Clinical trial NCT01652157: 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)
Official title: 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)
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Fibrosing Colonopathy
Keywords: prospective
observational
pancreatic insufficiency
pancreatic enzyme replacement therapy
fibrosing colonopathy
patients with cystic fibrosis
Lead sponsor: AbbVie
Sponsor class: INDUSTRY
Collaborators: name: Cystic Fibrosis Foundation
name: Digestive Care, Inc.
name: Nestle Health Science
name: VIVUS LLC
First posted: 2012-07-27
Enrollment: 17
Sex: ALL
Minimum age (years): 0
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Cystic fibrosis patients who are active in the Cystic Fibrosis Patient Registry

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.

Eligibility criteria: 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

Primary outcomes:
measure: Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving ZENPEP
timeFrame: up to 10 years
measure: Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving ULTRESA
timeFrame: up to 10 years
measure: Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving PERTZYE.
timeFrame: up to 10 years
measure: Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving CREON
timeFrame: up to 10 years
measure: Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving PANCREAZE
timeFrame: up to 10 years
measure: Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients not receiving pancreatic enzyme therapy.
timeFrame: up to 10 years
measure: Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving non-sponsor pancreatic enzyme replacement therapy
timeFrame: up to 10 years
Secondary outcomes:
measure: Odds ratio (95% confidence interval) to quantify the association between possible risk factors and confirmed fibrosing colonopathy
timeFrame: up to 10 years
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Children's Hospital of Alabama /ID# 78920
state: Alabama
city: Anchorage
country: United States
facility: Alaska Medical Center /ID# 79256
state: Alaska
city: Phoenix
country: United States
facility: Phoenix Children's Hospital /ID# 78906
state: Arizona
city: Tucson
country: United States
facility: University of Arizona Cancer Center - North Campus /ID# 78939
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Children's Hospital /ID# 79265
state: Arkansas
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences /ID# 76785
state: Arkansas
city: La Jolla
country: United States
facility: Ucsd /Id# 105039
state: California
city: Loma Linda
country: United States
facility: Loma Linda University /ID# 79237
state: California
city: Long Beach
country: United States
facility: Miller Childrens Hospital /ID# 78896
state: California
city: Los Angeles
country: United States
facility: Kaiser Permanente Regional Met /ID# 79235
state: California
city: Los Angeles
country: United States
facility: Usc /Id# 76793
state: California
city: Oakland
country: United States
facility: Children's Hosp and Res Ctr /ID# 79295
state: California
city: Oakland
country: United States
facility: Kaiser Permanente /ID# 78899
state: California
city: Sacramento
country: United States
facility: UC Davis Comprehensive Cancer Center - Main /ID# 118935
state: California
city: San Francisco
country: United States
facility: California Pacific Medical Ctr /ID# 76775
state: California
city: San Francisco
country: United States
facility: Univ California, San Francisco /ID# 78925
state: California
city: Stanford
country: United States
facility: Stanford University /ID# 95156
state: California
city: Aurora
country: United States
facility: Univ of Colorado Cancer Center /ID# 79277
state: Colorado
city: Denver
country: United States
facility: National Jewish Health /ID# 76784
state: Colorado
city: Hartford
country: United States
facility: CT Childrens Medical Ctr, US /ID# 78923
state: Connecticut
city: New Haven
country: United States
facility: Yale University /ID# 78940
state: Connecticut
city: Wilmington
country: United States
facility: Nemours/Alfred duPont Hospital for Children /ID# 78945
state: Delaware
city: Washington
country: United States
facility: Children's National Medical Center /ID# 79373
state: District of Columbia
city: Altamonte Springs
country: United States
facility: Central FL Pulmonary Altamonte /ID# 76776
state: Florida
city: Gainesville
country: United States
facility: University of Florida - Archer /ID# 76790
state: Florida
city: Gainesville
country: United States
facility: University of Florida - Archer /ID# 78928
state: Florida
city: Hollywood
country: United States
facility: Joe DiMaggio Children's Hospital at Memorial /ID# 79234
state: Florida
city: Jacksonville
country: United States
facility: Nemour Child Clin FL, US /ID# 78898
