alpha1-antitrypsin Deficiency Clinical Trial
Official title:
Multi-center Study of the Liver Disease in European Patients With alpha1-antitrypsin Deficiency
Alpha1-antitrypsin deficiency (AATD) is the third most common genetic disorder leading to
death worldwide. Apart from lung disease, AATD also leads to liver involvement in up to 50%
of patients. Hence, liver involvement is the second most common cause of morbidity and
mortality in AATD patients. But the natural history of disease in adults is not well
understood and specific therapies are still in the phase of preclinical studies. Despite
these facts and the therapeutic and preventative potential, the AATD-related liver disease
is still largely being neglected by both the patients and the healthcare professionals.
To improve the hepatologic care of patients with AATD, the investigators initiated a
prospective multi-center study in Europe that systematically evaluates the liver function in
these patients and their relatives. The investigators cooperate with both patient
organizations as well as with lung centers specialized on AATD-related lung disease.
Status | Recruiting |
Enrollment | 500 |
Est. completion date | December 2020 |
Est. primary completion date | April 2020 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 18 Years to 99 Years |
Eligibility |
Inclusion Criteria: - All patients with known AATD (every genotype) - Relatives of patients with known AATD Exclusion Criteria: - Children - Pregnant women - Patients who cannot give informed consent |
Country | Name | City | State |
---|---|---|---|
Germany | RWTH Aachen University | Aachen | North Rhine Westphalia |
Lead Sponsor | Collaborator |
---|---|
RWTH Aachen University |
Germany,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of participants with Liver fibrosis, Hepatic decompensation, Liver cancer, Death or Liver transplantation | 5 years |
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