- ICH GCP
- Amerikanska kliniska prövningsregistret
- Klinisk prövning NCT05178277
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.
Studieöversikt
Status
Betingelser
Detaljerad beskrivning
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.
Studietyp
Inskrivning (Förväntat)
Kontakter och platser
Studiekontakt
- Namn: Katerina Kusalova, Ing
- Telefonnummer: +420723949465
- E-post: kusalova@biostatistika.cz
Studieorter
-
-
Czech Republic
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Praha, Czech Republic, Tjeckien, 14059
- Rekrytering
- Thomayer University Hospital
-
Huvudutredare:
- Jan Chlumsky, MD
-
-
Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
- Barn
- Vuxen
- Äldre vuxen
Tar emot friska volontärer
Kön som är behöriga för studier
Testmetod
Studera befolkning
Beskrivning
Inclusion Criteria:
- Patients with alpha-1-antitrypsin deficiency
Exclusion Criteria:
- Patient disagreement with inclusion in the study
Studieplan
Hur är studien utformad?
Designdetaljer
Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
---|---|---|
Changes of lung function parameters over time
Tidsram: within one year after completion
|
assessement of the rate of decline of FEV1 (ml, %predicted), measured annually
|
within one year after completion
|
Changes of exercise tolerance tolerance over time
Tidsram: within one year after completion
|
assessment of changes of peak oxygen consumption (peakVO2, ml/kg/min) measured every two years
|
within one year after completion
|
Changes of respiratory function over time
Tidsram: within one year after completion
|
assessement of the rate of decline of TLco (mol/min/kPa, %predicted), measured annually
|
within one year after completion
|
Changes of quality of life over time
Tidsram: within one year after completion
|
assessement of the rate of decline of COPD assessment test (CAT, points), measured annually
|
within one year after completion
|
Relationship of pulmonary function and lung CT densitometry to better determine phenotypes of COPD due to AAT deficiency
Tidsram: within one year after completion
|
Assessement of any possible relationship of primary outcomes 1-3 using LAA (low attenuation area, %) and distribution of emphysema (craniocaudal distribution of emhysema, points)
|
within one year after completion
|
Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
---|---|---|
Behavior of individuals with no or minimal lung involvement
Tidsram: within one year after completion
|
Assessement of prognosis of deficient non-smokers by monitoring primary outcome parameters 1-3
|
within one year after completion
|
Progression of other organ disorders, namely liver
Tidsram: within one year after completion
|
Laboratory detection of changes in liver tests (ALT, AST, ALP, GGT, ukat/l)
|
within one year after completion
|
Samarbetspartners och utredare
Sponsor
Samarbetspartners
Utredare
- Huvudutredare: Jan MD Chlumsky, PhD, Thomayer University Hospital
Studieavstämningsdatum
Studera stora datum
Studiestart (Faktisk)
Primärt slutförande (Förväntat)
Avslutad studie (Förväntat)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Faktisk)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- IBA1115
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