state: Florida
city: Miami
country: United States
facility: University of Miami, Miller School of Medicine /ID# 78927
state: Florida
city: Orlando
country: United States
facility: Nemours Children's Clinic /ID# 78917
state: Florida
city: Orlando
country: United States
facility: Arnold Palmer Hosp Children /ID# 96075
state: Florida
city: Saint Petersburg
country: United States
facility: All Children's Hospital /ID# 79264
state: Florida
city: Atlanta
country: United States
facility: Emory University Hospital /ID# 79394
state: Georgia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta - Ferry Rd /ID# 116275
state: Georgia
city: Augusta
country: United States
facility: Georgia Regents University /ID# 79395
state: Georgia
city: Boise
country: United States
facility: St Luke's Cystic Fibrosis Center of Idaho /ID# 79259
state: Idaho
city: Chicago
country: United States
facility: Duplicate_Childrens Hospital of Chicago /ID# 79296
state: Illinois
city: Chicago
country: United States
facility: University of Chicago /ID# 78924
state: Illinois
city: Maywood
country: United States
facility: Loyola University Medical Ctr /ID# 76780
state: Illinois
city: Park Ridge
country: United States
facility: Advocate Lutheran Gen Children /ID# 76781
state: Illinois
city: Peoria
country: United States
facility: OSF Saint Francis Medical Ctr /ID# 78905
state: Illinois
city: Fort Wayne
country: United States
facility: Lutheran Hospital of Indiana /ID# 79238
state: Indiana
city: Indianapolis
country: United States
facility: Indiana University /ID# 121395
state: Indiana
city: Indianapolis
country: United States
facility: Indiana University /ID# 78903
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics /ID# 78921
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Health Sy /ID# 78929
state: Kansas
city: Wichita
country: United States
facility: Via Christi Specialty Clinics /ID# 78946
state: Kansas
city: Lexington
country: United States
facility: Kentucky Clinic /ID# 78922
state: Kentucky
city: Louisville
country: United States
facility: University of Louisville /ID# 78926
state: Kentucky
city: New Orleans
country: United States
facility: Tulane Univ /ID# 78916
state: Louisiana
city: Shreveport
country: United States
facility: Louisiana State Univ HSC /ID# 78943
state: Louisiana
city: Portland
country: United States
facility: MMP Women's Health /ID# 78894
state: Maine
city: Baltimore
country: United States
facility: Johns Hopkins University /ID# 78893
state: Maryland
city: Boston
country: United States
facility: Tufts-New England Medical Ctr /ID# 79254
state: Massachusetts
city: Boston
country: United States
facility: Massachusetts General Hospital /ID# 78895
state: Massachusetts
city: Boston
country: United States
facility: Boston Childrens Hospital /ID# 79276
state: Massachusetts
city: Springfield
country: United States
facility: Baystate Medical Center /ID# 103437
state: Massachusetts
city: Worcester
country: United States
facility: UMass Memorial Medical Center /ID# 78954
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Hospitals /ID# 99035
state: Michigan
city: Detroit
country: United States
facility: Children's Hospital of MI /ID# 79278
state: Michigan
city: East Lansing
country: United States
facility: Michigan State University /ID# 78950
state: Michigan
city: Grand Rapids
country: United States
facility: Helen DeVos Childrens Hospital /ID# 79397
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota /ID# 76791
state: Minnesota
city: Jackson
country: United States
facility: Univ of Mississippi Med Ctr,US /ID# 78934
state: Mississippi
city: Columbia
country: United States
facility: University of Missouri /ID# 109755
state: Missouri
city: Kansas City
country: United States
facility: The Children's Mercy Hospital /ID# 78948
state: Missouri
city: Saint Louis
country: United States
facility: Cardinal Glennon Children's Ho /ID# 79275
state: Missouri
city: Saint Louis
country: United States
facility: Children's Hospital St Louis /ID# 78910
state: Missouri
city: Omaha
country: United States
facility: Univ Nebraska Med Ctr /ID# 78932
state: Nebraska
city: Lebanon
country: United States
facility: Dartmouth-Hitchcock Medical Center /ID# 78957
state: New Hampshire
city: Livingston
country: United States
facility: St. Barnabas Medical Center /ID# 79258
state: New Jersey
city: Long Branch
country: United States
facility: Monmouth Medical Center /ID# 78901
state: New Jersey
city: Morristown
country: United States
facility: Goryeb Chidlren's Hospital /ID# 76782
state: New Jersey
city: New Brunswick
country: United States
facility: Rutgers Robert Wood Johnson /ID# 119978
state: New Jersey
city: Albany
country: United States
facility: Albany Medical College /ID# 76774
state: New York
city: Buffalo
country: United States
