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EARCO REGISTRY. History Of Patients With Alpha-1 Antitrypsin (EARCO)

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ClinicalTrials.gov Identifier: NCT04180319
Recruitment Status : Recruiting
First Posted : November 27, 2019
Last Update Posted : February 7, 2020
Sponsor:
Collaborator:
European Respiratory Society
Information provided by (Responsible Party):
Hospital Universitari Vall d'Hebron Research Institute

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. .


Condition or disease
Alpha 1-Antitrypsin Deficiency

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.

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 3000 participants
Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
Official Title: A Pan-European Multi-Centre Observational Study To Determine The Natural History Of Patients With Alpha-1 Antitrypsin
Actual Study Start Date : February 5, 2020
Estimated Primary Completion Date : February 5, 2021
Estimated Study Completion Date : February 5, 2025





Primary Outcome Measures :
  1. Evolution of lung function The Natural History Of Patients With Alpha-1 Antitrypsin Deficiency [ Time Frame: 5 years ]
    Annual measurement of forced expiratory volume in the first second (FEV1) in liters

  2. Mortality [ Time Frame: 5 years ]
    Mortality during the duration of the study

  3. Evolution of respiratory specific health related quaility of life [ Time Frame: 5 years ]
    Annual measurements of COPD Assessment Test (CAT) (scale 0 to 40 points)

  4. Evolution of generic health related quaility of life [ Time Frame: 5 years ]
    Annual measurement of EQ-5D


Secondary Outcome Measures :
  1. Incidence of liver disease [ Time Frame: 5 years ]
    Alterations in liver enzymes and/or liver imaging tests during follow-up

  2. Evolution of respiratory symptoms [ Time Frame: 5 years ]
    identification of respìratory symptoms during follow-up



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

The study population will consist of individuals with diagnosed severe alpha-1 antitrypsin deficiency regardless of the clinical expression and severity.

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.

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04180319


Contacts
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Contact: MARC MIRAVITLLES, MD-PhD 34932746107 marcm@separ.es
Contact: EDUARDO LOEB, MD 34932746107 eduardo.loeb@vhir.org

Locations
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Spain
Marc Miravitlles Recruiting
Barcelona, Spain, 08035
Contact: Marc Miravitlles, MD -PhD    +34932746107    marcm@separ.es   
Contact: Eduardo Loeb, MD    +34932746107    eloebm@gmail.com   
Sponsors and Collaborators
Hospital Universitari Vall d'Hebron Research Institute
European Respiratory Society
Investigators
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Principal Investigator: MARC MIRAVITLLES, MD-PhD Hospital Vall d'Hebron
Principal Investigator: Timm Greulich, MD-PhD University Medical Centre Giessen and Marburg, Germany
Additional Information:
Publications:
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Responsible Party: Hospital Universitari Vall d'Hebron Research Institute
ClinicalTrials.gov Identifier: NCT04180319    
Other Study ID Numbers: EARCO
First Posted: November 27, 2019    Key Record Dates
Last Update Posted: February 7, 2020
Last Verified: November 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Hospital Universitari Vall d'Hebron Research Institute:
Natural History
Epidemiology
Registry
alpha 1-Antitrypsin Deficiency
Additional relevant MeSH terms:
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Alpha 1-Antitrypsin Deficiency
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Emphysema
Pathologic Processes