A Pan-European Multi-Centre Observational Study To Determine The Natural History Of Patients With Alpha-1 Antitrypsin
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 3,000
- 试验地点
- 1
- 主要终点
- Mortality
研究概览
简要总结
European Alpha-1 Research Collaboration (EARCO) is a pan-European network committed to promoting clinical research and education in alpha-1 antitrypsin deficiency (AATD). The core project is the pan-European AATD Registry, a collaboration which will offer longitudinal real-world data for patients with AATD. EARCO has a global vision to increase the early diagnosis of alpha-1 antitrypsin deficiency (AATD), understand better the natural history of the disease and ensure optimal access to effective care, placing emphasis on ambitions that serve collective needs of the AATD research community and bringing people with AAT deficiency to the centre of the research environment in a real-world context.
The study population will consist of individuals with diagnosed severe alpha-1 antitrypsin deficiency regardless of the clinical expression and severity.
The study objectives are:
- To generate long-term, high-quality clinical data covering a pan-European population of AATD individuals in all age groups and all stages of disease severity.
- To understand the natural history and prognosis of AATD better with the goal to create and validate prognostic tools to support medical decision making.
- To investigate the effect of augmentation therapy on the progression of emphysema and to examine its impact on clinical and functional outcomes, such as FEV1, quality of life and mortality in a "real-life" population
- To learn more about the course of the disease in patients suffering from severe AATD with genotypes different from Pi*ZZ We expect to collect detailed information from around 1,000 patients from at least 10 countries during the first year, expanding to 3,000 from more than 25 countries over the 5 years of the CRC and continue a long term follow-up.
We expect to collect detailed information from around 1,000 patients from at least 10 countries during the first year, expanding to 3,000 from more than 25 countries over the 5 years of the CRC and continue a long term follow-up. .
详细描述
EARCO takes advantage of existing AATD registries that have been developed at the national level. Several countries have established registries in which AATD patients are included and followed-up with clinical and biological data collected. However, these registries differ in terms of inclusion criteria, data collected and frequency and extent of follow-up. Within EARCO, we will harmonize the data collection process and assess the quality of the data within a short time frame after the data are generated and entered into the database.
We expect to collect detailed information from around 1,000 patients from at least 10 countries during the first year, expanding to 3,000 from more than 25 countries over the 5 years of the CRC and continue a long term follow-up. .
The study objectives are:
- To generate long-term, high-quality clinical data covering a pan-European population of AATD individuals in all age groups and all stages of disease severity.
- To understand the natural history and prognosis of AATD better with the goal to create and validate prognostic tools to support medical decision making.
- To investigate the effect of augmentation therapy on the progression of emphysema and to examine its impact on clinical and functional outcomes, such as FEV1, quality of life and mortality in a "real-life" population
- To learn more about the course of the disease in patients suffering from severe AATD with genotypes different from Pi*ZZ
Study design:
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed Alpha-1 antitrypsin deficiency, defined as
- •AAT serum level < 11 µM (50 mg/dl) and/or
- •proteinase inhibitor genotypes ZZ, SZ, and compound heterozygotes or homozygotes of other rare deficient variants.
排除标准
- •Patients unwilling or unable to participate in the study
结局指标
主要结局
Mortality
时间窗: 5 years
Mortality during the duration of the study
Evolution of respiratory specific health related quaility of life
时间窗: 5 years
Annual measurements of COPD Assessment Test (CAT) (scale 0 to 40 points)
Evolution of generic health related quaility of life
时间窗: 5 years
Annual measurement of EQ-5D
Evolution of lung function The Natural History Of Patients With Alpha-1 Antitrypsin Deficiency
时间窗: 5 years
Annual measurement of forced expiratory volume in the first second (FEV1) in liters
次要结局
- Evolution of respiratory symptoms(5 years)
- Incidence of liver disease(5 years)