Cystic Fibrosis Foundation (CFF) Biomarkers of Exacerbation

Study Overview
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Brief Summary
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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.

Official Title
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Multi-center Trial to Validate Protein Biomarkers of a Pulmonary Exacerbation in Cystic Fibrosis

Conditions
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Cystic Fibrosis

Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 10 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| Changes in serum white blood cell and absolute neutrophil counts obtained pre and post IV antibiotic therapy |  | Up to 21 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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cystic fibrosis, pulmonary exacerbation, biomarkers