Cystic Fibrosis Foundation (CFF) Biomarkers of Exacerbation

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00788359
• Other study ID #: 07-0366
• Status: Completed
• Phase: N/A
• First received: November 6, 2008
• Last updated: December 1, 2014
• Start date: December 2007
• Est. completion date: July 2014

Study information

• Verified date: December 2014
• Source: University of Colorado, Denver
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 123
• Est. completion date: July 2014
• Est. primary completion date: July 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 10 Years and older

Eligibility

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

Study Design

Observational Model: Case-Only, Time Perspective: Prospective

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Locations

Country	Name	City	State
United States	University of Michigan	Ann Arbor	Michigan
United States	Case Western Reserve University	Cleveland	Ohio
United States	National Jewish Medical and Research Center	Denver	Colorado
United States	Riley Hospital for Children	Indianapolis	Indiana
United States	Vanderbilt University Medical Center	Nashville	Tennessee

Sponsors (2)

Lead Sponsor	Collaborator
University of Colorado, Denver	Cystic Fibrosis Foundation Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Secondary	Changes in pulmonary function (particularly FEV1) measured by spirometry pre and post IV antibiotic therapy		Up to 21 days	No
Secondary	Changes in bacterial densities (P. aeruginosa and other CF pathogens) in sputum samples obtained pre and post IV antibiotic therapy		Up to 21 days	No
Secondary	Changes in serum white blood cell and absolute neutrophil counts obtained pre and post IV antibiotic therapy		Up to 21 days	No