Cystic FibrosisNCT00788359
Cystic Fibrosis Foundation (CFF) Biomarkers of Exacerbation
An observational study of Cystic Fibrosis, run by University of Colorado, Denver at 5 sites in United States.
Trial summary
Clinical and translational research in cystic fibrosis (CF) is hampered by a lack of biomarkers that can be used to identify promising new therapies. There is an urgent need for development and validation of biomarkers that more quickly predict the usefulness of potential drugs in CF and might prognosticate clinical… Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 123 participants
- Where
- 5 sitesUnited States
- Study dates
- Dec 2007to Jul 2014
- 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 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals with CF greater than or equal to 10 years of age who are being started on intravenous (IV) antibiotics for a clinically diagnosed pulmonary exacerbation. Patients must demonstrate at least 3 of the 11 criteria for pulmonary exacerbation as defined by a Cystic Fibrosis Foundation (CFF) Consensus Conference. Treatment with a minimum of two IV antibiotics is required. We expect to enroll an approximately equal number of males and females. Most CF patients in our clinics are of white, non-Hispanic origin and we anticipate this ethnic mix to persist in this study. The majority of pediatric CF subjects will be admitted to the hospital for treatment purposes whereas many adults receive their IV antibiotics at home.
Who may be able to join
- Diagnosis of CF as evidenced by a sweat chloride test >60mEq/L or by the presence of two known CF genetic mutations
- Male or female greater than or equal to 10 years of age
- Initiation of intravenous antibiotic therapy for a clinically diagnosed acute pulmonary exacerbation
- Ability to perform reproducible pulmonary function tests
- Willing to comply with the study procedures 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 quality of the data
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
University of Michigan
Ann Arbor, Michigan, 48109
Case Western Reserve University
Cleveland, Ohio, 44106
National Jewish Medical and Research Center
Denver, Colorado, 80206
Riley Hospital for Children
Indianapolis, Indiana, 46202
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
About this study
Official title: Multi-center Trial to Validate Protein Biomarkers of a Pulmonary Exacerbation in Cystic Fibrosis
Clinical and translational research in cystic fibrosis (CF) is hampered by a lack of biomarkers that can be used to identify promising new therapies. There is an urgent need for development and validation of biomarkers that more quickly predict the usefulness of potential drugs in CF and might prognosticate clinical course. In particular, combinations of protein biomarkers that can be obtained non-invasively offer great promise. The goal of this project is to determine whether protein biomarkers in blood can demonstrate a beneficial effect of treatment over two weeks. We intend to initially target an acute pulmonary exacerbation in CF because we know that subjects being treated with intravenous antibiotics and enhanced mucus clearance display clinical improvements within two weeks. We propose to prospectively collect blood samples from a large cohort of well-characterized CF persons serially during inpatient admissions for a pulmonary exacerbation and longitudinally during annual visits. Through this proposal, we hope to identify a CF lung injury biomarker panel that increases in the setting of an acute pulmonary exacerbation and improves rapidly following intravenous antibiotic therapy. Additionally, we will begin to explore whether this CF lung injury biomarker panel might also prognosticate clinical course including decline in pulmonary function. Finally, this study will serve as an important source of blood samples that will be banked for future biomarker and therapeutic studies designed to benefit the entire CF community.
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, pulmonary exacerbation, biomarkers
- Medical subject headings
- Cystic Fibrosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in concentration of individual protein biomarkers and various combinations of biomarkers in blood samples obtained pre and post IV antibiotic therapy
Measured: up to 21 days
Secondary outcomes (3)
Changes in pulmonary function (particularly FEV1) measured by spirometry pre and post IV antibiotic therapy
Measured: Up to 21 days
Changes in bacterial densities (P. aeruginosa and other CF pathogens) in sputum samples obtained pre and post IV antibiotic therapy
Measured: Up to 21 days
Changes in serum white blood cell and absolute neutrophil counts obtained pre and post IV antibiotic therapy
Measured: Up to 21 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Nov 2008 (estimated)
- Study startDec 2007
- Primary completionJul 2012
- Study completionJul 2014
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.
- More trials run by University of Colorado, DenverThe sponsor's other registered studies.
- 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 123 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Colorado, Denver
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00788359
- Sponsor's protocol ID
- 07-0366
- Status last verified
- Dec 2014
- First submitted
- 6 Nov 2008
- Last updated
- 2 Dec 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
Heltshe SL, Goss CH, Thompson V, Sagel SD, Sanders DB, Marshall BC, Flume PA. Short-term and long-term response to pulmonary exacerbation treatment in cystic fibrosis. Thorax. 2016 Mar;71(3):223-9. doi: 10.1136/thoraxjnl-2014-206750. Epub 2015 Apr 24.
