European Alpha 1 Registry (EARCO)
Research type
Research Study
Full title
A Pan-European Multi-Centre Observational Study To Determine The Natural History Of Patients With Alpha-1 Antitrypsin Deficiency And The Impact Of Augmentation Therapy
IRAS ID
265728
Contact name
Alice Turner
Contact email
Sponsor organisation
University Hospitals Birmingham NHS Foundation Trust
Duration of Study in the UK
2 years, 11 months, 31 days
Research summary
Alpha 1 antitrypsin deficiency (AATD) is a rare genetic condition which can cause lung and liver disease. In order to understand why some patients are affected by it more badly than others, and how the lung disease changes over time, we need to study large groups of patients and compare their clinical characteristics. Whilst many countries have done this work nationally in the past, it is important to collaborate internationally so that we can study enough patients, and it is for this reason that this European study has been set up. All patients with AATD, defined by an AAT level <50mg/dl, are eligible. Participating centres will collect data on patient characteristics, such as age, sex and lung function, every year for at least 3 years. In addition we will collect data on treatment, in particular a specific AATD treatment (augmentation) which replaces the AAT that patients lack through a drip. Augmentation is available in some European countries but not all of them, thus the registry is an opportunity to look at real-life effects of this therapy and explore patient characteristics that relate to how well it works. Each centre will collect a minimum dataset, which in the UK is largely contained within usual care, so the study will not be burdensome to patients. A steering group which includes patient representatives has guided the choice of research questions to be assessed within the resultant dataset.
REC name
London - Camden & Kings Cross Research Ethics Committee
REC reference
19/LO/1106
Date of REC Opinion
16 Jul 2019
REC opinion
Favourable Opinion