - Cleveland, Ohio, United States, 44106
    - Case Western Reserve Univ /ID# 78904
  - Columbus, Ohio, United States, 43205
    - Nationwide Childrens Hospital /ID# 78897
  - Dayton, Ohio, United States, 45404
    - The Childrens Medical Center /ID# 78915
  - Toledo, Ohio, United States, 43606
    - ProMedica Toledo Hospital /ID# 78913
- Oklahoma
  - Oklahoma City, Oklahoma, United States, 73104
    - Univ Oklahoma HSC /ID# 79279
- Oregon
  - Portland, Oregon, United States, 97227
    - Kaiser Permanente, NW /ID# 79236
  - Portland, Oregon, United States, 97239
    - Oregon Health and Science University /ID# 78900
- Pennsylvania
  - Danville, Pennsylvania, United States, 17822
    - Geisinger Medical Center /ID# 79233
  - Hershey, Pennsylvania, United States, 17033
    - Penn State Child Hosp.Hersh,PA /ID# 79398
  - Philadelphia, Pennsylvania, United States, 19104
    - Children's Hosp Philadelphia /ID# 79280
  - Philadelphia, Pennsylvania, United States, 19129
    - Drexel Univ College of Med /ID# 76777
  - Philadelphia, Pennsylvania, United States, 19134
    - St. Christopher's Hospital /ID# 78908
  - Pittsburgh, Pennsylvania, United States, 15213-2583
    - Child Hosp of Pittsburgh,PA /ID# 79281
- Rhode Island
  - Providence, Rhode Island, United States, 02903
    - Rhode Island Hospital /ID# 79274
- South Carolina
  - Charleston, South Carolina, United States, 29425
    - Medical University of South Carolina /ID# 79262
- South Dakota
  - Sioux Falls, South Dakota, United States, 57104-8805
    - Sanford Research/USD /ID# 78909
- Tennessee
  - Memphis, Tennessee, United States, 38103
    - LeBonheur Children's Med Ctr /ID# 78938
  - Nashville, Tennessee, United States, 37232-0011
    - Vanderbilt Univ Med Ctr /ID# 78947
- Texas
  - Austin, Texas, United States, 78723
    - Specially for Children /ID# 79213
  - Dallas, Texas, United States, 75390-7208
    - UT Southwestern Medical Center /ID# 78914
  - Fort Worth, Texas, United States, 76104
    - Cook Children's Med. Center /ID# 78949
  - Houston, Texas, United States, 77030-3411
    - Baylor College of Medicine /ID# 79273
  - San Antonio, Texas, United States, 78229
    - Univ Texas HSC San Antonio /ID# 79374
  - Tyler, Texas, United States, 75708
    - The Univ Texas HSC at Tyler /ID# 79261
- Utah
  - Salt Lake City, Utah, United States, 84112-5500
    - University of Utah /ID# 76794
- Vermont
  - Burlington, Vermont, United States, 05401-1473
    - University of Vermont Medical Center /ID# 78933
- Virginia
  - Charlottesville, Virginia, United States, 22908
    - University of Virginia /ID# 78942
  - Norfolk, Virginia, United States, 23507
    - Child Hosp of the King's Dtr's /ID# 79293
  - Richmond, Virginia, United States, 23298
    - Children's Hospital of Richmon /ID# 78955
- Washington
  - Seattle, Washington, United States, 98105
    - Seattle Children's Hospital /ID# 78902
  - Spokane, Washington, United States, 99204-2336
    - Arthritis Northwest, PLLC /ID# 79257
- West Virginia
  - Morgantown, West Virginia, United States, 26506
    - West Virginia Univ School Med /ID# 78953
- Wisconsin
  - Madison, Wisconsin, United States, 53792-0001
    - Univ of Wisconsin Hosp/Clinics /ID# 78944
  - Milwaukee, Wisconsin, United States, 53226-3522
    - Froedtert & the Medical College of Wisconsin /ID# 76778
  - Milwaukee, Wisconsin, United States, 53226
    - Children's Hospital Wisconsin - Milwaukee Campus /ID# 79294

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 second to 99 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Cystic fibrosis patients who are active in the Cystic Fibrosis Patient Registry

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Observational Models: Cohort
Time Perspectives: Prospective

Number of groups / cohorts

Cohorts and Interventions

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

Group / Cohort

Intervention / Treatment

Cystic fibrosis (CF) patients in the CF Patient Registry

Patients diagnosed with cystic fibrosis at participating sites who are providing data to the Cystic Fibrosis Patient Registry

Biological: ULTRESA