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Alpha 1 Antitrypsin Deficiency Clinical Trials

A listing of Alpha 1 Antitrypsin Deficiency medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.


Found (7) clinical trials

Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency

Alpha-1 Antitrypsin (A1AT) is a major inhibitor of serine proteases and plays an important role in the lung as an inhibitor of neutrophil elastase. A1AT deficiency is associated with decreases in plasma A1AT levels and is associated with a great risk for developing emphysema, COPD, bronchiectasis, and asthma. Much of ...


Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency

The primary objective is to determine if Carbamazepine therapy in patients with severe liver disease due to Alpha-1-Antitrypsin Deficiency leads to a significant reduction in the hepatic accumulation of ATZ. The other objectives are: To determine whether Carbamazepine treatment reduces hepatic fibrosis in alpha-1-antitrypsin deficient patients with severe liver disease. ...


Polygen Defi-Alpha: Genetic Polymorphisms Study in Children With Alpha-1 Antitrypsin Deficiency Included in the DEFI-ALPHA Cohort

The deficiency of alpha-1 antitrypsin (DA1AT) is a genetic disorder of variable clinical expression, initially described in adults with pulmonary emphysema patients. In children, it is the second cause of neonatal cholestasis after biliary atresia and is a common indication for liver transplantation. Several genotypes for SERPINA1 gene coding for ...

Phase N/A

Environment Effect on Six-Minute Walk Test Performance

Participants will be asked to come to the Clinical Research Center (CRC) for a visit lasting about one hour. Participants will be asked to review and sign an informed consent document to participate in this trial. If participants agree to participate and sign the consent, the following procedures will be ...

Phase N/A

Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency

Emphysema is a common type of lung disease in patients with alpha-1 antitrypsin deficiency (AATD). Emphysema refers to destruction of the fine network of air spaces and blood vessels in the lung, and results in what looks like "holes" in the lung. Emphysema is associated with an increased risk of ...


Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency

The study primarily aims to establish desmosine and isodesmosine concentrations in plasma, sputum and urine measured as markers of elastin degradation systemically in the lung and also markers of inflammation and fibrinogen. Assessment of vital signs, lab tests, carbon monoxide diffusing capacity, oxygen saturation, pulmonary function tests, ECGs, physical exams ...


A clinical trial to evaluate treatments using ARO-AAT Injection for patients

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-AAT in healthy adult volunteers.