EARCO REGISTRY. History Of Patients With Alpha-1 Antitrypsin

Alpha 1-Antitrypsin Deficiency Clinical Trial

— EARCO  

Official title:

A Pan-European Multi-Centre Observational Study To Determine The Natural History Of Patients With Alpha-1 Antitrypsin

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04180319
• Other study ID #: EARCO
• Status: Recruiting
• Start date: February 5, 2020
• Est. completion date: February 5, 2025

Study information

• Verified date: November 2019
• Source: Hospital Universitari Vall d'Hebron Research Institute
• Contact: MARC MIRAVITLLES, MD-PhD
• Phone: 34932746107
• Email: marcm[@]separ.es
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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. .

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 3000
• Est. completion date: February 5, 2025
• Est. primary completion date: February 5, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency

Locations

Country	Name	City	State
Spain	Marc Miravitlles	Barcelona

Sponsors (2)

Lead Sponsor	Collaborator
Hospital Universitari Vall d'Hebron Research Institute	European Respiratory Society

Country where clinical trial is conducted

Spain, 

References & Publications (2)

Miravitlles M, Chorostowska-Wynimko J, Ferrarotti I, McElvaney NG, O'Hara K, Stolk J, Stockley RA, Turner A, Wilkens M, Greulich T; EARCO Clinical Research Collaboration; Members of the EARCO Clinical Research Collaboration. The European Alpha-1 Research Collaboration (EARCO): a new ERS Clinical Research Collaboration to promote research in alpha-1 antitrypsin deficiency. Eur Respir J. 2019 Feb 14;53(2). pii: 1900138. doi: 10.1183/13993003.00138-2019. Print 2019 Feb. — View Citation

Miravitlles M, Dirksen A, Ferrarotti I, Koblizek V, Lange P, Mahadeva R, McElvaney NG, Parr D, Piitulainen E, Roche N, Stolk J, Thabut G, Turner A, Vogelmeier C, Stockley RA. European Respiratory Society statement: diagnosis and treatment of pulmonary disease in a(1)-antitrypsin deficiency. Eur Respir J. 2017 Nov 30;50(5). pii: 1700610. doi: 10.1183/13993003.00610-2017. Print 2017 Nov. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evolution of lung function The Natural History Of Patients With Alpha-1 Antitrypsin Deficiency	Annual measurement of forced expiratory volume in the first second (FEV1) in liters	5 years
Primary	Mortality	Mortality during the duration of the study	5 years
Primary	Evolution of respiratory specific health related quaility of life	Annual measurements of COPD Assessment Test (CAT) (scale 0 to 40 points)	5 years
Primary	Evolution of generic health related quaility of life	Annual measurement of EQ-5D	5 years
Secondary	Incidence of liver disease	Alterations in liver enzymes and/or liver imaging tests during follow-up	5 years
Secondary	Evolution of respiratory symptoms	identification of respìratory symptoms during follow-up	5 years

External Links

•   Access to EARCO website