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)
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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) |
- 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.
- 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 |
Cystic fibrosis patients who are active in the Cystic Fibrosis Patient Registry
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| Contact: Lynn G Allen | 206-890-2710 | lynngallen@allenclinicalresearch.com |
| Contact: Barbra L Fogarty, BA | 206-884-7592 | barbra.fogarty@seattlechildrens.org |
Show 101 Study Locations| Study Director: | Gerhard J Leitz | Janssen Research & Development, LLC |
| Study Director: | Dawn M Carlson, MD | 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: | June 10, 2013 |
| 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 June 18, 2013