{"id":"alpha-1-proteinase-inhibitor","safety":{"commonSideEffects":[{"rate":null,"effect":"Infusion reactions"},{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Dizziness"},{"rate":null,"effect":"Fever"}]},"_chembl":{"chemblId":"CHEMBL4297879","moleculeType":"Protein"},"_dailymed":{"setId":"a9a5b46e-04da-41bd-bb5f-c4936b664fef","title":"ARALAST NP (ALPHA-1-PROTEINASE INHIBITOR (HUMAN)) KIT [TAKEDA PHARMACEUTICALS AMERICA, INC.]"},"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Alpha-1 antitrypsin is a serine protease inhibitor that protects tissues from neutrophil elastase and other proteases. In alpha-1 antitrypsin deficiency, unopposed protease activity leads to progressive lung and liver damage. Exogenous AAT replacement therapy restores the protease-antiprotease balance, slowing or halting tissue destruction.","oneSentence":"Alpha-1-proteinase inhibitor (AAT) replaces or augments deficient alpha-1 antitrypsin to prevent elastin degradation in the lungs and other tissues.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T04:08:19.903Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Alpha-1 antitrypsin deficiency with emphysema"},{"name":"Alpha-1 antitrypsin deficiency with progressive lung disease"}]},"trialDetails":[{"nctId":"NCT00001532","phase":"","title":"Role of Genetic Factors in the Development of Lung Disease","status":"RECRUITING","sponsor":"National Heart, Lung, and Blood Institute (NHLBI)","startDate":"1996-09-13","conditions":"Cystic Fibrosis, Pulmonary Fibrosis, Tuberous Sclerosis","enrollment":3500},{"nctId":"NCT06389877","phase":"PHASE1, PHASE2","title":"A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)","status":"RECRUITING","sponsor":"Beam Therapeutics Inc.","startDate":"2024-06-19","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":106},{"nctId":"NCT06996756","phase":"PHASE1","title":"Gene Therapy for Alpha 1- Antitrypsin Deficiency","status":"RECRUITING","sponsor":"Weill Medical College of Cornell University","startDate":"2025-02-26","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":16},{"nctId":"NCT07431112","phase":"PHASE1","title":"A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)","status":"RECRUITING","sponsor":"AIRNA Corporation","startDate":"2026-02-27","conditions":"Alpha 1 Antitrypsin Deficiency","enrollment":54},{"nctId":"NCT07326592","phase":"PHASE4","title":"Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza / Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD)","status":"NOT_YET_RECRUITING","sponsor":"CSL Behring","startDate":"2026-04-15","conditions":"Alpha1 Antitrypsin Deficiency, Alpha1-Proteinase Inhibitor Deficiency, Emphysema","enrollment":270},{"nctId":"NCT06165341","phase":"PHASE3","title":"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)","status":"RECRUITING","sponsor":"Takeda","startDate":"2024-03-01","conditions":"Alpha1-Antitrypsin Deficiency","enrollment":50},{"nctId":"NCT05677971","phase":"PHASE3","title":"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","status":"RECRUITING","sponsor":"Takeda","startDate":"2023-03-06","conditions":"Alpha1-Antitrypsin Deficiency","enrollment":160},{"nctId":"NCT01983241","phase":"PHASE3","title":"Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Grifols Therapeutics LLC","startDate":"2013-11","conditions":"Pulmonary Emphysema in Alpha-1 PI Deficiency","enrollment":345},{"nctId":"NCT02796937","phase":"PHASE3","title":"Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency","status":"ENROLLING_BY_INVITATION","sponsor":"Grifols Therapeutics LLC","startDate":"2016-07","conditions":"Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency","enrollment":290},{"nctId":"NCT05550844","phase":"","title":"Emphysema and FLNA Mutation (E-FLNA)","status":"COMPLETED","sponsor":"University