Brief Title
EARCO REGISTRY. History Of Patients With Alpha-1 Antitrypsin
Official Title
A Pan-European Multi-Centre Observational Study To Determine The Natural History Of Patients With Alpha-1 Antitrypsin
Brief Summary
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. .
Detailed Description
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:
The EARCO registry is a non-interventional, multi-centre, pan-European, prospective,
repetitive, observational cohort study enrolling patients with AATD (as confirmed by serum
level and genotyping). Patients will be managed according to their local procedures and
policies with no interference from the study team. Patients will give informed consent to
participate, and participating investigators will collect data prospectively. The following
domains are covered: demographics, proteinase inhibitor genotype and other laboratory
analyses, comorbidities, lung function, transient elastography of the liver, exacerbations,
quality of life (QoL), chest CT (if applicable), and treatment.
The study protocol was reviewed by the Research Ethics Committee of the Vall d'Hebron
University Hospital of Barcelona, Spain, and the study is sponsored by Vall d'Hebron Research
Institute, Barcelona, Spain. The study website will be located at www.AATD.eu. The registry
was developed according to recommendations on the design, implementation, governance and
long-term sustainability of disease registries in the European Union (EU). It is funded as a
clinical research collaboration (CRC) under the umbrella of the European Respiratory Society
(ERS). Clinical Research Collaborations (CRCs) are pan-European, multi-centre networks of
principal investigators aiming to advance science and clinical research within a specific
disease area (www.ersnet.org/ers-funding/clinical-research-collaboration.html). The European
Alpha-1 Clinical Research Collaboration (EARCO) was established as an ERS CRC with the
objective of developing a European AATD Registry and bringing researchers together
internationally to promote clinical research, clinical care and education.
Study Type
Observational [Patient Registry]
Primary Outcome
Evolution of lung function The Natural History Of Patients With Alpha-1 Antitrypsin Deficiency
Secondary Outcome
Incidence of liver disease
Condition
Alpha 1-Antitrypsin Deficiency
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
3000
Start Date
February 5, 2020
Completion Date
February 5, 2025
Primary Completion Date
February 5, 2021
Eligibility Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- Patients unwilling or unable to participate in the study
Gender
All
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
MARC MIRAVITLLES, MD-PhD, 34932746107, [email protected]
Location Countries
Spain
Location Countries
Spain
Administrative Informations
NCT ID
NCT04180319
Organization ID
EARCO
Responsible Party
Sponsor
Study Sponsor
Hospital Universitari Vall d'Hebron Research Institute
Collaborators
European Respiratory Society
Study Sponsor
MARC MIRAVITLLES, MD-PhD, Principal Investigator, Hospital Vall d'Hebron
Verification Date
November 2019