Cystic Fibrosis Foundation (CFF) Biomarkers of Exacerbation

Completed

• Conditions: Cystic Fibrosis

• Registration Number: NCT00788359
• Lead Sponsor: University of Colorado, Denver

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2007-12
• Study First Submitted: 2008-11-06
• Study First Submitted QC: 2008-11-06
• Study First Posted: 2008-11-10
• Primary Completion: 2012-07
• Study Completion: 2014-07
• Status Verified: 2014-12
• Last Update Submitted: 2014-12-01
• Last Update Posted: 2014-12-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 123

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

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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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change in concentration of individual protein biomarkers and various combinations of biomarkers in blood samples obtained pre and post IV antibiotic therapy	up to 21 days

Secondary Outcome Measures

Name	Time	Method
Changes in serum white blood cell and absolute neutrophil counts obtained pre and post IV antibiotic therapy	Up to 21 days
Changes in pulmonary function (particularly FEV1) measured by spirometry pre and post IV antibiotic therapy	Up to 21 days
Changes in bacterial densities (P. aeruginosa and other CF pathogens) in sputum samples obtained pre and post IV antibiotic therapy	Up to 21 days

Trial Locations

Locations (5)

Riley Hospital for Children; 🇺🇸 Indianapolis, Indiana, United States

National Jewish Medical and Research Center; 🇺🇸 Denver, Colorado, United States

Case Western Reserve University; 🇺🇸 Cleveland, Ohio, United States

University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States