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CompletedNCT00381628Updated Feb 15, 2019Results posted

Nasal Epithelial Cells/Blood Lymphocyte Markers for Cystic Fibrosis (CF)/CF Pulmonary Exacerbations

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 type
Observational
Model
Case-control
Time perspective
Prospective
Enrollment
59
Ages
15 Years and older
Sex
All
01

Study 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 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.

02

Conditions studied

  • Cystic Fibrosis

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Keywords

  • Anatomy
  • cells
  • epithelial cells
  • blood cells
  • cystic fibrosis
  • equipment and supplies
  • disposable equipment
  • reagent kits
03

In context

Cystic Fibrosis

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 →

Lead sponsor

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.

04

Who can participate

Ages eligible
15 Years and older
Sexes eligible
All
Accepts healthy volunteers
Yes
Sampling method
Non-probability sample

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

Eligibility criteria

Stable CF Patients:

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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:

  • 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.

Inclusion Criteria - Asthma patients

  • Male or female >= 15 years of age
  • Physician diagnosed asthma
  • Clinically stable with no evidence of acute upper or lower respiratory tract infection or current pulmonary exacerbation within the previous month

Exclusion Criteria - Asthma patients

  • 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.

Inclusion Criteria for Healthy Volunteers

  • 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

Exclusion Criteria for Healthy Volunteers

  • 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
05

Study design

Observational model
Case-control
Time perspective
Prospective
Enrollment
59 participants (actual)
Patient registry
No

Groups and cohorts

  • 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.

    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.

    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.

    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

    Procedure: epithelial cells and blood lymphocyte extraction

Interventions

  • Procedureepithelial 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.

06

What researchers measure

Primary outcomes

  1. The Number of Participants With Blood and Sputum Samples Collected

    Blood and sputum samples for general science research collaborators

    Time frame: Baseline

07

Results

Posted Feb 15, 2017

Participant flow

Participant flow — Overall Study
MilestoneStable Subjects With CFExacerbating Subjects With CFStable Subjects With AsthmaHealthy Volunteers
Started1827014
Completed1827014
Not completed0000

Outcome measures

PrimaryThe Number of Participants With Blood and Sputum Samples Collected

Blood and sputum samples for general science research collaborators

Time frame:
Baseline
Reported as:
Count of participants · Participants
The Number of Participants With Blood and Sputum Samples Collected
ParticipantsStable Subjects With CFExacerbating Subjects With CFStable Subjects With AsthmaHealthy Volunteers
The Number of Participants With Blood and Sputum Samples Collected1827014

Adverse events

Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
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%)
Most frequent other events
Most frequent other events
EventStable Subjects With CFExacerbating Subjects With CFStable Subjects With AsthmaHealthy Volunteers
EpistaxisRespiratory, thoracic and mediastinal disorders1/180/27—0/14

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Stable Subjects With CFExacerbating Subjects With CFStable Subjects With AsthmaHealthy VolunteersTotal
<=18 years22—04
Between 18 and 65 years1525—1454
>=65 years10—01
Sex: Female, Male
Sex: Female, Male(Participants)Stable Subjects With CFExacerbating Subjects With CFStable Subjects With AsthmaHealthy VolunteersTotal
Female410—923
Male1417—536
Region of Enrollment
Region of Enrollment(Participants)Stable Subjects With CFExacerbating Subjects With CFStable Subjects With AsthmaHealthy VolunteersTotal
United States182701459
08

Study locations

1 site
  • Rainbow Babies and Children's Hospital
    Cleveland, Ohio 44106, United States
09

Timeline

Tracking since Sep 25, 2026
Not marked actual or estimatedDates not marked actual or estimated

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.

How these figures are worked out, and which studies are left out of them.

Registered
Sep 27, 2006
First posted
Sep 28, 2006
Start
Sep 2006
Primary completionActual
Sep 2011
CompletionActual
Sep 2011
Results first posted
Feb 15, 2017
Last update
Feb 15, 2019
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Feb 15, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT00381628
Collaborators
Cystic Fibrosis Foundation
Responsible party
James F. Chmiel (Associate Professor of Pediatrics, University Hospitals Cleveland Medical Center) — Principal investigator
First posted
Sep 28, 2006
Start date
Sep 2006
Primary completion
Sep 2011
Completion
Sep 2011
Results posted
Feb 15, 2017
Last update
Feb 15, 2019

Study contacts

James F Chmiel, MD, MPH
principal investigator · University Hospitals Cleveland Medical Center

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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