Hospital, Lille","startDate":"2023-02-01","conditions":"Emphysema","enrollment":8},{"nctId":"NCT05297812","phase":"","title":"Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease","status":"ACTIVE_NOT_RECRUITING","sponsor":"Columbia University","startDate":"2022-03-23","conditions":"Alpha 1-Antitrypsin Deficiency, Genetic Disease","enrollment":286},{"nctId":"NCT06505603","phase":"","title":"PiMZ Longitudinal Cohort (PiMZ Logic)","status":"RECRUITING","sponsor":"Columbia University","startDate":"2025-05-30","conditions":"Alpha 1-Antitrypsin Deficiency, Emphysema or COPD","enrollment":80},{"nctId":"NCT02363946","phase":"PHASE1","title":"A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)","status":"TERMINATED","sponsor":"Arrowhead Pharmaceuticals","startDate":"2015-02","conditions":"Alpha-1 Antitrypsin Deficiency","enrollment":65},{"nctId":"NCT07145385","phase":"","title":"Prevalence of Alpha-1 Antitrypsin Deficiency in Non-Cirrhotic Liver Cancer","status":"NOT_YET_RECRUITING","sponsor":"University Hospital, Bordeaux","startDate":"2026-01","conditions":"Retrospective Cohort of Patients Diagnosed With HCC on Non-Cirrhotic Liver","enrollment":71},{"nctId":"NCT03362242","phase":"PHASE1","title":"Study of ARO-AAT in Normal Adult Volunteers","status":"COMPLETED","sponsor":"Arrowhead Pharmaceuticals","startDate":"2018-03-12","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":45},{"nctId":"NCT01862211","phase":"NA","title":"Polygen Defi-Alpha: Genetic Polymorphisms Study in Children With Alpha-1 Antitrypsin Deficiency, Included in the DEFI-ALPHA Cohort","status":"COMPLETED","sponsor":"Hospices Civils de Lyon","startDate":"2013-05","conditions":"Children With a Deficiency of Alpha-1 Antitrypsin","enrollment":296},{"nctId":"NCT02900183","phase":"PHASE2","title":"Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels","status":"WITHDRAWN","sponsor":"Arrowhead Pharmaceuticals","startDate":"2016-10","conditions":"Alpha-1 Antitrypsin Deficiency","enrollment":""},{"nctId":"NCT05485155","phase":"PHASE2","title":"Zemaira Eosinophilic Esophagitis Pilot Study","status":"RECRUITING","sponsor":"Children's Hospital Medical Center, Cincinnati","startDate":"2025-05-15","conditions":"Eosinophilic Esophagitis","enrollment":15},{"nctId":"NCT06677307","phase":"PHASE1, PHASE2","title":"A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110","status":"ACTIVE_NOT_RECRUITING","sponsor":"Korro Bio, Inc.","startDate":"2025-01-13","conditions":"Alpha-1 Anti-trypsin Deficiency, AATD","enrollment":64},{"nctId":"NCT04722887","phase":"PHASE1, PHASE2","title":"A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency","status":"COMPLETED","sponsor":"Grifols Therapeutics LLC","startDate":"2021-08-13","conditions":"Alpha1-Antitrypsin Deficiency","enrollment":17},{"nctId":"NCT00500123","phase":"","title":"The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study","status":"RECRUITING","sponsor":"University of Florida","startDate":"2001-01","conditions":"Alpha-1 Antitrypsin Deficiency","enrollment":50000},{"nctId":"NCT07242079","phase":"","title":"Effect Of Pulmonary Rehabilitation in Patients With Alpha-1 Antitrypsin Deficiency","status":"COMPLETED","sponsor":"University of Parma","startDate":"2024-11-15","conditions":"Alpha-1 Antitrypsin Deficiency (AATD)","enrollment":50},{"nctId":"NCT07227207","phase":"PHASE1, PHASE2","title":"A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)","status":"NOT_YET_RECRUITING","sponsor":"Tessera Therapeutics, Inc.","startDate":"2025-12","conditions":"Alpha-1 Antitrypsin Deficiency (AATD)","enrollment":72},{"nctId":"NCT07135427","phase":"PHASE4","title":"Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency","status":"RECRUITING","sponsor":"University of Alabama at Birmingham","startDate":"2025-09-03","conditions":"Alpha 1-Antitrypsin, COPD, Antibody Deficiency","enrollment":30},{"nctId":"NCT01851642","phase":"","title":"Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs","status":"RECRUITING","sponsor":"University