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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00727285
Recruitment Status : Completed
First Posted : August 1, 2008
Last Update Posted : March 29, 2017
Cystic Fibrosis Foundation Therapeutics
Information provided by (Responsible Party):
National Jewish Health

Brief Summary:
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 or disease
Cystic Fibrosis Pulmonary Exacerbation

Study Type : Observational
Actual Enrollment : 60 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: CF Leukocyte Genes as Biomarkers for Novel Therapies
Actual Study Start Date : February 2008
Actual Primary Completion Date : October 2011
Actual Study Completion Date : October 2012

CF patients followed by the Adult CF Program at National Jewish Health meeting criteria for an acute pulmonary exacerbation.

Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. Correlation of changes in PBMC gene expression with changes in FEV1 [ Time Frame: 14-21 days ]
  2. Correlation of changes in PBMC gene expression with changes in WBC counts. [ Time Frame: 14-21 days ]

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
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.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00727285

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

Publications of Results:
Responsible Party: National Jewish Health Identifier: NCT00727285     History of Changes
Other Study ID Numbers: NICK07A0
First Posted: August 1, 2008    Key Record Dates
Last Update Posted: March 29, 2017
Last Verified: March 2017

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

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