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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 (20) clinical trials

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

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

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

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

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

Management of Patients With Alpha-1 Antitrypsin Deficiency Associated Emphysema

All patients who suffer from this disease are followed every 6 months during a 5 years period. FEV1 decline is the primary endpoint. Quality of life assessed using the Saint George's Respiratory Questionary is recorded too.

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

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

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