Long-term Study in US Cystic Fibrosis Patients Receiving Digestive Enzyme Supplements to Assess Any Narrowing of the Large Intestine Causing Adverse Intestinal Symptoms (Fibrosing Colonopathy)

This study is currently recruiting participants.
Verified February 2014 by AbbVie
Sponsor:
Collaborators:
Aptalis Pharma
Cystic Fibrosis Foundation
Janssen Research & Development, LLC
Information provided by (Responsible Party):
AbbVie ( AbbVie (prior sponsor, Abbott) )
ClinicalTrials.gov Identifier:
NCT01652157
First received: July 25, 2012
Last updated: February 13, 2014
Last verified: February 2014
  Purpose

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.


Condition Intervention
Fibrosing Colonopathy in Patients With Cystic Fibrosis
Biological: CREON
Biological: ZENPEP
Biological: ULTRESA
Biological: PANCREAZE
Biological: other non-sponsor pancreatic enzyme replacement therapy
Other: No pancreatic enzyme replacement therapy

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Long-term Study in US Cystic Fibrosis Patients Receiving Digestive Enzyme Supplements to Assess Any Narrowing of the Large Intestines Causing Adverse Intestinal Symptoms (Fibrosing Colonopathy)

Resource links provided by NLM:


Further study details as provided by AbbVie:

Primary Outcome Measures:
  • Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving CREON [ Time Frame: up to 10 years ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: up to 10 years ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: up to 10 years ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: up to 10 years ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: up to 10 years ] [ Designated as safety issue: Yes ]
    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 replacement therapy [ Time Frame: up to 10 years ] [ Designated as safety issue: Yes ]
    Fibrosing colonopathy is confirmed by an independent adjudication panel based on prospectively defined criteria.


Secondary Outcome Measures:
  • Odds ratio (95% confidence interval) to quantify the association between possible risk factors and confirmed fibrosing colonopathy [ Time Frame: up to 10 years ] [ Designated as safety issue: Yes ]
    Potential risk factors include demographics, medical history, and concurrent medications, including daily dosage and length of specific pancreatic enzyme replacement therapy.


Estimated Enrollment: 21960
Study Start Date: July 2012
Estimated Study Completion Date: July 2022
Estimated Primary Completion Date: July 2022 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
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: CREON
pancreatic enzyme replacement therapy
Other Name: pancrelipase
Biological: ZENPEP
pancreatic enzyme replacement therapy
Other Name: pancrelipase
Biological: ULTRESA
pancreatic enzyme replacement therapy
Other Name: pancrelipase
Biological: PANCREAZE
pancreatic enzyme replacement therapy
Other Name: pancrelipase
Biological: other non-sponsor pancreatic enzyme replacement therapy
pancreatic enzyme replacement therapy
Other Name: pancrelipase
Other: No pancreatic enzyme replacement therapy
Cystic fibrosis patients in the cystic fibrosis patient registry not receiving any pancreatic enzyme replacement therapy (approximately 10%)

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 patients participating in the registry from eligible sites, as well as new cystic fibrosis patients enrolled in the registry at these sites over a 2-year enrollment period, will serve as the base study population (estimated to include 21,960 cystic fibrosis patients). The enrollment period will start with the first patient encounter documented in the registry from any participating site. Cystic fibrosis patients in the base study population will be followed at their regular clinical care visits over the 10-year follow-up period. During this follow-up period, any patients presenting with signs and symptoms of suspected fibrosing colonopathy, based on a prospective definition, will be approached to obtain 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 cystic fibrosis 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.

This harmonized protocol reflects equal sponsorship not only by the registering Sponsor, AbbVie, but also the Collaborators, Aptalis Pharma and Janssen Research & Development, LLC.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

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

Criteria

Inclusion Criteria:

  • 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01652157

Contacts
Contact: Lynn G Allen 206-890-2710 lynngallen@allenclinicalresearch.com
Contact: Barbra L Fogarty, BA 206-884-7592 barbra.fogarty@seattlechildrens.org

  Show 125 Study Locations
Sponsors and Collaborators
AbbVie (prior sponsor, Abbott)
Aptalis Pharma
Cystic Fibrosis Foundation
Janssen Research & Development, LLC
Investigators
Study Director: Gerhard J Leitz Janssen Research & Development, LLC
Study Director: Pedro Quintana Diez, MD, PhD AbbVie
Study Director: Ruth Thieroff-Ekerdt, MD Aptalis Pharma
  More Information

Additional Information:
No publications provided

Responsible Party: AbbVie ( AbbVie (prior sponsor, Abbott) )
ClinicalTrials.gov Identifier: NCT01652157     History of Changes
Other Study ID Numbers: CFFC-OB-11
Study First Received: July 25, 2012
Last Updated: February 13, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by AbbVie:
prospective
pancreatic insufficiency
observational
fibrosing colonopathy
pancreatic enzyme replacement therapy

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes
Pancrelipase
Pancreatin
Gastrointestinal Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 15, 2014