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Clinical Trials for Alpha 1-Antitrypsin Deficiency

• A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)
• Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)
• An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease
• Open Label Extension Study of INBRX-101 in Adults With AATD Emphysema
• Study of INBRX-101 Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema
• A Phase 1, First-in-human Study of VX-668
• Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
• A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
• A Phase 1, First-in-human Study of VX-634
• An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)
• A Study of Belcesiran in Patients With A1ATD-Associated Liver Disease
• Study to Evaluate the Safety and Efficacy of Liquid Alpha1-Proteinase Inhibitor (Human) in Hospitalized Participants With Coronavirus Disease (COVID-19)
• Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
• A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiXZ Subjects
• Evaluate Efficacy and Safety of "Kamada-AAT for Inhalation" in Patients With AATD
• Study of DCR-A1AT in Healthy Adult Volunteers
• Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
• Study of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
• Safety, Tolerability and Pharmacodynamic Effect of ARO-AAT
• Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency
• A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
• Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency
• A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo.
• A Proof of Concept Pilot Trial of Alpha-1-Antitrypsin for Pre-Emption Of Steroid-Refractory Acute GVHD
• Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency
• Study of ARO-AAT in Normal Adult Volunteers
• Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency
• Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency
• Alpha-1 Antitrypsin (AAT) Enhances Islet Autograft Survival in Patients With Chronic Pancreatitis
• Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels
• A Study of an Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
• A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)
• Single Dose Administration of Alpha-1 Anti-Trypsin for the Amelioration of Organ Injury in Patients Undergoing Cardiac Surgery
• Pilot Study of alpha1-antitrypsin to Treat Neuromyelitis Optica Relapses
• Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"
• Alpha-1 Anti-Trypsin (AAT) Treatment in Acute Myocardial Infarction
• Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs
• Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)
• A Pilot Study of Alpha-1-Antitrypsin (AAT) in Steroid Refractory Acute Graft vs Host Disease
• Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.
• Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency
• The Effects of Alpha-1 Antitrypsin (AAT) on the Progression of Type 1 Diabetes
• Study of the Safety and Efficacy of Intravenous Alpha-1 Antitrypsin in Type 1 Diabetes Mellitus
• Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
• Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients
• Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency
• Aralast alpha1-proteinase Inhibitor Surveillance Study
• The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency
• Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults
• Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)
• Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency
• Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency
• 4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?