of Florida","startDate":"2007-08-09","conditions":"Alpha-1 Antitrypsin Deficiency, AAT Deficiency, AATD","enrollment":220},{"nctId":"NCT03805789","phase":"PHASE2, PHASE3","title":"The Safety and Efficacy of Alpha-1 Antitrypsin (AAT) for the Prevention of Graft-versus-host Disease (GVHD) in Patients Receiving Hematopoietic Cell Transplant","status":"ACTIVE_NOT_RECRUITING","sponsor":"CSL Behring","startDate":"2019-03-27","conditions":"Acute-graft-versus-host Disease","enrollment":222},{"nctId":"NCT03945292","phase":"PHASE2","title":"Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)","status":"COMPLETED","sponsor":"Arrowhead Pharmaceuticals","startDate":"2019-08-07","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":40},{"nctId":"NCT06512454","phase":"","title":"A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems","status":"RECRUITING","sponsor":"Takeda","startDate":"2024-09-25","conditions":"Alpha1-Antitrypsin Deficiency","enrollment":500},{"nctId":"NCT05897424","phase":"PHASE2","title":"Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema","status":"RECRUITING","sponsor":"Sanofi","startDate":"2024-06-20","conditions":"Alpha 1-Antitrypsin Deficiency, Emphysema","enrollment":185},{"nctId":"NCT03946449","phase":"PHASE2","title":"Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)","status":"COMPLETED","sponsor":"Arrowhead Pharmaceuticals","startDate":"2019-10-31","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":16},{"nctId":"NCT00571272","phase":"","title":"Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)","status":"SUSPENDED","sponsor":"Arbor Research Collaborative for Health","startDate":"2007-11-30","conditions":"Liver Diseases, Alagille Syndrome, Alpha 1-Antitrypsin Deficiency","enrollment":1675},{"nctId":"NCT05899673","phase":"PHASE3","title":"An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease","status":"ACTIVE_NOT_RECRUITING","sponsor":"Takeda","startDate":"2023-08-08","conditions":"Alpha1-Antitrypsin Deficiency","enrollment":31},{"nctId":"NCT06405633","phase":"PHASE1, PHASE2","title":"A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD","status":"RECRUITING","sponsor":"Wave Life Sciences Ltd.","startDate":"2024-07-19","conditions":"Alpha-1 Antitrypsin Deficiency","enrollment":24},{"nctId":"NCT07193615","phase":"EARLY_PHASE1","title":"Study of YOLT-202 in the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)","status":"ACTIVE_NOT_RECRUITING","sponsor":"RenJi Hospital","startDate":"2025-06-18","conditions":"Alpha-1 Antitrypsin Deficiency (AATD)","enrollment":18},{"nctId":"NCT07152834","phase":"","title":"Screening for Alpha-1 Antitrypsin Deficiency in Patients With Airway Obstruction","status":"RECRUITING","sponsor":"Muğla Sıtkı Koçman University","startDate":"2025-08-06","conditions":"Alpha 1-antitrypsin Deficiency (AATD)","enrollment":734},{"nctId":"NCT06738017","phase":"PHASE1","title":"Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants","status":"RECRUITING","sponsor":"BioMarin Pharmaceutical","startDate":"2025-02-21","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":12},{"nctId":"NCT05643495","phase":"PHASE2","title":"A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype","status":"TERMINATED","sponsor":"Vertex Pharmaceuticals Incorporated","startDate":"2023-02-23","conditions":"Alpha-1 Antitrypsin Deficiency","enrollment":14},{"nctId":"NCT04204252","phase":"PHASE3","title":"Phase III, Efficacy and Safety of \"Kamada-AAT for Inhalation\"","status":"RECRUITING","sponsor":"Kamada, Ltd.","startDate":"2019-11-25","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":220},{"nctId":"NCT04167514","phase":"PHASE3","title":"Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone","status":"COMPLETED","sponsor":"CSL Behring","startDate":"2020-01-09","conditions":"Graft Versus Host