Are you healthy and looking to help advance medical science?

You may be eligible to participate in a autoimmune disease clinical study, and could be compensated for your time.

Are you healthy and looking to help advance medical science? You may be eligible to participate in a autoimmune disease clinical study, and could be compensated for your time.

What is a clinical trial? Is participating in a clinical trial right for you? Learn more

Autoimmune Disease Clinical Trial in Bethesda MD

NCT00943514 | Observational

Are you healthy and looking to help advance medical science?

You may be eligible to participate in a autoimmune disease clinical study, and could be compensated for your time.

Are you healthy and looking to help advance medical science? You may be eligible to participate in a autoimmune disease clinical study, and could be compensated for your time.

Recruiting

Male & Female

5 Years +

This study is looking to recruit 900 Participants

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.

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Details for the study

Population

Children age 5 years and above and adults with chronic or recurring respiratory infections including pulmonary nontuberculous mycobacterial disease

Brief Title

Natural History of Bronchiectasis

Official Title

Natural History of Bronchiectasis

Brief Summary

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.

Detailed Description

Bronchiectasis, or abnormal dilation of the airways, is a condition typically characterized
by chronic and recurring respiratory tract infections. Frequently, depending on the
underlying cause, these infections involve the entire respiratory tract resulting in sinus,
ear, and lung disease. This condition used to be more common in children prior to
immunizations for childhood infections. It continues to be a significant problem in
developing countries and in specific groups of individuals in the U.S. Cystic fibrosis (CF)
is the most commonly associated genetic condition and tremendous strides have been made in
recent years in understanding the mechanisms of this disease that are leading to a multitude
of emerging novel treatment strategies. The mechanisms of other causes for bronchiectasis
have not evolved to this degree, and many of the disease-specific treatments being assessed
for cystic fibrosis may not be effective for non-CF bronchiectasis. Often bronchiectasis can
be associated with chronic infections from environmental germs such as the nontuberculous
mycobacteria.

Outcome Measures

Outcome measures are the tests that investigators perform to prove whether or not a treatment being tested in a clinical trial is having any effect.

Primary

Identify causes and/or underlying conditions associated with bronchiectasis

Identify causes and/or underlying conditions associated with bronchiectasis.

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

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 acid-fast bacteria (AFB) smear/Nocardia cultures, wet mount/fungal cultures, and AFB smear/mycobacterial cultures 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.

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

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

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.

Study Criteria

PARTICIPANT INCLUSION CRITERIA:
1. Children age 5 years and above and adults referred to the NIH with chronic or
recurring respiratory infections will be eligible for participation.
2. Males and females will be considered without regard to race or ethnicity or upper
age limit.
3. 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 or 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.
4. Subjects must have a primary or referring physician
5. Subjects must be willing to have samples stored
INCLUSION CRITERIA FOR RELATIVES:
As a part of this protocol we may obtain blood, sputum, urine, or buccal swabs from some
blood relatives of patients on the study, with the hope of isolating and characterizing the
primary host defense defect(s) or genetic links responsible for airway infection
susceptibility and/or bronchiectasis seen within families. Male and female relatives will
be accepted without limitation due to age. These relatives may have pertinent
disease-related history obtained, but will neither receive treatment nor have any other
protocol procedures done unless they are enrolled on the study.
PARTICIPANT EXCLUSION CRITERIA:
1. 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.
2. Patients who are unable or unwilling to provide informed consent either directly or
via appropriately designated surrogate.
3. 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.
4. Children under the age of 5 will be excluded from this protocol due to the difficulty
of performing pertinent assays in infants and younger children, difficulty
distinguishing significance of respiratory infections which occur commonly in younger
children, and the lack of adequate facilities and equipment for management of children
younger than 2 years.