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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 (19) clinical trials

Alpha-1 Antitrypsin Deficiency Adult Liver Study

Alpha-1 Antitrypsin Deficiency (AATD) is a genetic disorder resulting in a low level of a protein called alpha-1 antitrypsin (AAT). This deficiency can cause life-threatening liver disease and/or lung disease at various ages. Some patients experience life-threatening liver disease in childhood or liver cancer as adults. There is no specific ...

Phase N/A

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

Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs

AAT deficiency is a genetic disorder that affects around 100,000 people in the USA, including 1-3% of all people diagnosed with chronic obstructive pulmonary disease (COPD). In AAT deficient people diagnosed with COPD, it was originally believed the cause of the disease was due to a lack of supply of ...

Phase N/A

AL1TER : Alpha-1 Therapy Evaluation and Research Patient Registry

This is a longitudinal, observational, non-interventional registry study, designed to collect both retrospective and prospective data, in order to acquire real-world data on product prescribing, product use by patients, and product performance for patients receiving 1-PI therapy in a homecare and/or a Coram ambulatory infusion suite. There is no limit ...

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

Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency

The variable natural clinical course of alpha-1 anti-trypsin deficiency (AATD) disease and strong influence of environmental exposures such as smoking, implicate a major role for epigenetic mechanisms in modifying AATD disease penetrance. The goal of this study proposal is to investigate epigenetic regulation of alveolar macrophage (AM) inflammation and function ...

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 ...


Patients are needed to participate in a clinical research study for the treatment of alpha-1-Antitrypsin deficiency

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.