Disease (GVHD)","enrollment":136},{"nctId":"NCT07125664","phase":"PHASE2","title":"Intravenous Alpha-1 Antitrypsin for Hospitalized Patients With COPD Exacerbations (AECOPD Study)","status":"NOT_YET_RECRUITING","sponsor":"Fondazione IRCCS Policlinico San Matteo di Pavia","startDate":"2025-10-01","conditions":"Acute Exacerbation Chronic Obstructive Pulmonary Disease","enrollment":36},{"nctId":"NCT02014415","phase":"","title":"Alpha-1 Antitrypsin Deficiency Adult Liver Study","status":"ACTIVE_NOT_RECRUITING","sponsor":"St. Louis University","startDate":"2013-12","conditions":"Alpha-1 Antitrypsin Deficiency","enrollment":120},{"nctId":"NCT05856331","phase":"PHASE2","title":"Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema","status":"COMPLETED","sponsor":"Sanofi","startDate":"2023-10-12","conditions":"Alpha 1-Antitrypsin Deficiency, Emphysema","enrollment":99},{"nctId":"NCT06049082","phase":"PHASE1","title":"A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency","status":"RECRUITING","sponsor":"Krystal Biotech, Inc.","startDate":"2024-02-15","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":15},{"nctId":"NCT02713997","phase":"PHASE4","title":"Anti-inflammatory Therapy to Improve Outcomes After TPIAT","status":"COMPLETED","sponsor":"University of Minnesota","startDate":"2016-12","conditions":"Pancreatitis, Chronic; Diabetes; Transplant","enrollment":43},{"nctId":"NCT02691611","phase":"","title":"Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency","status":"COMPLETED","sponsor":"National Jewish Health","startDate":"2015-12","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":39},{"nctId":"NCT04799873","phase":"","title":"Alpha-1-Antitrypsin-Deficiency in COVID-19","status":"COMPLETED","sponsor":"Universität des Saarlandes","startDate":"2020-04-08","conditions":"Covid19","enrollment":10},{"nctId":"NCT06549712","phase":"NA","title":"The Fondazione Genomic SARS-CoV-2 Study COVID-19","status":"COMPLETED","sponsor":"Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico","startDate":"2020-03-23","conditions":"COVID-19, Genetic Predisposition","enrollment":4000},{"nctId":"NCT06892236","phase":"NA","title":"Preparation of IPSC for Cell Gene Editing for the Treatment of AATD","status":"ENROLLING_BY_INVITATION","sponsor":"Fondazione IRCCS Policlinico San Matteo di Pavia","startDate":"2025-01-15","conditions":"Alpha1-antitrypsin Deficiency","enrollment":3},{"nctId":"NCT06582329","phase":"PHASE2","title":"Effect of Alpha-1 Antitrypsin Supplementation on Alcohol-Associated Hepatitis","status":"NOT_YET_RECRUITING","sponsor":"Medical University Innsbruck","startDate":"2025-04","conditions":"Alcohol-Associated Hepatitis","enrollment":16},{"nctId":"NCT04621006","phase":"","title":"The Contact Activation System and Ulcerative Colitis","status":"COMPLETED","sponsor":"Esbjerg Hospital - University Hospital of Southern Denmark","startDate":"2021-05-01","conditions":"Ulcerative Colitis","enrollment":102},{"nctId":"NCT06622668","phase":"PHASE1, PHASE2","title":"NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease","status":"WITHDRAWN","sponsor":"Intellia Therapeutics","startDate":"2024-11-20","conditions":"Lung Disease, Pulmonary Disease, AATD","enrollment":""},{"nctId":"NCT04764448","phase":"PHASE2","title":"A Study of Belcesiran in Patients With AATLD","status":"TERMINATED","sponsor":"Dicerna Pharmaceuticals, Inc., a Novo Nordisk company","startDate":"2021-02-12","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":16},{"nctId":"NCT05671887","phase":"","title":"DREAM: Double Lung Transplant REgistry Aimed for Lung-limited Malignancies","status":"RECRUITING","sponsor":"Northwestern University","startDate":"2022-11-16","conditions":"Lung Cancer, Bilateral Cancer, Lung Transplant","enrollment":125},{"nctId":"NCT03804021","phase":"","title":"Long-Term Follow-up Study of