Cystic FibrosisNCT00381628
Nasal Epithelial Cells/Blood Lymphocyte Markers for Cystic Fibrosis (CF)/CF Pulmonary Exacerbations
An observational study of Cystic Fibrosis, run by University Hospitals Cleveland Medical Center at 1 site in United States.
Trial summary
Study Hypothesis: We hypothesize that cellular markers from nasal epithelial cells and blood lymphocytes can serve as potential biomarkers reflect the underlying inflammatory state of the lung and will be helpful in determining the presence of a CF pulmonary exacerbation and its overall severity. Read more about this study
- Who is it for?
- Ages 15 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- epithelial cells and blood lymphocyte extraction
- Type of treatment
- Surgery or procedure
- How many people
- 59 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2006to Sep 2011
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 15 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Nasal epithelial cells and blood will be obtained from four groups of subjects age 15 years and older: Stable subjects with CF at baseline, Subjects with CF at the beginning and end of treatment for a pulmonary exacerbation, stable subjects with asthma, and healthy voulnteers
Who may be able to join
- Male or female >= 15 years of age
- Confirmed diagnosis of CF
- Clinically stable with no evidence of acute upper respiratory tract infection or current pulmonary exacerbation within the previous month
- Ability to understand and sign a written informed consent and comply with the requirements of the study
- Male or female >= 15 years of age
- Physician diagnosed asthma
Show all 11Show fewer
- Clinically stable with no evidence of acute upper or lower respiratory tract infection or current pulmonary exacerbation within the previous month
- Male or female >= 18 years of age
- Free of any chronic medical condition
- Clinically stable with no evidence of acute upper or lower respiratory tract infection within the previous month
- Ability to understand and sign a written informed consent and comply with the requirements of the study
Who cannot join
- Chronic use of a medication with anti-neutrophil or anti-inflammatory effect (ibuprofen, systemic or inhaled corticosteroids, or other immunosuppressive agents, etc
- Oxygen saturation <92% on room air
- Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the subject or the quality of the data
- CF patients with pulmonary exacerbations:
- Male of female >= 15 years of age Confirmed diagnosis of CF
- Patient meets a modified definition for a pulmonary exacerbation based upon Fuchs criteria which is treated with intravenous antibiotics for any 4 of the following 12 signs or symptoms:
Show all 31Show fewer
- Increased sputum production
- New or increased coughing up of blood
- Increased cough
- Increased dyspnea with exertion
- Malaise, fatigue or lethargy
- Anorexia or weight loss
- Fever
- Sinus pain or tenderness
- Changes in sinus discharge
- New findings on chest examination
- Decline in forced expiratory volume in 1 second (FEV1) > 10% since previous visit
- Radiographic changes indicative of pulmonary infection
- Ability to understand and sign a written informed consent and comply with the requirements of the study
- Exclusion criteria for CF patients with pulmonary exacerbation:
- Concurrent use a medication with anti-neutrophil or anti-inflammatory effect within the previous 4 weeks
- Presence of a condition or abnormality that in the opinion of the investigator would compromise the safety of the subject or the quality of the data.
- Chronic use of a medication with anti-neutrophil or anti-inflammatory effect within the previous 4 weeks
- Treated for an asthma exacerbation with the previous 4 weeks
- Treated with oral corticosteroids within the previous 4 weeks
- Oxygen saturation <92% on room air
- Presence of a condition or abnormality that in the opinion of the investigator would compromise the safety of the subject or the quality of the data.
- Use of a medication with anti-neutrophil or anti-inflammatory effect within the previous 4 months
- Presence of any chronic medical condition
- Oxygen saturation <92% on room air
- Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the subject or the quality of the data
Trial locations and contacts
1 site in 1 country.
United States1 site
Rainbow Babies and Children's Hospital
Cleveland, Ohio, 44106
About this study
Official title: Use of Nasal Epithelial Cells and Blood Lymphocytes to Identify Markers for Cystic Fibrosis and Cystic Fibrosis Pulmonary Exacerbations
Study Hypothesis: We hypothesize that cellular markers from nasal epithelial cells and blood lymphocytes can serve as potential biomarkers reflect the underlying inflammatory state of the lung and will be helpful in determining the presence of a CF pulmonary exacerbation and its overall severity.
Read the detailed description
Cystic fibrosis (CF) is the most common lethal genetic disease in the US afflicting approximately 30,000 people. Chronic disease of the respiratory tract, which is responsible for early death, affects both the upper and lower airways.
We propose to utilize cells (blood lymphocytes and nasal epithelial cells) that are readily accessible and are known to express the cystic fibrosis transmembrane conductance regulator (CFTR) and therefore candidates to express markers of the downstream consequences of CFTR deficiency.
