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

NCT01626157 | Completed

• 1 other identifier: SAAVED11A0
• Study Type: observational
• Target: 122
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Change in leukocyte associated protein expression by flow cytometry and by leukocyte specific ELISA in response to acute exacerbation therapy

  10-21 days

Secondary Outcomes (5)

• Change in FEV1 in response to acute exacerbation therapy

  10-21 days

• Change in sputum IL-8 and neutrophil elastase in response to acute exacerbation therapy

  10-21 days

• Change in bacterial density in response to exacerbation therapy

  10-21 days

• Change in CRP in response to acute exacerbation therapy

  10-21 days

• Time to next exacerbation

  up to 3 years

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Adult CF patients \> 18 years of age undergoing treatment for acute pulmonary exacerbation

You may qualify if:

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

You may not qualify if:

• 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.
• Use of systemic steroids

Sponsors & Collaborators

• Milene Saavedra: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (1)

National Jewish Health

Denver, Colorado, 80206, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Milene Saavedra, MD

  National Jewish Health

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: June 20, 2012
• First Posted: June 22, 2012
• Study Start: May 1, 2011
• Primary Completion: January 1, 2018
• Study Completion: January 1, 2018
• Last Updated: March 23, 2020

Record last verified: 2020-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)