Cystic Fibrosis Foundation (CFF) Biomarkers of Exacerbation

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
University of Colorado, Denver
ClinicalTrials.gov Identifier:
NCT00788359
First received: November 6, 2008
Last updated: December 11, 2013
Last verified: November 2012
  Purpose

Clinical and translational research in cystic fibrosis (CF) is hampered by a lack of biomarkers that can be used to identify promising new therapies. There is an urgent need for development and validation of biomarkers that more quickly predict the usefulness of potential drugs in CF and might prognosticate clinical course. In particular, combinations of protein biomarkers that can be obtained non-invasively offer great promise. The goal of this project is to determine whether protein biomarkers in blood can demonstrate a beneficial effect of treatment over two weeks. We intend to initially target an acute pulmonary exacerbation in CF because we know that subjects being treated with intravenous antibiotics and enhanced mucus clearance display clinical improvements within two weeks. We propose to prospectively collect blood samples from a large cohort of well-characterized CF persons serially during inpatient admissions for a pulmonary exacerbation and longitudinally during annual visits. Through this proposal, we hope to identify a CF lung injury biomarker panel that increases in the setting of an acute pulmonary exacerbation and improves rapidly following intravenous antibiotic therapy. Additionally, we will begin to explore whether this CF lung injury biomarker panel might also prognosticate clinical course including decline in pulmonary function. Finally, this study will serve as an important source of blood samples that will be banked for future biomarker and therapeutic studies designed to benefit the entire CF community.


Condition
Cystic Fibrosis

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Multi-center Trial to Validate Protein Biomarkers of a Pulmonary Exacerbation in Cystic Fibrosis

Resource links provided by NLM:


Further study details as provided by University of Colorado, Denver:

Primary Outcome Measures:
  • Change in concentration of individual protein biomarkers and various combinations of biomarkers in blood samples obtained pre and post IV antibiotic therapy [ Time Frame: up to 21 days ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Changes in pulmonary function (particularly FEV1) measured by spirometry pre and post IV antibiotic therapy [ Time Frame: Up to 21 days ] [ Designated as safety issue: No ]
  • Changes in bacterial densities (P. aeruginosa and other CF pathogens) in sputum samples obtained pre and post IV antibiotic therapy [ Time Frame: Up to 21 days ] [ Designated as safety issue: No ]
  • Changes in serum white blood cell and absolute neutrophil counts obtained pre and post IV antibiotic therapy [ Time Frame: Up to 21 days ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples Without DNA

Blood (plasma)


Estimated Enrollment: 130
Study Start Date: December 2007
Estimated Study Completion Date: June 2014
Primary Completion Date: July 2012 (Final data collection date for primary outcome measure)
  Eligibility

Ages Eligible for Study:   10 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Individuals with CF greater than or equal to 10 years of age who are being started on intravenous (IV) antibiotics for a clinically diagnosed pulmonary exacerbation. Patients must demonstrate at least 3 of the 11 criteria for pulmonary exacerbation as defined by a Cystic Fibrosis Foundation (CFF) Consensus Conference. Treatment with a minimum of two IV antibiotics is required. We expect to enroll an approximately equal number of males and females. Most CF patients in our clinics are of white, non-Hispanic origin and we anticipate this ethnic mix to persist in this study. The majority of pediatric CF subjects will be admitted to the hospital for treatment purposes whereas many adults receive their IV antibiotics at home.

Criteria

Inclusion Criteria:

  • Diagnosis of CF as evidenced by a sweat chloride test >60mEq/L or by the presence of two known CF genetic mutations
  • Male or female greater than or equal to 10 years of age
  • Initiation of intravenous antibiotic therapy for a clinically diagnosed acute pulmonary exacerbation
  • Ability to perform reproducible pulmonary function tests
  • Willing to comply with the study procedures and willingness to provide written consent

Exclusion Criteria:

  • Presence of a condition or abnormality that, in the opinion of the Principal Investigator (PI), would compromise the safety of the patient or quality of the data
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00788359

Locations
United States, Colorado
National Jewish Medical and Research Center
Denver, Colorado, United States, 80206
United States, Indiana
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, Ohio
Case Western Reserve University
Cleveland, Ohio, United States, 44106
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
Sponsors and Collaborators
University of Colorado, Denver
Cystic Fibrosis Foundation
  More Information

No publications provided

Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT00788359     History of Changes
Other Study ID Numbers: 07-0366
Study First Received: November 6, 2008
Last Updated: December 11, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by University of Colorado, Denver:
cystic fibrosis
pulmonary exacerbation
biomarkers

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

ClinicalTrials.gov processed this record on July 24, 2014