PubMed 25911223
Study at a glance
From the registry record, last updated by the sponsor on 2 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-center Trial to Validate Protein Biomarkers of a Pulmonary Exacerbation in Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00788359
- Acronym / sponsor ID
- 07-0366
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, pulmonary exacerbation, biomarkers
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 123 participants (actual)
- Lead sponsor
- University of Colorado, DenverUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- Dec 2007
- Primary completion
- Jul 2012 (actual)
- Study completion
- Jul 2014 (actual)
- First posted
- 10 Nov 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.
Clinical and translational research in cystic fibrosis (CF) is hampered by a lack of biomarkers that can be used to identify promising new therapies. There is an urgent need for development and validation of biomarkers that more quickly predict the usefulness of potential drugs in CF and might prognosticate clinical course. In particular, combinations of protein biomarkers that can be obtained non-invasively offer great promise. The goal of this project is to determine whether protein biomarkers in blood can demonstrate a beneficial effect of treatment over two weeks. We intend to initially target an acute pulmonary exacerbation in CF because we know that subjects being treated with intravenous antibiotics and enhanced mucus clearance display clinical improvements within two weeks. We propose to prospectively collect blood samples from a large cohort of well-characterized CF persons serially during inpatient admissions for a pulmonary exacerbation and longitudinally during annual visits. Through this proposal, we hope to identify a CF lung injury biomarker panel that increases in the setting of an acute pulmonary exacerbation and improves rapidly following intravenous antibiotic therapy. Additionally, we will begin to explore whether this CF lung injury biomarker panel might also prognosticate clinical course including decline in pulmonary function. Finally, this study will serve as an important source of blood samples that will be banked for future biomarker and therapeutic studies designed to benefit the entire CF community.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals with CF greater than or equal to 10 years of age who are being started on intravenous (IV) antibiotics for a clinically diagnosed pulmonary exacerbation. Patients must demonstrate at least 3 of the 11 criteria for pulmonary exacerbation as defined by a Cystic Fibrosis Foundation (CFF) Consensus Conference. Treatment with a minimum of two IV antibiotics is required. We expect to enroll an approximately equal number of males and females. Most CF patients in our clinics are of white, non-Hispanic origin and we anticipate this ethnic mix to persist in this study. The majority of pediatric CF subjects will be admitted to the hospital for treatment purposes whereas many adults receive their IV antibiotics at home.
Key inclusion criteria (5 listed)
- Diagnosis of CF as evidenced by a sweat chloride test >60mEq/L or by the presence of two known CF genetic mutations
- Male or female greater than or equal to 10 years of age
- Initiation of intravenous antibiotic therapy for a clinically diagnosed acute pulmonary exacerbation
- Ability to perform reproducible pulmonary function tests
- Willing to comply with the study procedures and willingness to provide written consent
Key exclusion criteria (1 listed)
- Presence of a condition or abnormality that, in the opinion of the Principal Investigator (PI), would compromise the safety of the patient or 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
Change in concentration of individual protein biomarkers and various combinations of biomarkers in blood samples obtained pre and post IV antibiotic therapy
Time frame: up to 21 days
3 secondary outcomes
Changes in pulmonary function (particularly FEV1) measured by spirometry pre and post IV antibiotic therapy
Time frame: Up to 21 days
Changes in bacterial densities (P. aeruginosa and other CF pathogens) in sputum samples obtained pre and post IV antibiotic therapy
Time frame: Up to 21 days
Changes in serum white blood cell and absolute neutrophil counts obtained pre and post IV antibiotic therapy
Time frame: Up to 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 2 Dec 2014.
- Derived from this study: PubMed 25911223Heltshe SL, Goss CH, Thompson V, Sagel SD, Sanders DB, Marshall BC, Flume PA. Short-term and long-term response to pulmonary exacerbation treatment in cystic fibrosis. Thorax. 2016 Mar;71(3):223-9. doi: 10.1136/thoraxjnl-2014-206750. Epub 2015 Apr 24.
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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.
The record lists no contact. Use the official record to reach the sponsor.
5 sites 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 University of Colorado, Denver at 5 sites 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 1 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 2 Dec 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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 concentration of individual protein biomarkers and various combinations of biomarkers in blood samples obtained pre and post IV antibiotic therapy (time frame: up to 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 NCT00788359, 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 2 Dec 2014.