| Recruiting | A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD) NCT07431112 | AIRNA Corporation | Phase 1 |
| Recruiting | PiMZ Longitudinal Cohort (PiMZ Logic) NCT06505603 | Columbia University | — |
| Recruiting | Gene Therapy for Alpha 1- Antitrypsin Deficiency NCT06996756 | Weill Medical College of Cornell University | Phase 1 |
| Recruiting | Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants NCT06738017 | BioMarin Pharmaceutical | Phase 1 |
| Withdrawn | NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease NCT06622668 | Intellia Therapeutics | Phase 1 / Phase 2 |
| Active Not Recruiting | A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ NCT06405633 | Wave Life Sciences Ltd. | Phase 1 / Phase 2 |
| Active Not Recruiting | Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema NCT05897424 | Sanofi | Phase 2 |
| Recruiting | A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency NCT06389877 | Beam Therapeutics Inc. | Phase 1 / Phase 2 |
| Recruiting | A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency NCT06049082 | Krystal Biotech, Inc. | Phase 1 |
| Completed | Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema NCT05856331 | Sanofi | Phase 2 |
| Terminated | A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype NCT05643495 | Vertex Pharmaceuticals Incorporated | Phase 2 |
| Completed | A Phase 1, First-in-human Study of VX-668 NCT05727800 | Vertex Pharmaceuticals Incorporated | Phase 1 |
| Completed | A Phase 1, First-in-human Study of VX-634 NCT05579431 | Vertex Pharmaceuticals Incorporated | Phase 1 |
| Completed | Baby Detect : Genomic Newborn Screening NCT05687474 | Centre Hospitalier Universitaire de Liege | — |
| Active Not Recruiting | Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease NCT05297812 | Columbia University | — |
| Unknown | Functional and Structural Lung Imaging in Chronic Obstructive Pulmonary Disease NCT04966221 | University of Nottingham | — |
| Withdrawn | An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AAT NCT05146882 | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | Phase 2 |
| Terminated | A Study of Belcesiran in Patients With AATLD NCT04764448 | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | Phase 2 |
| Unknown | EARCO REGISTRY. History Of Patients With Alpha-1 Antitrypsin NCT04180319 | Hospital Universitari Vall d'Hebron Research Institute | — |
| Terminated | Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype NCT04167345 | Vertex Pharmaceuticals Incorporated | Phase 2 |
| Recruiting | Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation" NCT04204252 | Kamada, Ltd. | Phase 3 |
| Completed | Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Diseas NCT03946449 | Arrowhead Pharmaceuticals | Phase 2 |
| Completed | Study of DCR-A1AT in Healthy Adult Volunteers NCT04174118 | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | Phase 1 |
| Completed | Respreeza® Self-administration and Learning Program (AmAREtTI Study) NCT04262284 | CSL Behring | — |
| Completed | Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT) NCT03945292 | Arrowhead Pharmaceuticals | Phase 2 |
| Completed | Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency NCT03815396 | Inhibrx Biosciences, Inc | Phase 1 |
| Recruiting | Alpha-1 Research Registry NCT04157049 | Alpha-1 Foundation | — |
| Unknown | Effects of Different Exercise Training Modalities in Alpha-1 Antitrypsin Deficiency Patients NCT03802357 | Schön Klinik Berchtesgadener Land | N/A |
| Completed | Long-Term Follow-up Study of ADVM-043 NCT03804021 | Adverum Biotechnologies, Inc. | — |
| Completed | A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Pla NCT03636347 | Mereo BioPharma | Phase 2 |
| Withdrawn | Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjec NCT03385395 | Octapharma | Phase 2 |
| Recruiting | Exploring the Utility of Hyperpolarized 129Xe MRI in Healthy Volunteers and Patients With Lung Disease NCT03455686 | McMaster University | N/A |
| Completed | Study of ARO-AAT in Normal Adult Volunteers NCT03362242 | Arrowhead Pharmaceuticals | Phase 1 |
| Recruiting | Czech AATD Registry NCT05178277 | Thomayer University Hospital | — |
| Completed | Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency NCT02168686 | Adverum Biotechnologies, Inc. | Phase 1 / Phase 2 |
| Unknown | The Effects of Discontinuation of Vitamin K Antagonists on the Rate of Elastin Degradation NCT03285100 | Canisius-Wilhelmina Hospital | — |
| Completed | Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Defici NCT03114020 | Gerard Turino | Phase 2 |
| Completed | Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency NCT03008915 | Columbia University | Phase 2 |
| Completed | The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency NCT03039335 | Biocerna LLC | — |
| Withdrawn | Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Level NCT02900183 | Arrowhead Pharmaceuticals | Phase 2 |
