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

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: NCT01626157
Recruitment Status : Active, not recruiting
First Posted : June 22, 2012
Last Update Posted : April 5, 2017
Cystic Fibrosis Foundation Therapeutics
Information provided by (Responsible Party):
Milene Saavedra, National Jewish Health

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.

Condition or disease
Cystic Fibrosis

Study Type : Observational
Actual Enrollment : 120 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Novel Assay to Quantify Treatment Response in CF
Study Start Date : May 2011
Estimated Primary Completion Date : January 2018
Estimated Study Completion Date : January 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Primary Outcome Measures :
  1. Change in leukocyte associated protein expression by flow cytometry and by leukocyte specific ELISA in response to acute exacerbation therapy [ Time Frame: 10-21 days ]

Secondary Outcome Measures :
  1. Change in FEV1 in response to acute exacerbation therapy [ Time Frame: 10-21 days ]
  2. Change in sputum IL-8 and neutrophil elastase in response to acute exacerbation therapy [ Time Frame: 10-21 days ]
  3. Change in bacterial density in response to exacerbation therapy [ Time Frame: 10-21 days ]
  4. Change in CRP in response to acute exacerbation therapy [ Time Frame: 10-21 days ]
  5. Time to next exacerbation [ Time Frame: up to 3 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Adult CF patients > 18 years of age undergoing treatment for acute pulmonary exacerbation

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

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): NCT01626157

United States, Colorado
National Jewish Health
Denver, Colorado, United States, 80206
Sponsors and Collaborators
Milene Saavedra
Cystic Fibrosis Foundation Therapeutics
Principal Investigator: Milene Saavedra, MD National Jewish Health

Responsible Party: Milene Saavedra, Assistant Professor, National Jewish Health Identifier: NCT01626157     History of Changes
Other Study ID Numbers: SAAVED11A0
First Posted: June 22, 2012    Key Record Dates
Last Update Posted: April 5, 2017
Last Verified: April 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

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