Cystic FibrosisNCT00727285
Cystic Fibrosis (CF) Leukocyte Genes as Biomarkers for Novel Therapies
An observational study of Cystic Fibrosis and Pulmonary Exacerbation, run by National Jewish Health at 1 site in United States.
Trial summary
Presently, effectiveness of treatments for CF lung disease is judged by improvement in lung function (FEV1). However, in CF patients, FEV1 can range from severely decreased to normal, and improvements may occur slowly. Thus, clinical trials require many patients over prolonged periods to evaluate medications. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis, Pulmonary Exacerbation
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2008to Oct 2012
- 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: CF patients followed by the Adult CF Program at National Jewish Health, who are ≥ 18 years and present with signs and symptoms of an acute pulmonary exacerbation. All subjects will be treated with at least two pathogen specific I.V. antibiotics for a minimum of 10 days, along with standard guidelines for other aspects of care for an acute pulmonary exacerbation. Concomitant use of inhaled antibiotics and systemic steroids will be allowed, and typical co-infections or co-morbidities will not result in exclusion.
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.
- Willingness to comply with study procedure and willingness to provide written consent.
Who cannot join
- Participation in an investigational drug study within one month of enrollment.
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: CF Leukocyte Genes as Biomarkers for Novel Therapies
Presently, effectiveness of treatments for CF lung disease is judged by improvement in lung function (FEV1). However, in CF patients, FEV1 can range from severely decreased to normal, and improvements may occur slowly. Thus, clinical trials require many patients over prolonged periods to evaluate medications. As the pace of drug development accelerates, it is no longer possible to test all of the promising candidate therapies using conventional study designs. A sensitive technique for assessing lung inflammation has been developed which uses the expression of genes located in circulating blood cells. Mononuclear cells pass repeatedly through the blood vessels of the lung, and are exposed to many of the inflammatory products that are present in the airways. Over the past 4 years the investigators have identified a small group of candidate genes that are unregulated or downregulated in response to antibiotic treatment. The investigators now propose to prospectively test this method of quantifying lung inflammation in a large group of CF patients undergoing treatment of pulmonary exacerbations. Blood will be sampled before and after antibiotic treatment for a pulmonary exacerbation, and the relative change in gene expression will be compared to improvement in FEV1 and other clinical responses, to determine the utility of this method for use in studies. If successful, this technique could allow for a rapid and noninvasive method to gauge immediate effects by new treatments, and assist caregivers in determining optimal treatment strategies for the individual.
- Conditions
- Cystic Fibrosis, Pulmonary Exacerbation
- Keywords
- Cystic Fibrosis, pulmonary exacerbation, gene expression, biomarker, inflammation, CF pulmonary exacerbation
- Medical subject headings
- Cystic Fibrosis, Inflammation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| A | CF patients followed by the Adult CF Program at National Jewish Health meeting criteria for an acute pulmonary exacerbation. |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Primary analysis is the change in expression of individual and combinations of mononuclear cell genes, obtained pre- and post-antibiotic therapy.
Measured: 14-21 days
Secondary outcomes (2)
Correlation of changes in PBMC gene expression with changes in FEV1
Measured: 14-21 days
Correlation of changes in PBMC gene expression with changes in WBC counts.
Measured: 14-21 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2008 (estimated)
- Study startFeb 2008
- Primary completionOct 2011
- Study completionOct 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Jewish Health
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
- Investigators
- Jerry A Nick, M.D., Principal investigator, National Jewish Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00727285
- Sponsor's protocol ID
- NICK07A0
- Status last verified
- Mar 2017
- First submitted
- 28 Jul 2008
- Last updated
- 29 Mar 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Nick JA, Sanders LA, Ickes B, Briones NJ, Caceres SM, Malcolm KC, Brayshaw SJ, Chacon CS, Barboa CM, Jones MC, St Clair C, Taylor-Cousar JL, Nichols DP, Sagel SD, Strand M, Saavedra MT. Blood mRNA biomarkers for detection of treatment response in acute pulmonary exacerbations of cystic fibrosis. Thorax. 2013 Oct;68(10):929-37. doi: 10.1136/thoraxjnl-2012-202278. Epub 2013 Jun 19.
