Use of Nasal Epithelial Cells and Blood Lymphocytes to Identify Markers for Cystic Fibrosis and Cystic Fibrosis Pulmonary Exacerbations
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 59
- 试验地点
- 1
- 主要终点
- The Number of Participants With Blood and Sputum Samples Collected
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 年龄范围
- 15 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •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
排除标准
- •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
- •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
结局指标
主要结局
The Number of Participants With Blood and Sputum Samples Collected
时间窗: Baseline
Blood and sputum samples for general science research collaborators
次要结局
未报告次要终点
研究者
James F. Chmiel
Associate Professor of Pediatrics
University Hospitals Cleveland Medical Center