Natural History of Bronchiectasis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age5-100
SponsorNational Heart, Lung, and Blood Institute (NHLBI)
About this trial
Background:
* Bronchiectasis is a disease characterized by airways that are inflamed, abnormally dilated, and chronically infected. Individuals with bronchiectasis have a history of chronic and recurring respiratory infections. Depending on the underlying cause, these infections may involve the entire respiratory tract, resulting in sinus, ear, and lung disease. * Bronchiectasis continues to be a significant problem in developing countries and in specific groups of individuals, particularly in people who have cystic fibrosis. Although treatments are available or under development for bronchiectasis related to cystic fibrosis, many of the disease-specific treatments may not be effective for bronchiectasis not related to cystic fibrosis.
Objectives:
\- To study the natural history of bronchiectasis to identify inherited and immune factors that may explain why certain individuals have chronic recurring infections.
Eligibility:
* Individuals 5 years of age and older who have an established diagnosis of bronchiectasis or a history of chronic/recurring respiratory infections. * Direct family members (e.g., parents, siblings, children) of patients in the study may also be asked to participate.
Design:
* Potential participants will be screened with an initial clinic evaluation and full medical history, as well as a general quality of life and respiratory symptom questionnaire. * The following standard procedures may be done as part of the study: air sampling from the nose; imaging studies, which may include an x-ray or computed tomography (CT), lung function tests; and collection of samples of blood, urine, and sputum (phlegm or mucus). Other tests may be performed as required by the researchers, and will be explained to patients as needed. * Both patients and relatives (if asked to participate) will provide the following samples: blood or buccal (cheek swab) cells for genetic testing, sputum, and urine. * To prevent infections and potential disease progression, patients may receive standard medical care and treatment for bronchiectasis and related infections during this protocol.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
PARTICIPANT
Children age 5 years and above and adults referred to the NIH with chronic or recurring respiratory infections will be eligible for participation.
Males and females will be considered without regard to race or ethnicity or upper age limit.
Emphasis will be primarily on non CF bronchiectasis for elucidation of mechanisms of infection susceptibility. However, select patients with cystic fibrosis or acquired immune defects (such as HIV) may be studied if relevant host defects are suspected, if needed for comparison purposes, or to evaluate and study the management of chronic pathogen associated conditions such as nontuberculous mycobacterial infections or allergic bronchopulmonary aspergillosis.
Disqualifiers
We wish to avoid enrolling subjects, especially children, who have common respiratory problems (aeroallergen sensitivity, asthma, gastric esophageal reflux) that are not associated with an underlying abnormality in host defenses. Evidence of significance of chronic or recurring infections suggestive of an underlying airway surface or systemic host defense defect should be documented by one or more of the following: a) sinus or lower airway cultures positive for bacterial, fungal, or mycobacterial pathogens characteristic of these defects; or b) radiographic evidence of sinusitis with mucosal thickening and/or air-fluid levels; or c) radiographic evidence of bronchiectasis; d) severity of otitis media requiring placement of tympanic membrane pressure equalization tubes; e) severity of sinusitis requiring surgical intervention.
Patients who are unable or unwilling to provide informed consent either directly or via appropriately designated surrogate.
Any patient who, in the opinion of the Investigator, is unable or unwilling to comply with regular follow-up or is unlikely to provide pertinent information regarding disease progression or response to treatment may be excluded from longitudinal follow-up.
Trial population
Children age 5 years and above and adults with chronic or recurring respiratory infections including pulmonary nontuberculous mycobacterial disease
Trial design
Design model
Other
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
900 Participants
are grouped into 2 trial groupsGroup A: 1
Group B: 2
Trial outcomes
Primary outcomes
1
Identify causes and/or underlying conditions associated with bronchiectasis
Identify causes and/or underlying conditions associated with bronchiectasis.
Time frame
Patients will be seen at least every 6 months for the first 2 years, and then at 1-5 year intervals to follow the course of their disease for 5 years or until they no longer wish to return.Secondary outcomes
1
Monitor a cohort of bronchiectasis patients to better understand factors associated with progression of disease and to assess outcomes of management and therapeutic strategies.
1)Assessment of genetic, systemic immune, and/or epithelial surface defense mechanisms: a) Collection and storage of blood and sputum b) Use and storage of other relevant biologic specimens 2) Assessment: a) St. George s Respiratory Questionnaire b) Pulmonary Symptom Severity Score c) Medical Research Council Dyspnea Scale d) Six-minute walk e) Computerized tomography score f) Spirometry: g) Lower airway microbiology: Gram s stain/bacterial cultures, modified AFB smear/Nocardia cultures, wet mount/fungal cultures, and AFBsmears/mycobacterial cultures as well as novel biomarkers ofmycobacteria,antimicrobial resistance and microbial changes on theprogression of bronchiectasis. h) Inflammatory markers: sedimentation rate, C-reactive protein, beta-2 microglobulin i) Screen adult patients for anxiety and depression and correlate with standardized measures of respiratory symptoms, disease severity, and quality of life.
Time frame
Patients will be seen at least every 6 months for the first 2 years, and then at 1-5 year intervals to follow the course of their disease for 5 years or until they no longer wish to return.2
Define mechanisms and pathophysiology for the development and progression of bronchiectasis
Genetic, systemic immune, and/or epithelial surface defense mechanisms involved in airway infection susceptibility and/or development of bronchiectasis through the
Time frame
Patients will be seen at least every 6 months for the first 2 years, and then at 1-5 year intervals to follow the course of their disease for 5 years or until they no longer wish to return.Sponsors and contacts
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Source ClinicalTrials.gov