An observational study in Cystic Fibrosis, sponsored by University Hospitals Cleveland Medical Center. Completed at 1 site in United States. Open to participants aged 15 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2019-02-15.
Sponsored by University Hospitals Cleveland Medical Center · Observational
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.
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.
1,605 studies on the registry are indexed under Cystic Fibrosis; 196 are open to participants now.
This study's enrollment of 59 is below the median of 85 across 489 observational studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →University Hospitals Cleveland Medical Center is the lead sponsor of 293 studies on the registry; 43 are open to participants now.
Of its 35 completed or terminated interventional studies of FDA-regulated products, 30 (86%) have results posted.
Counted across the registry records on this site, refreshed daily.
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
Stable CF Patients:
Inclusion Criteria:
Exclusion Criteria:
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:
Exclusion criteria for CF patients with pulmonary exacerbation:
Inclusion Criteria - Asthma patients
Exclusion Criteria - Asthma patients
Inclusion Criteria for Healthy Volunteers
Exclusion Criteria for 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.
Procedure: epithelial cells and blood lymphocyte extraction
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.
Procedure: epithelial cells and blood lymphocyte extraction
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.
Procedure: epithelial cells and blood lymphocyte extraction
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
Procedure: epithelial cells and blood lymphocyte extraction
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.
The Number of Participants With Blood and Sputum Samples Collected
Blood and sputum samples for general science research collaborators
Time frame: Baseline
| Milestone | Stable Subjects With CF | Exacerbating Subjects With CF | Stable Subjects With Asthma | Healthy Volunteers |
|---|---|---|---|---|
| Started | 18 | 27 | 0 | 14 |
| Completed | 18 | 27 | 0 | 14 |
| Not completed | 0 | 0 | 0 | 0 |
Blood and sputum samples for general science research collaborators
| Participants | Stable Subjects With CF | Exacerbating Subjects With CF | Stable Subjects With Asthma | Healthy Volunteers |
|---|---|---|---|---|
| The Number of Participants With Blood and Sputum Samples Collected | 18 | 27 | 0 | 14 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Stable Subjects With CF | — | 0/18 (0%) | 1/18 (5.6%) |
| Exacerbating Subjects With CF | — | 0/27 (0%) | 0/27 (0%) |
| Stable Subjects With Asthma | — | — | — |
| Healthy Volunteers | — | 0/14 (0%) | 0/14 (0%) |
| Event | Stable Subjects With CF | Exacerbating Subjects With CF | Stable Subjects With Asthma | Healthy Volunteers |
|---|---|---|---|---|
| EpistaxisRespiratory, thoracic and mediastinal disorders | 1/18 | 0/27 | — | 0/14 |
| Age, Categorical(Participants) | Stable Subjects With CF | Exacerbating Subjects With CF | Stable Subjects With Asthma | Healthy Volunteers | Total |
|---|---|---|---|---|---|
| <=18 years | 2 | 2 | — | 0 | 4 |
| Between 18 and 65 years | 15 | 25 | — | 14 | 54 |
| >=65 years | 1 | 0 | — | 0 | 1 |
| Sex: Female, Male(Participants) | Stable Subjects With CF | Exacerbating Subjects With CF | Stable Subjects With Asthma | Healthy Volunteers | Total |
|---|---|---|---|---|---|
| Female | 4 | 10 | — | 9 | 23 |
| Male | 14 | 17 | — | 5 | 36 |
| Region of Enrollment(Participants) | Stable Subjects With CF | Exacerbating Subjects With CF | Stable Subjects With Asthma | Healthy Volunteers | Total |
|---|---|---|---|---|---|
| United States | 18 | 27 | 0 | 14 | 59 |
The record gives a start of Sep 2006, a primary completion of Sep 2011 and a completion of Sep 2011. It does not say whether these dates are actual or estimated.
Results were first posted in Feb 2017, 5 years 5 months after primary completion.
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This study is completed, as verified in Jan 2019. You cannot join it, but the record below documents what was studied.
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