A Novel Assay to Quantify Treatment Response in Cystic Fibrosis (CF)

Cystic Fibrosis Clinical Trial

Official title:

A Novel Assay to Quantify Treatment Response in CF

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01626157
• Other study ID #: SAAVED11A0
• Status: Completed
• Start date: May 2011
• Est. completion date: January 2018

Study information

• Verified date: March 2020
• Source: National Jewish Health
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Inflammation is present in the Cystic fibrosis (CF) airway from the time of infancy, and worsens with the onset of chronic infection. Therapies with proven benefit are associated with decreased airway inflammation. Thus, sensitive and reproducible biomarkers of airway inflammation have long been sought as a necessary component to improved clinical care and to facilitate therapeutic trials for CF. FEV1, the standard outcome measure in CF, is recognized as an insensitive outcome measure. the investigators have identified a panel of 10 genes which sensitively predict resolution of pulmonary inflammation, in response to therapy of an acute pulmonary exacerbation. With the goal of yielding a technically simple but unique CF biomarker assay, the investigators have tested whether proteins signified by these genes show large changes in expression following treatment of acute pulmonary exacerbations. Protein quantifications are among the most common measurements performed in clinical laboratories around the world. Based on preliminary findings that changes in white blood cell proteins mirror changes seen in the genes, the investigators propose to identify top candidate proteins, from the investigators gene panel, which change in response to exacerbation therapy. Once identified, these proteins will be quantified directly with a new blood test which is inexpensive and simple to perform. The investigators propose to validate this blood test in a single site trial. If successful, this proposal will yield a biomarker assay that will be available to validate in a multi-site trial and provide unique insights into mechanisms that regulate white blood cell activation and recruitment in CF lung disease.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 122
• Est. completion date: January 2018
• Est. primary completion date: January 2018
• 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 and produce sputum.

5. Willingness to comply with study procedure and willingness to provide written consent.

Exclusion Criteria:

1. 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.

2. Use of systemic steroids

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Locations

Country	Name	City	State
United States	National Jewish Health	Denver	Colorado

Sponsors (2)

Lead Sponsor	Collaborator
Milene Saavedra	Cystic Fibrosis Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in leukocyte associated protein expression by flow cytometry and by leukocyte specific ELISA in response to acute exacerbation therapy		10-21 days
Secondary	Change in FEV1 in response to acute exacerbation therapy		10-21 days
Secondary	Change in sputum IL-8 and neutrophil elastase in response to acute exacerbation therapy		10-21 days
Secondary	Change in bacterial density in response to exacerbation therapy		10-21 days
Secondary	Change in CRP in response to acute exacerbation therapy		10-21 days
Secondary	Time to next exacerbation		up to 3 years