A marker that indicates the inflammatory state of the lung would be useful to identify infective/inflammatory exacerbations as opposed to worsening due to pulmonary vascular disease or simply upper airway infection. This marker might help to guide therapy for intensity and duration. Evidence in mice suggest that lymphocytes may be a driving force for inflammation in the CF lung, particularly during exacerbations, and also that human CF lymphocytes have dysfunctional production of cytokines.
Specific Aims:
To identify markers in nasal epithelial cells or blood lymphocytes that distinguish CF patients from those with functional CFTR (healthy volunteers and patients with asthma). If successful this could become a marker for CFTR correction by drugs or other systemic therapies.
To identify markers in blood lymphocytes that will identify inflammatory status (ie, distinguish an active exacerbation from return to clinical stability) in CF patients. This could become a marker for infectious exacerbations of CF airway disease.
- Conditions
- Cystic Fibrosis
- Being tested
- epithelial cells and blood lymphocyte extraction
- Keywords
- Anatomy, cells, epithelial cells, blood cells, cystic fibrosis, equipment and supplies, disposable equipment, reagent kits
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
epithelial cells and blood lymphocyte extractionProcedure or surgery
Nasal curettage will be performed from each nostril to obtain nasal epithelial cells. Venipuncture will be performed and up to 60-ml of blood will be obtained from which neutrophils will be isolated.
| Study group | Role | What they receive |
|---|---|---|
| Stable subjects with CF | These subjects will undergo epithelial cells and blood lymphocyte extraction. Nasal curettage will be performed to obtain nasal epithelial cells and blood will be obtained to isolate circulating lymphocytes one time from these subjects (age 15 years and above) when they are in their usual state of well-health. These cells will be studied in vitro. Receives: Procedure: epithelial cells and blood lymphocyte extraction | |
| Exacerbating subjects with CF | These subjects will undergo epithelial cells and blood lymphocyte extraction. Nasal curettage will be performed to obtain nasal epithelial cells and blood will be obtained to isolate circulating lymphocytes one time from these subjects (age 15 years and above) at the beginning and end of treatment for a pulmonary exacerbation. These cells will be studied in vitro. Receives: Procedure: epithelial cells and blood lymphocyte extraction | |
| Stable subjects with asthma | These subjects will undergo epithelial cells and blood lymphocyte extraction. Nasal curettage will be performed to obtain nasal epithelial cells and blood will be obtained to isolate circulating lymphocytes one time from these subjects (age 15 years and above) when they are in their usual state of well-health. These cells will be studied in vitro. This is the disease control group. Receives: Procedure: epithelial cells and blood lymphocyte extraction | |
| Healthy volunteers | These subjects will undergo epithelial cells and blood lymphocyte extraction. Nasal curettage will be performed to obtain nasal epithelial cells and blood will be obtained to isolate circulating lymphocytes one time from these subjects (age 15 years and above) when they are in their usual state of well-health. These cells will be studied in vitro. This is the control group Receives: Procedure: epithelial cells and blood lymphocyte extraction |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Number of Participants With Blood and Sputum Samples Collected
Blood and sputum samples for general science research collaborators
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2006 (estimated)
- Study startSep 2006
- Primary completionSep 2011
- Study completionSep 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University Hospitals Cleveland Medical CenterThe 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-control
- Time perspective
- Prospective
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospitals Cleveland Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Principal investigator, James F. Chmiel, University Hospitals Cleveland Medical Center
- Investigators
- James F Chmiel, MD, MPH, Principal investigator, University Hospitals Cleveland Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00381628
- Sponsor's protocol ID
- Protocol 08-06-23
- Status last verified
- Jan 2019
- First submitted
- 27 Sep 2006
- Last updated
- 15 Feb 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use of Nasal Epithelial Cells and Blood Lymphocytes to Identify Markers for Cystic Fibrosis and Cystic Fibrosis Pulmonary Exacerbations
- ClinicalTrials.gov ID
- NCT00381628
- Acronym / sponsor ID
- Protocol 08-06-23
- Conditions
- Cystic Fibrosis
- Keywords
- Anatomy, cells, epithelial cells, blood cells, cystic fibrosis, equipment and supplies, disposable equipment, reagent kits
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 59 participants (actual)
- Lead sponsor
- University Hospitals Cleveland Medical CenterUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- Sep 2006
- Primary completion
- Sep 2011 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 28 Sep 2006 (estimated)
- Results
- Posted 15 Feb 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study Hypothesis: We hypothesize that cellular markers from nasal epithelial cells and blood lymphocytes can serve as potential biomarkers reflect the underlying inflammatory state of the lung and will be helpful in determining the presence of a CF pulmonary exacerbation and its overall severity.
Detailed description
Cystic fibrosis (CF) is the most common lethal genetic disease in the US afflicting approximately 30,000 people. Chronic disease of the respiratory tract, which is responsible for early death, affects both the upper and lower airways.
