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

Cystic Fibrosis Clinical Trial

Official title:

CF Leukocyte Genes as Biomarkers for Novel Therapies

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00727285
• Other study ID #: NICK07A0
• Status: Completed
• Phase: N/A
• First received: July 28, 2008
• Last updated: March 27, 2017
• Start date: February 2008
• Est. completion date: October 2012

Study information

• Verified date: March 2017
• Source: National Jewish Health
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 60
• Est. completion date: October 2012
• Est. primary completion date: October 2011
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis
• Pulmonary Exacerbation

Locations

Country	Name	City	State
United States	National Jewish Health	Denver	Colorado

Sponsors (2)

Lead Sponsor	Collaborator
National Jewish Health	Cystic Fibrosis Foundation Therapeutics

Country where clinical trial is conducted

United States, 

References & Publications (1)

Nick JA, Sanders LA, Ickes B, Briones NJ, Caceres SM, Malcolm KC, Brayshaw SJ, Chacon CS, Barboa CM, Jones MC, St Clair C, Taylor-Cousar JL, Nichols DP, Sagel SD, Strand M, Saavedra MT. Blood mRNA biomarkers for detection of treatment response in acute pu — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The Primary analysis is the change in expression of individual and combinations of mononuclear cell genes, obtained pre- and post-antibiotic therapy.		14-21 days
Secondary	Correlation of changes in PBMC gene expression with changes in FEV1		14-21 days
Secondary	Correlation of changes in PBMC gene expression with changes in WBC counts.		14-21 days