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.
調査の概要
状態
詳細な説明
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.
研究の種類
入学 (予想される)
連絡先と場所
研究連絡先
- 名前:Katerina Kusalova, Ing
- 電話番号:+420723949465
- メール:kusalova@biostatistika.cz
研究場所
-
-
Czech Republic
-
Praha、Czech Republic、チェコ、14059
- 募集
- Thomayer University Hospital
-
主任研究者:
- Jan Chlumsky, MD
-
-
参加基準
適格基準
就学可能な年齢
- 子
- 大人
- 高齢者
健康ボランティアの受け入れ
受講資格のある性別
サンプリング方法
調査対象母集団
説明
Inclusion Criteria:
- Patients with alpha-1-antitrypsin deficiency
Exclusion Criteria:
- Patient disagreement with inclusion in the study
研究計画
研究はどのように設計されていますか?
デザインの詳細
この研究は何を測定していますか?
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
---|---|---|
Changes of lung function parameters over time
時間枠: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
時間枠: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
時間枠: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
時間枠: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
時間枠: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
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
---|---|---|
Behavior of individuals with no or minimal lung involvement
時間枠: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
時間枠:within one year after completion
|
Laboratory detection of changes in liver tests (ALT, AST, ALP, GGT, ukat/l)
|
within one year after completion
|
協力者と研究者
捜査官
- 主任研究者:Jan MD Chlumsky, PhD、Thomayer University Hospital
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (予想される)
研究の完了 (予想される)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- IBA1115
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
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