Clinical trial NCT01626157: A Novel Assay to Quantify Treatment Response in Cystic Fibrosis (CF)
Official title: A Novel Assay to Quantify Treatment Response in CF
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis
Lead sponsor: National Jewish Health
Sponsor class: OTHER
Collaborators: name: Cystic Fibrosis Foundation
First posted: 2012-06-22
Enrollment: 122
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Adult CF patients \> 18 years of age undergoing treatment for acute pulmonary exacerbation

Brief 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.


Eligibility 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 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

Primary outcomes:
measure: Change in leukocyte associated protein expression by flow cytometry and by leukocyte specific ELISA in response to acute exacerbation therapy
timeFrame: 10-21 days
Secondary outcomes:
measure: Change in FEV1 in response to acute exacerbation therapy
timeFrame: 10-21 days
measure: Change in sputum IL-8 and neutrophil elastase in response to acute exacerbation therapy
timeFrame: 10-21 days
measure: Change in bacterial density in response to exacerbation therapy
timeFrame: 10-21 days
measure: Change in CRP in response to acute exacerbation therapy
timeFrame: 10-21 days
measure: Time to next exacerbation
timeFrame: up to 3 years
Other outcomes:
Locations:
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
Study officials:
affiliation: National Jewish Health
name: Milene Saavedra, MD
role: PRINCIPAL_INVESTIGATOR
References: