CFF Biomarkers of Exacerbation - Full Text View

Sponsors and Collaborators:	University of Colorado at Denver and Health Sciences Center Cystic Fibrosis Foundation
Information provided by:	University of Colorado at Denver and Health Sciences Center
ClinicalTrials.gov Identifier:	NCT00788359

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

Genetics Home Reference related topics: cystic fibrosis

MedlinePlus related topics: Antibiotics Cystic Fibrosis

U.S. FDA Resources

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

Further study details as provided by University of Colorado at Denver and Health Sciences Center:

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

Biospecimen Description:

Blood (plasma)

Estimated Enrollment:	130
Study Start Date:	December 2007

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

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

Contacts

Contact: Scott D Sagel, MD	720-777-6181	sagel.scott@tchden.org
Contact: Meg M Anthony, BS	720-777-2945	anthony.meg@tchden.org

Locations

United States, Colorado
National Jewish Medical and Research Center	Recruiting
Denver, Colorado, United States, 80206
Principal Investigator: Milene Saavedra, MD
United States, Indiana
Riley Hospital for Children	Recruiting
Indianapolis, Indiana, United States, 46202
Principal Investigator: Greg Montgomery, MD
United States, Michigan
University of Michigan	Recruiting
Ann Arbor, Michigan, United States, 48109
Principal Investigator: Samya Nasr, MD
United States, Ohio
Case Western Reserve University	Recruiting
Cleveland, Ohio, United States, 44106
Principal Investigator: James Chmiel, MD
United States, Tennessee
Vanderbilt University Medical Center	Recruiting
Nashville, Tennessee, United States, 37232
Principal Investigator: Bonnie Slovis, MD

Sponsors and Collaborators

University of Colorado at Denver and Health Sciences Center

Cystic Fibrosis Foundation

More Information

Responsible Party:	University of Colorado Denver School of Medicine ( Scott Sagel, MD/Assistant Professor of Pediatrics )
Study ID Numbers:	#SAGEL07A0
Study First Received:	November 6, 2008
Last Updated:	November 6, 2008
ClinicalTrials.gov Identifier:	NCT00788359
Health Authority:	United States: Institutional Review Board

Keywords provided by University of Colorado at Denver and Health Sciences Center:

cystic fibrosis
pulmonary exacerbation
biomarkers

Study placed in the following topic categories:

Digestive System Diseases
Genetic Diseases, Inborn
Respiratory Tract Diseases
Cystic Fibrosis
Fibrosis

Lung Diseases
Infant, Newborn, Diseases
Pancreatic Diseases
Cystic fibrosis

Additional relevant MeSH terms:

Pathologic Processes

ClinicalTrials.gov processed this record on January 16, 2009