Natural History of Bronchiectasis

De qué trata este estudio

El registro indica que este estudio recluta participantes. La disponibilidad puede variar por centro.

Tipo de estudio

Observacional

Fase

No aplica / no indicada

Rango de edad

5 años - 100 años

Sexo admitido

Todos los sexos

Voluntarios sanos

Aceptados

Participantes

900 Estimado

Estos son puntos de partida, no una decisión de elegibilidad. El equipo debe revisar todos los requisitos.

Acerca de la investigación

Bronchiectasis is a chronic inflammatory and muco-obstructive condition that results in abnormal dilation of the airways, decline in lung function, frequent exacerbations, and chronic respiratory tract infections. Cystic fibrosis (CF) is the most well studied genetic condition that causes bronchiectasis. Tremendous strides made in understanding the mechanisms of CF have led to a multitude of novel treatment strategies that have revolutionized the field. In contrast, the mechanisms of other causes for bronchiectasis remain undetermined and the disease-specific treatments for cystic fibrosis are not effective for non-CF bronchiectasis. Bronchiectasis not caused by CF carries a substantial socioeconomic burden, is a lifelong condition with no cure and, in most cases, has no identifiable cause. Prioritizing research in this important respiratory disease is essential because it affects individuals from children through older adulthood.

Background:

This protocol is aimed at exploring underlying causes and mechanisms for the development of bronchiectasis. Participants will undergo testing based on their individual clinical presentations to identify underlying causative factors. Additionally, some patients will be followed over time to better understand the natural history of evolving bronchiectasis and to assess the effect of management strategies on the course of the disease and on their chronic infections like the nontuberculous mycobacteria. As potential novel therapies develop, participants may be offered the opportunity to participate in clinical trials.

Objectives:

The primary objective of this study is to identify causes and/or underlying conditions associated with bronchiectasis.

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.

¿Quién podría participar?

La lista siguiente contiene el texto del patrocinador. Puede incluir condiciones, tratamientos previos, resultados de pruebas y otros requisitos. Aparecer en tu búsqueda no significa que califiques.

• INCLUSION CRITERIA:

1. Children age 5 years and above and adults referred to the NIH with chronic or recurring respiratory infections and/or bronchiectasis 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 bronchiectasis development and 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.
4. Subjects must have a primary or referring physician
5. Subjects must be willing to have samples (blood, tissue, sputum, and stool) stored
6. Inclusion Criteria for Relatives including children ages 5 years and above; relatives without a known diagnosis of bronchiectasis, including those who are asymptomatic, may be enrolled if they are willing to complete study assessments. These participants will be used for comparative analyses and/or screening for previously undiagnosed disease.

INCLUSION OF VULNERABLE PARTICIPANTS

Rationale for inclusion of children:

The research that is conducted on this protocol is regulated by Health and Human Services, and in compliance with federal guidelines Title 45 Code of Federal Regulations, Part 46, Subpart D. The groups of children that may be included are:

Affected minors: Bronchiectasis may have early onset and a significant impact on the lives of these patients. It is important to include this population in our study in order to understand the natural history and progression of this condition in children as they often have physiological differences as compared to adult patients. Affected minors may undergo testing that will be tailored to their specific clinical presentation.

Minors that are related to affected individuals: Children that are relatives of index patients may not be affected or show symptoms. It is important for relatives including minor relatives to be enrolled in our study, in order to help us identify the possible conditions like pre-bronchiectasis to better understand the mechanism of the disease progression. Unaffected child relatives may only undergo tests that are no greater than minimal risk.

Rational for inclusion of affected pregnant and or nursing women: Pregnant and or nursing women including pregnant minors may be included in accordance with Federal Regulations at Subpart B of 45 CFR 46. Bronchiectasis-related symptoms and respiratory status may change during pregnancy. Including pregnant participants in this observational natural history protocol will allow the study to capture longitudinal changes in clinical status and other relevant data during pregnancy and the postpartum period.

Study procedures conducted during pregnancy and breastfeeding will be limited to those determined to pose no more than minimal risk to the participant and fetus or breastfeeding infant. Procedures considered greater than minimal risk -including computed tomography scans, bronchoscopy with bronchoalveolar lavage and endobronchial biopsy, and research blood collection- will not be performed during pregnancy or breastfeeding. These procedures may be completed after the participant is no longer pregnant and, when applicable, has discontinued breastfeeding, in accordance with the protocol and investigator judgment.

EXCLUSION CRITERIA:

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1. Patients who are unable or unwilling to provide informed consent
2. 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.

PARTICIPATION OF CHILDREN:

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.

Población del estudio

Children age 5 years and above and adults referred to the NIH with chronic or recurring respiratory infections and/or bronchiectasis will be eligible for participation. Relatives including 5 years and above.

Tratamiento, grupos y participación

El registro puede describir tratamientos y duración sin incluir un calendario completo. Pide al equipo las visitas, procedimientos, riesgos, costos y alternativas. Mantén tu atención habitual salvo que tu médico te indique un cambio.

Asignación

No informado

Enmascaramiento

No informado

Qué evalúan los investigadores

Estas medidas indican qué se estudia. No prueban que un tratamiento funcione.

Medidas principales

Medidas secundarias

Centros de investigación (1)

El estado general puede diferir del estado de un centro. Confirma la disponibilidad antes de trasladarte.

1 centros

Equipo y fechas

Patrocinador principal

National Heart, Lung, and Blood Institute (NHLBI)

Inicio del estudio

1 ago 2009 Real

Finalización primaria

No informado

Finalización del estudio

No informado

Primera publicación

22 jul 2009 Estimado

Última actualización

9 nov 2026 Real

Contactos oficiales del estudio