Natural History of Bronchiectasis

• ClinicalTrials.gov Identifier: NCT00943514
• Recruitment Status: Recruiting
• First Posted: July 22, 2009
• Last Update Posted: April 1, 2021
• Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Information provided by (Responsible Party): National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Tracking Information

• First Submitted Date: July 21, 2009
• First Posted Date: July 22, 2009
• Last Update Posted Date: April 1, 2021
• Actual Study Start Date: August 1, 2009
• Primary Completion Date: Not Provided

Current Primary Outcome Measures (submitted: November 1, 2018)

We will document the presence of underlying associated conditions such as a. Variant cystic fibrosis, b. Primary ciliary dyskinesia, c. Common variable immunodeficiency, d. Allergic bronchopulmonary mycosis, e. Autoimmune disease, Autoimmune dis... [ Time Frame: 2 yrs ]

Collection of clinically indicated, standardized outcome measures will facilitate assessment of ongoing management strategies.

• Original Primary Outcome Measures: Not Provided

Current Secondary Outcome Measures (submitted: September 17, 2020)

• Genetic, systemic immune, and/or epithelial surface defense mechanisms involved in airway infection susceptibility and/or development of bronchiectasis [ Time Frame: 2 yrs ]
• Assessment of clinical outcomes utilizing standardized measuresduring follow-up visits [ Time Frame: 2 yrs ]

• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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.
• Study Type: Observational
• Study Design: Observational Model: Other; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Children age 5 years and above and adults with chronic or recurring respiratory infections including pulmonary nontuberculous mycobacterial disease

Condition

• Bronchiectasis
• Cystic Fibrosis
• Autoimmune Disease
• Common Variable Immunodeficiency

• Intervention: Not Provided

Study Groups/Cohorts

• 1
  chronic or recurring respiratory infections including pulmonary nontuberculous mycobacterial disease
• 2
  Relatives

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: October 9, 2020): 900
• Original Enrollment (submitted: July 21, 2009): 300
• Study Completion Date: Not Provided
• Primary Completion Date: Not Provided

Eligibility 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.

When a pediatric subject reaches age 18, continued participation will require reconsenting with the adult consent document.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 5 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Contacts

Contact: Chevalia Robinson, R.N.; (301) 496-3973; cr163w@nih.gov

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00943514
• Other Study ID Numbers: 090172; 09-H-0172
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Responsible Party: National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
• Study Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Collaborators: Not Provided

Investigators

• Principal Investigator: Kenneth N Olivier, M.D.; National Heart, Lung, and Blood Institute (NHLBI)

• PRS Account: National Institutes of Health Clinical Center (CC)
• Verification Date: October 8, 2020