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Czech AATD Registry

16. december 2021 opdateret af: Jan Chlumsky, M.D., Thomayer University Hospital

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.

Studieoversigt

Status

Rekruttering

Betingelser

Detaljeret beskrivelse

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.

Undersøgelsestype

Observationel

Tilmelding (Forventet)

300

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Studiesteder

  • Tjekkiet
    • Czech Republic
      • Praha, Czech Republic, Tjekkiet, 14059
        • Rekruttering
        • Thomayer University Hospital
        • Ledende efterforsker:
          • Jan Chlumsky, MD

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Prøveudtagningsmetode

Sandsynlighedsprøve

Studiebefolkning

Patients with alpha-1-antitrypsin deficiency

Beskrivelse

Inclusion Criteria:

  • Patients with alpha-1-antitrypsin deficiency

Exclusion Criteria:

  • Patient disagreement with inclusion in the study

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Changes of lung function parameters over time
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Behavior of individuals with no or minimal lung involvement
Tidsramme: 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
Tidsramme: within one year after completion
Laboratory detection of changes in liver tests (ALT, AST, ALP, GGT, ukat/l)
within one year after completion

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Thomayer University Hospital

Samarbejdspartnere

Masaryk University

Efterforskere

  • Ledende efterforsker: Jan MD Chlumsky, PhD, Thomayer University Hospital

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

1. januar 2018

Primær færdiggørelse (Forventet)

31. december 2033

Studieafslutning (Forventet)

31. december 2035

Datoer for studieregistrering

Først indsendt

12. november 2021

Først indsendt, der opfyldte QC-kriterier

16. december 2021

Først opslået (Faktiske)

5. januar 2022

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

5. januar 2022

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

16. december 2021

Sidst verificeret

1. december 2021

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • IBA1115

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Ingen

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Ingen

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Kliniske forsøg med Alpha-1-antitrypsin mangel

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Betingelser

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