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)

Fibrosing Colonopathy Clinical Trial

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01652157
• Other study ID #: CFFC-OB-11
• Status: Terminated
• Start date: July 31, 2012
• Est. completion date: April 8, 2020

Study information

• Verified date: March 2021
• Source: AbbVie
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 information / eligibility

• Status: Terminated
• Enrollment: 17
• Est. completion date: April 8, 2020
• Est. primary completion date: April 8, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 99 Years

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosing Colonopathy
• Fibrosis

Intervention

Biological:
• ULTRESA
pancreatic enzyme replacement therapy
• PANCREAZE
pancreatic enzyme replacement therapy
• CREON
pancreatic enzyme replacement therapy
• ZENPEP
pancreatic enzyme replacement therapy
• other non-sponsor pancreatic enzyme replacement therapy
pancreatic enzyme replacement therapy
• PERTZYE
pancreatic enzyme replacement therapy

Other:
• No pancreatic enzyme replacement therapy
Cystic fibrosis patients in the cystic fibrosis registry not receiving any pancreatic enzyme replacement therapy (approximately 10%)

Locations

Country	Name	City	State
United States	Akron Childrens Hospital /ID# 79263	Akron	Ohio
United States	Albany Medical College /ID# 76774	Albany	New York
United States	Central FL Pulmonary Altamonte /ID# 76776	Altamonte Springs	Florida
United States	Alaska Medical Center /ID# 79256	Anchorage	Alaska
United States	University of Michigan Hospitals /ID# 99035	Ann Arbor	Michigan
United States	Children's Healthcare of Atlanta - Ferry Rd /ID# 116275	Atlanta	Georgia
United States	Emory University Hospital /ID# 79394	Atlanta	Georgia
United States	Georgia Regents University /ID# 79395	Augusta	Georgia
United States	Univ of Colorado Cancer Center /ID# 79277	Aurora	Colorado
United States	Specially for Children /ID# 79213	Austin	Texas
United States	Johns Hopkins University /ID# 78893	Baltimore	Maryland
United States	Children's Hospital of Alabama /ID# 78920	Birmingham	Alabama
United States	Mid Dakota Clinic, PC /ID# 78958	Bismarck	North Dakota
United States	St Luke's Cystic Fibrosis Center of Idaho /ID# 79259	Boise	Idaho
United States	Boston Childrens Hospital /ID# 79276	Boston	Massachusetts
United States	Massachusetts General Hospital /ID# 78895	Boston	Massachusetts
United States	Tufts-New England Medical Ctr /ID# 79254	Boston	Massachusetts
United States	Cystic Fibrosis Therapeutics /ID# 78941	Buffalo	New York
United States	University of Vermont Medical Center /ID# 78933	Burlington	Vermont
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 Virginia /ID# 78942	Charlottesville	Virginia
United States	Duplicate_Childrens Hospital of Chicago /ID# 79296	Chicago	Illinois
United States	University of Chicago /ID# 78924	Chicago	Illinois
United States	Cincinnati Children's Hospital /ID# 79375	Cincinnati	Ohio
United States	University of Cincinnati /ID# 76789	Cincinnati	Ohio
United States	Case Western Reserve Univ /ID# 78904	Cleveland	Ohio
United States	University of Missouri /ID# 109755	Columbia	Missouri
United States	Nationwide Childrens Hospital /ID# 78897	Columbus	Ohio
United States	UT Southwestern Medical Center /ID# 78914	Dallas	Texas
United States	Geisinger Medical Center /ID# 79233	Danville	Pennsylvania
United States	The Childrens Medical Center /ID# 78915	Dayton	Ohio
United States	National Jewish Health /ID# 76784	Denver	Colorado
United States	Children's Hospital of MI /ID# 79278	Detroit	Michigan
United States	Duke University Medical Center /ID# 79393	Durham	North Carolina
United States	Michigan State University /ID# 78950	East Lansing	Michigan
United States	Lutheran Hospital of Indiana /ID# 79238	Fort Wayne	Indiana
United States	Cook Children's Med. Center /ID# 78949	Fort Worth	Texas
United States	University of Florida - Archer /ID# 76790	Gainesville	Florida
United States	University of Florida - Archer /ID# 78928	Gainesville	Florida
United States	Helen DeVos Childrens Hospital /ID# 79397	Grand Rapids	Michigan
United States	CT Childrens Medical Ctr, US /ID# 78923	Hartford	Connecticut
United States	Penn State Child Hosp.Hersh,PA /ID# 79398	Hershey	Pennsylvania
United States	Joe DiMaggio Children's Hospital at Memorial /ID# 79234	Hollywood	Florida
United States	Baylor College of Medicine /ID# 79273	Houston	Texas
United States	Indiana University /ID# 121395	Indianapolis	Indiana
United States	Indiana University /ID# 78903	Indianapolis	Indiana
United States	University of Iowa Hospitals and Clinics /ID# 78921	Iowa City	Iowa
United States	Univ of Mississippi Med Ctr,US /ID# 78934	Jackson	Mississippi
United States	Nemour Child Clin FL, US /ID# 78898	Jacksonville	Florida
United States	The Children's Mercy Hospital /ID# 78948	Kansas City	Missouri
United States	University of Kansas Health Sy /ID# 78929	Kansas City	Kansas
United States	Ucsd /Id# 105039	La Jolla	California
United States	Dartmouth-Hitchcock Medical Center /ID# 78957	Lebanon	New Hampshire
United States	Kentucky Clinic /ID# 78922	Lexington	Kentucky
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	St. Barnabas Medical Center /ID# 79258	Livingston	New Jersey
