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Cystic Fibrosis (CF) Leukocyte Genes as Biomarkers for Novel Therapies

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by:
National Jewish Health
ClinicalTrials.gov Identifier:
NCT00727285
First received: July 28, 2008
Last updated: July 21, 2011
Last verified: July 2011
History of Changes
  Purpose

Presently, effectiveness of treatments for CF lung disease is judged by improvement in lung function (FEV1). However, in CF patients, FEV1 can range from severely decreased to normal, and improvements may occur slowly. Thus, clinical trials require many patients over prolonged periods to evaluate medications. As the pace of drug development accelerates, it is no longer possible to test all of the promising candidate therapies using conventional study designs. A sensitive technique for assessing lung inflammation has been developed which uses the expression of genes located in circulating blood cells. Mononuclear cells pass repeatedly through the blood vessels of the lung, and are exposed to many of the inflammatory products that are present in the airways. Over the past 4 years the investigators have identified a small group of candidate genes that are unregulated or downregulated in response to antibiotic treatment. The investigators now propose to prospectively test this method of quantifying lung inflammation in a large group of CF patients undergoing treatment of pulmonary exacerbations. Blood will be sampled before and after antibiotic treatment for a pulmonary exacerbation, and the relative change in gene expression will be compared to improvement in FEV1 and other clinical responses, to determine the utility of this method for use in studies. If successful, this technique could allow for a rapid and noninvasive method to gauge immediate effects by new treatments, and assist caregivers in determining optimal treatment strategies for the individual.


Condition
Cystic Fibrosis
Pulmonary Exacerbation

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: CF Leukocyte Genes as Biomarkers for Novel Therapies

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by National Jewish Health:

Primary Outcome Measures:
  • The Primary analysis is the change in expression of individual and combinations of mononuclear cell genes, obtained pre- and post-antibiotic therapy. [ Time Frame: 14-21 days ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Correlation of changes in PBMC gene expression with changes in FEV1 [ Time Frame: 14-21 days ] [ Designated as safety issue: No ]
  • Correlation of changes in PBMC gene expression with changes in WBC counts. [ Time Frame: 14-21 days ] [ Designated as safety issue: No ]

Estimated Enrollment: 60
Study Start Date: February 2008
Estimated Study Completion Date: October 2011
Primary Completion Date: October 2010 (Final data collection date for primary outcome measure)
Groups/Cohorts
A
CF patients followed by the Adult CF Program at National Jewish Health meeting criteria for an acute pulmonary exacerbation.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

CF patients followed by the Adult CF Program at National Jewish Health, who are ≥ 18 years and present with signs and symptoms of an acute pulmonary exacerbation. All subjects will be treated with at least two pathogen specific I.V. antibiotics for a minimum of 10 days, along with standard guidelines for other aspects of care for an acute pulmonary exacerbation. Concomitant use of inhaled antibiotics and systemic steroids will be allowed, and typical co-infections or co-morbidities will not result in exclusion.

Criteria

Inclusion Criteria:

  1. Documented diagnosis of CF.
  2. Age 18 years old or greater.
  3. Presentation at the start of treatment for a pulmonary exacerbation of CF.
  4. Ability to perform reproducible Pulmonary Function Tests.
  5. Willingness to comply with study procedure and willingness to provide written consent.

Exclusion Criteria:

  1. Participation in an investigational drug study within one month of enrollment.
  2. Presence of a condition or abnormality that, in the opinion of the Principal Investigator (PI), would compromise the safety of the patient or the quality of the data.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00727285

Locations
United States, Colorado
National Jewish Health
Denver, Colorado, United States, 80206
Sponsors and Collaborators
National Jewish Health
Cystic Fibrosis Foundation
Investigators
Principal Investigator: Jerry A Nick, M.D. National Jewish Health
  More Information

No publications provided

Responsible Party: Jerry A. Nick/Principal Investigator, National Jewish Health
ClinicalTrials.gov Identifier: NCT00727285     History of Changes
Other Study ID Numbers: NICK07A0
Study First Received: July 28, 2008
Last Updated: July 21, 2011
Health Authority: United States: Institutional Review Board

Keywords provided by National Jewish Health:
Cystic Fibrosis
pulmonary exacerbation
gene expression
 biomarker
inflammation
CF pulmonary exacerbation

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
 Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on May 23, 2013