EARCO REGISTRY. History Of Patients With Alpha-1 Antitrypsin

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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