Utilidad de los diagnósticos de PCD para mejorar la atención clínica (PCD)
Un estudio clínico para recopilar datos sobre la utilidad de las pruebas que se utilizan para hacer un diagnóstico de discinesia ciliar primaria (DCP)
This cross-sectional and longitudinal observational study is to gather data on the utility of tests that are used to make a diagnosis of primary ciliary dyskinesia (PCD). There is new testing available, called nasal nitric oxide testing, that non-invasively measures nitric oxide levels in the sinus cavity. Individuals with PCD characteristically have low levels, but this testing does not have extensive data from everyday clinical practice. The objective of this proposal is to improve the diagnostic approach to children and adults with clinical concerns for primary ciliary dyskinesia (PCD).
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
This is an observational study to gather data on the utility of tests that are used to make a diagnosis of primary ciliary dyskinesia (PCD). Participant selection will be on a referral basis for the consideration of a clinical suspicion for PCD. Participants will be identified by pulmonary providers that consider testing for PCD based on the clinical symptoms of the participant. These providers will then refer for enrollment in the study based on the clinical considerations for a diagnosis of PCD. Participants will be consented during the time of testing at the Pulmonary department. Any patient referred by a clinician for concerns for PCD, including those who have had PCD genetics sent, or a ciliary biopsy performed will be enrolled in the study. In addition, any participant who is referred for nasal nitric oxide testing and meets the inclusion and exclusion criteria for this testing, will also need to provide informed consent and be enrolled in the Nasal NO REDCap registry. This equipment is not currently FDA approved or CLIA certified for PCD diagnostic testing and therefore referring clinicians will be informed that the results of this test should not influence clinical decision making regarding the diagnosis of PCD. However, there is growing evidence that this testing has utility as a non-invasive screening tool for PCD and continued data from the general pediatric pulmonary population is needed.
Tipo de estudio
Inscripción (Estimado)
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Melanie S Collins, MD
- Número de teléfono: 860-545-9440
- Correo electrónico: mcollin@connecticutchildrens.org
Copia de seguridad de contactos de estudio
- Nombre: Sigrid M Almeida, BS
- Número de teléfono: 8608375343
- Correo electrónico: salmeida@connecticutchildrens.org
Ubicaciones de estudio
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Connecticut
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Hartford, Connecticut, Estados Unidos, 06111
- Reclutamiento
- Pulmonary Division
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Contacto:
- Sigrid M Almeida, BS
- Número de teléfono: 8608375343
- Correo electrónico: salmeida@connecticutchildrens.org
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Contacto:
- Melanie S Collins, MD
- Número de teléfono: 8605459440
- Correo electrónico: mcollin@connecticutchildrens.org
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Método de muestreo
Población de estudio
The target population are ages 2 and up. Participant selection will be on a referral basis for the consideration of a clinical suspicion for PCD. Participants will be identified by pulmonary providers that consider testing for PCD based on the clinical symptoms of the participant. These providers will thenrefer for enrollment in the study based on the clinical considerations for a diagnosis of PCD.
Descripción
Inclusion Criteria:
- Age ≥ 2 years of age
- Must have two of the following clinical history points:
- Neonatal respiratory distress
- Chronic nasal congestion/runny nose
- Chronic cough
- Situs/laterality defects
- Bronchiectasis
- Ability to provide informed consent or consent of parent/guardian and ass
Exclusion Criteria:
- Recent history of sinus surgery or bloody nose in the past week
- Age < 2 years of age
- Inability to tolerate probe in nose
- Sinusitis or other respiratory exacerbation currently being treated with antibiotics
- Admitted to hospital for respiratory exacerbation (inpatient status)
- Inability to understand the requirements of the study or be unwilling to provide written informed consent (as evidenced by signature on an informed consent document approved by the IRB)
- Any other reason for which the study investigators feel the patient is not a good candidate to complete the testing
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Modelos observacionales: COHORT
- Perspectivas temporales: CROSS_SECTIONAL
Cohortes e Intervenciones
Grupo / Cohorte |
Intervención / Tratamiento |
|---|---|
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Nasal Nitric Oxide
Participants who are referred by his/her clinician for nasal NO testing and meet the inclusion and exclusion criteria will undergo testing. Clinical information regarding prior diagnostic testing will be collected at time of enrollment. Participants that have a confirmed diagnosis of PCD by genetics or ciliary biopsy at time of study entry |
Collection of already performed clinical data and nNO testing
Otros nombres:
|
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Creation of Nasal NO REDCap registry
Periodo de tiempo: 2-3 years
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To establish a research registry to characterize the features of individuals that undergo PCD diagnostic testing at Connecticut Children's Medical Center and UCONN. |
2-3 years
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Evaluation of utility of PCD diagnostic testing
Periodo de tiempo: 2-3 years
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To evaluate the utility of PCD diagnostic testing, including potential use of nasal nitric oxide in comparison to genetic testing and ciliary biopsy, in pulmonary practice at Connecticut Children's Medical Center and UCONN. |
2-3 years
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Refinement and Improvement of PCD Diagnostic Testing
Periodo de tiempo: 1-2 years
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To refine and improve current diagnostic processes for PCD diagnosis at CCMC using information gathered from the research registry established through Primary aims 1 and 2. |
1-2 years
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Colaboradores e Investigadores
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Melanie S Collins, MD, Connecticut Children's Medical Center
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- 23-052-CCMC
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Información sobre medicamentos y dispositivos, documentos del estudio
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Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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