Cystic Fibrosis Foundation (CFF) Biomarkers of Exacerbation

Sponsor

University of Colorado, Denver (Other)

Overall Status

Completed

CT.gov ID

NCT00788359

Collaborator

Cystic Fibrosis Foundation (Other)

123

Enrollment

Locations

Duration (Months)

24.6

Patients Per Site

0.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis

Study Design

Study Type:

Observational

Actual Enrollment :

123 participants

Observational Model:

Case-Only

Time Perspective:

Prospective

Official Title:

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

Study Start Date :

Dec 1, 2007

Actual Primary Completion Date :

Jul 1, 2012

Actual Study Completion Date :

Jul 1, 2014

Outcome Measures

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 [up to 21 days]

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

10 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Locations

	Site	City	State	Country	Postal Code
1	National Jewish Medical and Research Center	Denver	Colorado	United States	80206
2	Riley Hospital for Children	Indianapolis	Indiana	United States	46202
3	University of Michigan	Ann Arbor	Michigan	United States	48109
4	Case Western Reserve University	Cleveland	Ohio	United States	44106
5	Vanderbilt University Medical Center	Nashville	Tennessee	United States	37232