Cystic FibrosisNCT01626157
A Novel Assay to Quantify Treatment Response in Cystic Fibrosis (CF)
An observational study of Cystic Fibrosis, run by Milene Saavedra at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 122 participants
- Where
- 1 siteUnited States
- Study dates
- May 2011to Jan 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult CF patients > 18 years of age undergoing treatment for acute pulmonary exacerbation
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Novel Assay to Quantify Treatment Response in CF
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
- Medical subject headings
- Cystic Fibrosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in leukocyte associated protein expression by flow cytometry and by leukocyte specific ELISA in response to acute exacerbation therapy
Measured: 10-21 days
Secondary outcomes (5)
Change in FEV1 in response to acute exacerbation therapy
Measured: 10-21 days
Change in sputum IL-8 and neutrophil elastase in response to acute exacerbation therapy
Measured: 10-21 days
Change in bacterial density in response to exacerbation therapy
Measured: 10-21 days
Change in CRP in response to acute exacerbation therapy
Measured: 10-21 days
Time to next exacerbation
Measured: up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2012 (estimated)
- Study startMay 2011
- Primary completionJan 2018
- Study completionJan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 122 participants (actual)
Sponsor and investigators
- Lead sponsor
- Milene Saavedra
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor-investigator, Milene Saavedra, National Jewish Health
- Investigators
- Milene Saavedra, MD, Principal investigator, National Jewish Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01626157
- Sponsor's protocol ID
- SAAVED11A0
- Status last verified
- Mar 2020
- First submitted
- 20 Jun 2012
- Last updated
- 23 Mar 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Novel Assay to Quantify Treatment Response in CF
- ClinicalTrials.gov ID
- NCT01626157
- Acronym / sponsor ID
- SAAVED11A0
- Conditions
- Cystic Fibrosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 122 participants (actual)
- Lead sponsor
- Milene SaavedraUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- May 2011
- Primary completion
- Jan 2018 (actual)
- Study completion
- Jan 2018 (actual)
- First posted
- 22 Jun 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult CF patients > 18 years of age undergoing treatment for acute pulmonary exacerbation
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
5 secondary outcomes
Change in FEV1 in response to acute exacerbation therapy
Time frame: 10-21 days
Change in sputum IL-8 and neutrophil elastase in response to acute exacerbation therapy
Time frame: 10-21 days
Change in bacterial density in response to exacerbation therapy
Time frame: 10-21 days
Change in CRP in response to acute exacerbation therapy
Time frame: 10-21 days
Time to next exacerbation
Time frame: up to 3 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2020.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Milene Saavedra, MD principal investigator
National Jewish Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Milene Saavedra at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2020.