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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.

RESULTS

Found (19) clinical trials

A stage 1, prospective, randomized, placebo-controlled, double-blind study to evaluate the safety and efficacy of Alpha 1-proteinase Inhibitor (A1P1) augmentation therapy in subjects with A1P1 Deficiency and chronic obstructive pulmonary disease (COPD). This study is an approximately 2 year study that will evaluate the safety and effectiveness of the FDA ...

Phase N/A

What is Alpha-1 Antitrypsin Deficiency? Alpha-1 Antitrypsin Deficiency is a genetic (inherited) condition that may result in serious lung disease in adults and/or liver disease at any age. Alpha-1 Antitrypsin Deficiency occurs when there is a lack of a protein in the blood called alpha-1 antitrypsin, or AAT. AAT is ...

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

Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human) Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)

This is a multi-center, randomized, placebo-controlled, double blind clinical study to assess the efficacy and safety of two separate dose regimens of Alpha-1 MP versus placebo for 156 weeks (i.e., 3 years) using computed tomography (CT) of the lungs as the main measure of efficacy. The two Alpha-1 MP doses ...

Phase

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 ...

Phase

Evaluating the Genetic Causes and Progression of Cholestatic Liver Diseases (LOGIC)

Cholestasis is a rare condition that involves a reduction or obstruction of bile flow from the liver to the small intestine. When bile flow is hindered, a waste product pigment called bilirubin can escape into the bloodstream and build up to harmful levels. This may lead to the easily recognizable ...

Phase N/A

Alpha-1 Coded Testing(ACT) Study

Genetic testing for alpha-1 antitrypsin deficiency is sometimes delayed despite established testing indications. All genetic tests have risks and possible benefits. The ACT study evaluates the population demographics, reasons for testing, and outcomes through a confidential testing program. Co-morbidities of alpha-1 antitrypsin deficiency other than lung and liver disease are ...

Phase N/A

Alpha-1 Foundation Research Registry

The Registry was established in 1997 by the Alpha-1 Foundation to facilitate research initiatives and promote the development of improved treatments and a cure for Alpha-1. Located at the Medical University of South Carolina (MUSC) in Charleston, the Registry employs procedures that ensure the most stringent confidentiality of participants. The ...

Phase N/A

The Effects of Discontinuation of Vitamin K Antagonists on the Rate of Elastin Degradation

Background: Elastin is a unique protein providing elasticity, resilience and deformability to dynamic tissues, such as lungs and vasculature. Elastin fibers are characterized by their high affinity for calcium. However, calcified elastin is more prone to the degrading effects of proteases and, in turn, partially degraded elastin has an even ...

Phase N/A

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 ...

Phase