facility: Cystic Fibrosis Therapeutics /ID# 78941
state: New York
city: New Hyde Park
country: United States
facility: North Shore University Hospital /ID# 76779
state: New York
city: New York
country: United States
facility: Mount Sinai Beth Israel /ID# 76773
state: New York
city: New York
country: United States
facility: Columbia Univ Medical Center /ID# 79376
state: New York
city: Rochester
country: United States
facility: Univ Rochester Med Ctr /ID# 92713
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Medical University - Downtown /ID# 78912
state: New York
city: Valhalla
country: United States
facility: New York Medical College /ID# 78956
state: New York
city: Chapel Hill
country: United States
facility: Univ NC Chapel Hill /ID# 78937
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center /ID# 79393
state: North Carolina
city: Bismarck
country: United States
facility: Mid Dakota Clinic, PC /ID# 78958
state: North Dakota
city: Akron
country: United States
facility: Akron Childrens Hospital /ID# 79263
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital /ID# 79375
state: Ohio
city: Cincinnati
country: United States
facility: University of Cincinnati /ID# 76789
state: Ohio
city: Cleveland
country: United States
facility: Case Western Reserve Univ /ID# 78904
state: Ohio
city: Columbus
country: United States
facility: Nationwide Childrens Hospital /ID# 78897
state: Ohio
city: Dayton
country: United States
facility: The Childrens Medical Center /ID# 78915
state: Ohio
city: Toledo
country: United States
facility: ProMedica Toledo Hospital /ID# 78913
state: Ohio
city: Oklahoma City
country: United States
facility: Univ Oklahoma HSC /ID# 79279
state: Oklahoma
city: Portland
country: United States
facility: Kaiser Permanente, NW /ID# 79236
state: Oregon
city: Portland
country: United States
facility: Oregon Health and Science University /ID# 78900
state: Oregon
city: Danville
country: United States
facility: Geisinger Medical Center /ID# 79233
state: Pennsylvania
city: Hershey
country: United States
facility: Penn State Child Hosp.Hersh,PA /ID# 79398
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hosp Philadelphia /ID# 79280
state: Pennsylvania
city: Philadelphia
country: United States
facility: Drexel Univ College of Med /ID# 76777
state: Pennsylvania
city: Philadelphia
country: United States
facility: St. Christopher's Hospital /ID# 78908
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Child Hosp of Pittsburgh,PA /ID# 79281
state: Pennsylvania
city: Providence
country: United States
facility: Rhode Island Hospital /ID# 79274
state: Rhode Island
city: Charleston
country: United States
facility: Medical University of South Carolina /ID# 79262
state: South Carolina
city: Sioux Falls
country: United States
facility: Sanford Research/USD /ID# 78909
state: South Dakota
city: Memphis
country: United States
facility: LeBonheur Children's Med Ctr /ID# 78938
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt Univ Med Ctr /ID# 78947
state: Tennessee
city: Austin
country: United States
facility: Specially for Children /ID# 79213
state: Texas
city: Dallas
country: United States
facility: UT Southwestern Medical Center /ID# 78914
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Med. Center /ID# 78949
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine /ID# 79273
state: Texas
city: San Antonio
country: United States
facility: Univ Texas HSC San Antonio /ID# 79374
state: Texas
city: Tyler
country: United States
facility: The Univ Texas HSC at Tyler /ID# 79261
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah /ID# 76794
state: Utah
city: Burlington
country: United States
facility: University of Vermont Medical Center /ID# 78933
state: Vermont
city: Charlottesville
country: United States
facility: University of Virginia /ID# 78942
state: Virginia
city: Norfolk
country: United States
facility: Child Hosp of the King's Dtr's /ID# 79293
state: Virginia
city: Richmond
country: United States
facility: Children's Hospital of Richmon /ID# 78955
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital /ID# 78902
state: Washington
city: Spokane
country: United States
facility: Arthritis Northwest, PLLC /ID# 79257
state: Washington
city: Morgantown
country: United States
facility: West Virginia Univ School Med /ID# 78953
state: West Virginia
city: Madison
country: United States
facility: Univ of Wisconsin Hosp/Clinics /ID# 78944
state: Wisconsin
city: Milwaukee
country: United States
facility: Froedtert & the Medical College of Wisconsin /ID# 76778
state: Wisconsin
city: Milwaukee
country: United States
facility: Children's Hospital Wisconsin - Milwaukee Campus /ID# 79294
state: Wisconsin
Study officials:
affiliation: VIVUS LLC
name: Craig Peterson
role: STUDY_DIRECTOR
References: