Cystic Fibrosis Foundation (CFF) Biomarkers of Exacerbation

NCT00788359 | Completed

• 1 other identifier: 07-0366
• Study Type: observational
• Target: 123
• Locations: 1 country
• Sites: 5

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.

Conditions

Cystic Fibrosis

Keywords

cystic fibrosis; pulmonary exacerbation; biomarkers

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• 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

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Colorado, Denver: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (5)

National Jewish Medical and Research Center

Denver, Colorado, 80206, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Case Western Reserve University

Cleveland, Ohio, 44106, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Related Publications (1)

• Heltshe SL, Goss CH, Thompson V, Sagel SD, Sanders DB, Marshall BC, Flume PA. Short-term and long-term response to pulmonary exacerbation treatment in cystic fibrosis. Thorax. 2016 Mar;71(3):223-9. doi: 10.1136/thoraxjnl-2014-206750. Epub 2015 Apr 24.

  PMID: 25911223 DERIVED

Biospecimen

Retention: SAMPLES WITHOUT DNA

Blood (plasma)

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 6, 2008
• First Posted: November 10, 2008
• Study Start: December 1, 2007
• Primary Completion: July 1, 2012
• Study Completion: July 1, 2014
• Last Updated: December 2, 2014

Record last verified: 2014-12

Locations

US (5)