United States	Loma Linda University /ID# 79237	Loma Linda	California
United States	Miller Childrens Hospital /ID# 78896	Long Beach	California
United States	Monmouth Medical Center /ID# 78901	Long Branch	New Jersey
United States	Kaiser Permanente Regional Met /ID# 79235	Los Angeles	California
United States	Usc /Id# 76793	Los Angeles	California
United States	University of Louisville /ID# 78926	Louisville	Kentucky
United States	Univ of Wisconsin Hosp/Clinics /ID# 78944	Madison	Wisconsin
United States	Loyola University Medical Ctr /ID# 76780	Maywood	Illinois
United States	LeBonheur Children's Med Ctr /ID# 78938	Memphis	Tennessee
United States	University of Miami, Miller School of Medicine /ID# 78927	Miami	Florida
United States	Children's Hospital Wisconsin - Milwaukee Campus /ID# 79294	Milwaukee	Wisconsin
United States	Froedtert & the Medical College of Wisconsin /ID# 76778	Milwaukee	Wisconsin
United States	University of Minnesota /ID# 76791	Minneapolis	Minnesota
United States	West Virginia Univ School Med /ID# 78953	Morgantown	West Virginia
United States	Goryeb Chidlren's Hospital /ID# 76782	Morristown	New Jersey
United States	Vanderbilt Univ Med Ctr /ID# 78947	Nashville	Tennessee
United States	Rutgers Robert Wood Johnson /ID# 119978	New Brunswick	New Jersey
United States	Yale University /ID# 78940	New Haven	Connecticut
United States	North Shore University Hospital /ID# 76779	New Hyde Park	New York
United States	Tulane Univ /ID# 78916	New Orleans	Louisiana
United States	Columbia Univ Medical Center /ID# 79376	New York	New York
United States	Mount Sinai Beth Israel /ID# 76773	New York	New York
United States	Child Hosp of the King's Dtr's /ID# 79293	Norfolk	Virginia
United States	Children's Hosp and Res Ctr /ID# 79295	Oakland	California
United States	Kaiser Permanente /ID# 78899	Oakland	California
United States	Univ Oklahoma HSC /ID# 79279	Oklahoma City	Oklahoma
United States	Univ Nebraska Med Ctr /ID# 78932	Omaha	Nebraska
United States	Arnold Palmer Hosp Children /ID# 96075	Orlando	Florida
United States	Nemours Children's Clinic /ID# 78917	Orlando	Florida
United States	Advocate Lutheran Gen Children /ID# 76781	Park Ridge	Illinois
United States	OSF Saint Francis Medical Ctr /ID# 78905	Peoria	Illinois
United States	Children's Hosp Philadelphia /ID# 79280	Philadelphia	Pennsylvania
United States	Drexel Univ College of Med /ID# 76777	Philadelphia	Pennsylvania
United States	St. Christopher's Hospital /ID# 78908	Philadelphia	Pennsylvania
United States	Phoenix Children's Hospital /ID# 78906	Phoenix	Arizona
United States	Child Hosp of Pittsburgh,PA /ID# 79281	Pittsburgh	Pennsylvania
United States	Kaiser Permanente, NW /ID# 79236	Portland	Oregon
United States	MMP Women's Health /ID# 78894	Portland	Maine
United States	Oregon Health and Science University /ID# 78900	Portland	Oregon
United States	Rhode Island Hospital /ID# 79274	Providence	Rhode Island
United States	Children's Hospital of Richmon /ID# 78955	Richmond	Virginia
United States	Univ Rochester Med Ctr /ID# 92713	Rochester	New York
United States	UC Davis Comprehensive Cancer Center - Main /ID# 118935	Sacramento	California
United States	Cardinal Glennon Children's Ho /ID# 79275	Saint Louis	Missouri
United States	Children's Hospital St Louis /ID# 78910	Saint Louis	Missouri
United States	All Children's Hospital /ID# 79264	Saint Petersburg	Florida
United States	University of Utah /ID# 76794	Salt Lake City	Utah
United States	Univ Texas HSC San Antonio /ID# 79374	San Antonio	Texas
United States	California Pacific Medical Ctr /ID# 76775	San Francisco	California
United States	Univ California, San Francisco /ID# 78925	San Francisco	California
United States	Seattle Children's Hospital /ID# 78902	Seattle	Washington
United States	Louisiana State Univ HSC /ID# 78943	Shreveport	Louisiana
United States	Sanford Research/USD /ID# 78909	Sioux Falls	South Dakota
United States	Arthritis Northwest, PLLC /ID# 79257	Spokane	Washington
United States	Baystate Medical Center /ID# 103437	Springfield	Massachusetts
United States	Stanford University /ID# 95156	Stanford	California
United States	SUNY Upstate Medical University - Downtown /ID# 78912	Syracuse	New York
United States	ProMedica Toledo Hospital /ID# 78913	Toledo	Ohio
United States	University of Arizona Cancer Center - North Campus /ID# 78939	Tucson	Arizona
United States	The Univ Texas HSC at Tyler /ID# 79261	Tyler	Texas
United States	New York Medical College /ID# 78956	Valhalla	New York
United States	Children's National Medical Center /ID# 79373	Washington	District of Columbia
United States	Via Christi Specialty Clinics /ID# 78946	Wichita	Kansas
United States	Nemours/Alfred duPont Hospital for Children /ID# 78945	Wilmington	Delaware
United States	UMass Memorial Medical Center /ID# 78954	Worcester	Massachusetts

Sponsors (5)

Lead Sponsor	Collaborator
AbbVie	Cystic Fibrosis Foundation, Digestive Care, Inc., Nestle Health Science, VIVUS, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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	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

External Links

•   clinical study report synopsis