Czech AATD Registry
Czech Alpha-1 Antitrypsin Deficiency Registry, the National Observational Study.
Alpha-1-antitrypsin deficiency is the most common congenital disease of the respiratory system, leading to early pulmonary emphysema or bronchiectasis. Pulmonary involvement significantly accelerates active cigarette smoking. Patients with alpha-1-antitrypsin deficiency may also have liver cirrhosis, vasculitis, skin or intestinal disorders.
The AATD Registry is a non-interventional multicenter retrospective prospective longitudinal follow-up of patients with alpha-1-antitrypsin deficiency.
The aim of the AATD National Registry is to collect and analyze clinical data in patients with alpha-1 antitrypsin deficiency.
Descripción general del estudio
Estado
Condiciones
Descripción detallada
Alpha-1 antitrypsin deficiency is a genetic disorder that may result in lung disease or liver disease. It is assume that it affects 1 person from a cohort of 2,000-5,000 people of the general population. Among patients with COPD, the incidence of the disorder is significantly higher. The prognosis of these patients is incomparably worse compared to classic COPD, because it affects younger patients and the rate of lung tissue loss is faster. The diagnosis is made in patients with pre-existing COPD by examination of the plasma concentration of AAT. In case of its reduction, genetic examination is added. The progression of the disease is rapid and has been shown to be slowed by lifelong augmentation treatment with human AAT. However, in routine clinical practice, it is very difficult to assess the effectiveness of treatment, the progression of lung disease or the prognosis of the disease.
The AATD registry is a non-interventional multicenter retrospective prospective longitudinal follow-up of patients with alpha-1-antitrypsin deficiency. The national registry collects data from all patients with severe or rare AAT deficiency, regardless of the type of organ impairment and age, and thus provides a view of this genetic variation in the Czech population.
The aim of the AATD Registry is to collect and analyse clinical data of patients with alpha-1 antitrypsin deficiency and increase the professional awareness of this hereditary disease.
Tipo de estudio
Inscripción (Anticipado)
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Katerina Kusalova, Ing
- Número de teléfono: +420723949465
- Correo electrónico: kusalova@biostatistika.cz
Ubicaciones de estudio
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Czech Republic
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Praha, Czech Republic, Chequia, 14059
- Reclutamiento
- Thomayer University Hospital
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Investigador principal:
- Jan Chlumsky, MD
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Niño
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Método de muestreo
Población de estudio
Descripción
Inclusion Criteria:
- Patients with alpha-1-antitrypsin deficiency
Exclusion Criteria:
- Patient disagreement with inclusion in the study
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Changes of lung function parameters over time
Periodo de tiempo: within one year after completion
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assessement of the rate of decline of FEV1 (ml, %predicted), measured annually
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within one year after completion
|
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Changes of exercise tolerance tolerance over time
Periodo de tiempo: within one year after completion
|
assessment of changes of peak oxygen consumption (peakVO2, ml/kg/min) measured every two years
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within one year after completion
|
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Changes of respiratory function over time
Periodo de tiempo: within one year after completion
|
assessement of the rate of decline of TLco (mol/min/kPa, %predicted), measured annually
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within one year after completion
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Changes of quality of life over time
Periodo de tiempo: within one year after completion
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assessement of the rate of decline of COPD assessment test (CAT, points), measured annually
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within one year after completion
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Relationship of pulmonary function and lung CT densitometry to better determine phenotypes of COPD due to AAT deficiency
Periodo de tiempo: within one year after completion
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Assessement of any possible relationship of primary outcomes 1-3 using LAA (low attenuation area, %) and distribution of emphysema (craniocaudal distribution of emhysema, points)
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within one year after completion
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Behavior of individuals with no or minimal lung involvement
Periodo de tiempo: within one year after completion
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Assessement of prognosis of deficient non-smokers by monitoring primary outcome parameters 1-3
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within one year after completion
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Progression of other organ disorders, namely liver
Periodo de tiempo: within one year after completion
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Laboratory detection of changes in liver tests (ALT, AST, ALP, GGT, ukat/l)
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within one year after completion
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Colaboradores e Investigadores
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Jan MD Chlumsky, PhD, Thomayer University Hospital
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Anticipado)
Finalización del estudio (Anticipado)
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
- IBA1115
Información sobre medicamentos y dispositivos, documentos del estudio
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