We propose to utilize cells (blood lymphocytes and nasal epithelial cells) that are readily accessible and are known to express the cystic fibrosis transmembrane conductance regulator (CFTR) and therefore candidates to express markers of the downstream consequences of CFTR deficiency.
A marker that indicates the inflammatory state of the lung would be useful to identify infective/inflammatory exacerbations as opposed to worsening due to pulmonary vascular disease or simply upper airway infection. This marker might help to guide therapy for intensity and duration. Evidence in mice suggest that lymphocytes may be a driving force for inflammation in the CF lung, particularly during exacerbations, and also that human CF lymphocytes have dysfunctional production of cytokines.
Specific Aims:
To identify markers in nasal epithelial cells or blood lymphocytes that distinguish CF patients from those with functional CFTR (healthy volunteers and patients with asthma). If successful this could become a marker for CFTR correction by drugs or other systemic therapies.
To identify markers in blood lymphocytes that will identify inflammatory status (ie, distinguish an active exacerbation from return to clinical stability) in CF patients. This could become a marker for infectious exacerbations of CF airway disease.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Stable subjects with CF | – | Procedure: epithelial cells and blood lymphocyte extractionThese subjects will undergo epithelial cells and blood lymphocyte extraction. Nasal curettage will be performed to obtain nasal epithelial cells and blood will be obtained to isolate circulating lymphocytes one time from these subjects (age 15 years and above) when they are in their usual state of well-health. These cells will be studied in vitro. |
| Exacerbating subjects with CF | – | Procedure: epithelial cells and blood lymphocyte extractionThese subjects will undergo epithelial cells and blood lymphocyte extraction. Nasal curettage will be performed to obtain nasal epithelial cells and blood will be obtained to isolate circulating lymphocytes one time from these subjects (age 15 years and above) at the beginning and end of treatment for a pulmonary exacerbation. These cells will be studied in vitro. |
| Stable subjects with asthma | – | Procedure: epithelial cells and blood lymphocyte extractionThese subjects will undergo epithelial cells and blood lymphocyte extraction. Nasal curettage will be performed to obtain nasal epithelial cells and blood will be obtained to isolate circulating lymphocytes one time from these subjects (age 15 years and above) when they are in their usual state of well-health. These cells will be studied in vitro. This is the disease control group. |
| Healthy volunteers | – | Procedure: epithelial cells and blood lymphocyte extractionThese subjects will undergo epithelial cells and blood lymphocyte extraction. Nasal curettage will be performed to obtain nasal epithelial cells and blood will be obtained to isolate circulating lymphocytes one time from these subjects (age 15 years and above) when they are in their usual state of well-health. These cells will be studied in vitro. This is the control group |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Nasal epithelial cells and blood will be obtained from four groups of subjects age 15 years and older: Stable subjects with CF at baseline, Subjects with CF at the beginning and end of treatment for a pulmonary exacerbation, stable subjects with asthma, and healthy voulnteers
Key inclusion criteria (11 listed)
- Male or female >= 15 years of age
- Confirmed diagnosis of CF
- Clinically stable with no evidence of acute upper respiratory tract infection or current pulmonary exacerbation within the previous month
- Ability to understand and sign a written informed consent and comply with the requirements of the study
- Male or female >= 15 years of age
Key exclusion criteria (31 listed)
- Chronic use of a medication with anti-neutrophil or anti-inflammatory effect (ibuprofen, systemic or inhaled corticosteroids, or other immunosuppressive agents, etc
- Oxygen saturation <92% on room air
- Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the subject or the quality of the data
- Male of female >= 15 years of age Confirmed diagnosis of CF
- Patient meets a modified definition for a pulmonary exacerbation based upon Fuchs criteria which is treated with intravenous antibiotics for any 4 of the following 12 signs or symptoms:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| epithelial cells and blood lymphocyte extractionNasal curettage will be performed from each nostril to obtain nasal epithelial cells. Venipuncture will be performed and up to 60-ml of blood will be obtained from which neutrophils will be isolated. | Procedure or surgery | Exacerbating subjects with CF, Healthy volunteers, Stable subjects with CF, Stable subjects with asthma |
Primary outcome
The Number of Participants With Blood and Sputum Samples Collected
Time frame: Baseline
Blood and sputum samples for general science research collaborators
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Feb 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
James F Chmiel, MD, MPH principal investigator
University Hospitals Cleveland Medical Center
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 University Hospitals Cleveland Medical Center 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 11 inclusion and 31 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 15 Feb 2019. 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?
Yes. Results are posted in the registry (first posted 15 Feb 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Number of Participants With Blood and Sputum Samples Collected (time frame: Baseline). 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 15 Feb 2019.