PubMed 23783371
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- CF Leukocyte Genes as Biomarkers for Novel Therapies
- ClinicalTrials.gov ID
- NCT00727285
- Acronym / sponsor ID
- NICK07A0
- Conditions
- Cystic Fibrosis, Pulmonary Exacerbation
- Keywords
- Cystic Fibrosis, pulmonary exacerbation, gene expression, biomarker, inflammation, CF pulmonary exacerbation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- National Jewish HealthUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- Feb 2008 (actual)
- Primary completion
- Oct 2011 (actual)
- Study completion
- Oct 2012 (actual)
- First posted
- 1 Aug 2008 (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.
Presently, effectiveness of treatments for CF lung disease is judged by improvement in lung function (FEV1). However, in CF patients, FEV1 can range from severely decreased to normal, and improvements may occur slowly. Thus, clinical trials require many patients over prolonged periods to evaluate medications. As the pace of drug development accelerates, it is no longer possible to test all of the promising candidate therapies using conventional study designs. A sensitive technique for assessing lung inflammation has been developed which uses the expression of genes located in circulating blood cells. Mononuclear cells pass repeatedly through the blood vessels of the lung, and are exposed to many of the inflammatory products that are present in the airways. Over the past 4 years the investigators have identified a small group of candidate genes that are unregulated or downregulated in response to antibiotic treatment. The investigators now propose to prospectively test this method of quantifying lung inflammation in a large group of CF patients undergoing treatment of pulmonary exacerbations. Blood will be sampled before and after antibiotic treatment for a pulmonary exacerbation, and the relative change in gene expression will be compared to improvement in FEV1 and other clinical responses, to determine the utility of this method for use in studies. If successful, this technique could allow for a rapid and noninvasive method to gauge immediate effects by new treatments, and assist caregivers in determining optimal treatment strategies for the individual.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | – | –CF patients followed by the Adult CF Program at National Jewish Health meeting criteria for an acute pulmonary exacerbation. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- CF patients followed by the Adult CF Program at National Jewish Health, who are ≥ 18 years and present with signs and symptoms of an acute pulmonary exacerbation. All subjects will be treated with at least two pathogen specific I.V. antibiotics for a minimum of 10 days, along with standard guidelines for other aspects of care for an acute pulmonary exacerbation. Concomitant use of inhaled antibiotics and systemic steroids will be allowed, and typical co-infections or co-morbidities will not result in exclusion.
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.
- Willingness to comply with study procedure and willingness to provide written consent.
Key exclusion criteria (2 listed)
- Participation in an investigational drug study within one month of enrollment.
- 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.
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
The Primary analysis is the change in expression of individual and combinations of mononuclear cell genes, obtained pre- and post-antibiotic therapy.
Time frame: 14-21 days
2 secondary outcomes
Correlation of changes in PBMC gene expression with changes in FEV1
Time frame: 14-21 days
Correlation of changes in PBMC gene expression with changes in WBC counts.
Time frame: 14-21 days
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 29 Mar 2017.
- Results publication: PubMed 23783371Nick JA, Sanders LA, Ickes B, Briones NJ, Caceres SM, Malcolm KC, Brayshaw SJ, Chacon CS, Barboa CM, Jones MC, St Clair C, Taylor-Cousar JL, Nichols DP, Sagel SD, Strand M, Saavedra MT. Blood mRNA biomarkers for detection of treatment response in acute pulmonary exacerbations of cystic fibrosis. Thorax. 2013 Oct;68(10):929-37. doi: 10.1136/thoraxjnl-2012-202278. Epub 2013 Jun 19.
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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.
Jerry A Nick, M.D. 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 and Pulmonary Exacerbation, run by National Jewish Health 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 29 Mar 2017. 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?
The Primary analysis is the change in expression of individual and combinations of mononuclear cell genes, obtained pre- and post-antibiotic therapy. (time frame: 14-21 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00727285, which also lists 1 linked publication.
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 29 Mar 2017; AzTrials last refreshed its copy on 8 Nov 2026.