| Completed | Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency NCT02691611 | National Jewish Health | — |
| Terminated | Environment Effect on Six-Minute Walk Test Performance NCT02502201 | University of Florida | N/A |
| Completed | Microbioma in Sputa From COPD With Alpha-1 Antitrypsin Deficiency NCT02547532 | Fondazione Salvatore Maugeri | — |
| Completed | Alpha-1 Carrier Genomics Study NCT02810327 | Medical University of South Carolina | — |
| Terminated | A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD) NCT02363946 | Arrowhead Pharmaceuticals | Phase 1 |
| Withdrawn | Lung Volume Reduction Coils for Emphysema in Alpha-1 Antitrypsin Deficiency NCT02273349 | University Hospital, Saarland | N/A |
| Completed | Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation" NCT02001688 | Kamada, Ltd. | Phase 2 |
| Active Not Recruiting | Alpha-1 Antitrypsin Deficiency Adult Liver Study NCT02014415 | St. Louis University | — |
| Completed | Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD) NCT01810458 | University of Florida | — |
| Completed | Ex-vivo Perfusion and Ventilation of Lungs Recovered From Non-Heart-Beating Donors to Assess Transplant Suitab NCT01615484 | University of North Carolina, Chapel Hill | N/A |
| Completed | Genomic Research in Alpha-1 Antitrypsin Deficiency NCT01832220 | University of Pittsburgh | — |
| Completed | Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation. NCT01669421 | Michael Campos, MD | Phase 2 |
| Terminated | Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency NCT01379469 | Washington University School of Medicine | Phase 2 |
| Completed | Safety Study of Alfalastin (Human Alpha-1 Antitrypsin) Administered at Home NCT01676688 | Laboratoire français de Fractionnement et de Biotechnologies | — |
| Terminated | Phase 2 Study of Ex-vivo Perfusion and Ventilation of Lungs to Assess Transplant Suitability NCT01241942 | University of North Carolina, Chapel Hill | N/A |
| Completed | Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency NCT01054339 | Beacon Therapeutics | Phase 2 |
| Completed | Effects of Exercise Training in Chronic Obstructive Pulmonary Disease Versus Alpha-1-Antitrypsin-deficiency-pa NCT01380626 | Schön Klinik Berchtesgadener Land | N/A |
| Completed | Prevalence of Alpha-1 Antitrypsin Deficiency in Chronic Obstructive Pulmonary Disease (COPD) NCT01419158 | Alpha-1 Foundation | — |
| Suspended | Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC) NCT00571272 | Arbor Research Collaborative for Health | — |
| Recruiting | Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs NCT01851642 | University of Florida | — |
| Completed | The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency NCT00532805 | Medical University of South Carolina | — |
| Completed | Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Def NCT00460096 | Kamada, Ltd. | Phase 2 / Phase 3 |
| Completed | Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhib NCT00396006 | Baxalta now part of Shire | Phase 4 |
| Completed | The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Defici NCT00301366 | Grifols Therapeutics LLC | Phase 3 |
| Completed | Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficien NCT00295061 | Grifols Therapeutics LLC | Phase 3 |
| Completed | Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency NCT00430768 | University of Massachusetts, Worcester | Phase 1 |
| Completed | Pharmacokinetic Study of ARALAST (Human Alpha1- PI) NCT00242385 | Baxalta now part of Shire | Phase 1 |
| Unknown | Management of Patients With Alpha-1 Antitrypsin Deficiency Associated Emphysema NCT00700934 | Assistance Publique - Hôpitaux de Paris | — |
| Completed | Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency NCT00377416 | University of Massachusetts, Worcester | EARLY_Phase 1 |
| Completed | Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE) NCT00263887 | Grifols Therapeutics LLC | Phase 2 |
| Enrolling By Invitation | Alpha-1 Foundation DNA and Tissue Bank NCT00884455 | University of Florida | — |
| Completed | 4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency? NCT00067756 | University of Florida | Phase 2 |
| Recruiting | The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study NCT00500123 | University of Florida | — |
| Completed | Alpha-1 Foundation Research Registry NCT00499941 | Medical University of South Carolina | — |
| Terminated | Study of Genotype and Phenotype in Patients With Alpha 1-Antitrypsin Deficiency NCT00005098 | National Center for Research Resources (NCRR) | — |
| Completed | Characterization of the Pathobiology of Early Lung Destruction in Alpha 1-Antitrypsin Deficient Individuals NCT00001462 | National Heart, Lung, and Blood Institute (NHLBI) | — |
| Completed | Alpha1-antitrypsin Deficiency Registry NCT00005292 | National Heart, Lung, and Blood Institute (NHLBI) | — |
| No Longer Available | Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After NCT03172455 | Impatients N.V. trading as myTomorrows | — |