ADVM-043","status":"COMPLETED","sponsor":"Adverum Biotechnologies, Inc.","startDate":"2018-12-27","conditions":"Alpha-1 Antitrypsin Deficiency","enrollment":6},{"nctId":"NCT06602986","phase":"NA","title":"Measuring the Density of Iodine in Lipiodol Depositions: Detecting an Invisible Residual Tumor After Conventional Transarterial Chemoembolization for Hepatocellular Carcinoma","status":"NOT_YET_RECRUITING","sponsor":"Pak Emirates Military Hospital","startDate":"2024-10-12","conditions":"HCC - Hepatocellular Carcinoma","enrollment":96},{"nctId":"NCT05146882","phase":"PHASE2","title":"An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)","status":"WITHDRAWN","sponsor":"Dicerna Pharmaceuticals, Inc., a Novo Nordisk company","startDate":"2021-12","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":""},{"nctId":"NCT05582798","phase":"PHASE4","title":"Bronchiectasis Alpha-1 Augmentation Trial- Modulating Airway Neutrophil Function","status":"COMPLETED","sponsor":"University of Dundee","startDate":"2022-11-01","conditions":"Bronchiectasis Adult","enrollment":20},{"nctId":"NCT02922751","phase":"","title":"FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)","status":"COMPLETED","sponsor":"Arbor Research Collaborative for Health","startDate":"2016-11-16","conditions":"Biliary Atresia, Alagille Syndrome, Alpha1 Anti-Trypsin Deficiency","enrollment":552},{"nctId":"NCT03679598","phase":"PHASE2","title":"Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency","status":"COMPLETED","sponsor":"University of Alabama at Birmingham","startDate":"2019-04-08","conditions":"Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations, Emphysema or COPD","enrollment":63},{"nctId":"NCT01812447","phase":"NA","title":"Evaluation of the Spiration® Valve System for Emphysema to Improve Lung Function","status":"COMPLETED","sponsor":"Olympus Corporation of the Americas","startDate":"2013-06","conditions":"Emphysema","enrollment":172},{"nctId":"NCT04157049","phase":"","title":"Alpha-1 Research Registry","status":"RECRUITING","sponsor":"Alpha-1 Foundation","startDate":"2019-06-20","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":4000},{"nctId":"NCT02464878","phase":"PHASE2","title":"Multicenter Trial of the Effect of AAT on Islet Transplant Engraftment and Durability After Renal Transplant","status":"COMPLETED","sponsor":"Massachusetts General Hospital","startDate":"2017-01","conditions":"Kidney Transplant, Type 1 Diabetes","enrollment":2},{"nctId":"NCT02168686","phase":"PHASE1, PHASE2","title":"Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency","status":"COMPLETED","sponsor":"Adverum Biotechnologies, Inc.","startDate":"2017-11-28","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":6},{"nctId":"NCT01676688","phase":"","title":"Safety Study of Alfalastin (Human Alpha-1 Antitrypsin) Administered at Home","status":"COMPLETED","sponsor":"Laboratoire français de Fractionnement et de Biotechnologies","startDate":"2012-01","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":17},{"nctId":"NCT01810458","phase":"","title":"Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)","status":"COMPLETED","sponsor":"University of Florida","startDate":"2013-10","conditions":"Liver Fibrosis, Alpha-1 Antitrypsin Deficiency, AAT Deficiency","enrollment":109},{"nctId":"NCT02947087","phase":"PHASE1, PHASE2","title":"Alpha-1 Antitrypsin (AAT) Enhances Islet Autograft Survival in Patients With Chronic Pancreatitis","status":"COMPLETED","sponsor":"Medical University of South Carolina","startDate":"2017-01-11","conditions":"Chronic Pancreatitis","enrollment":48},{"nctId":"NCT05466747","phase":"PHASE4","title":"A Study of RYMPHYSIA for Alpha1-Proteinase Inhibitor (A1PI) Therapy in Adults With A1PI Deficiency and Chronic Obstructive Pulmonary Disease (COPD)-Emphysema","status":"WITHDRAWN","sponsor":"Takeda","startDate":"2024-01-01","conditions":"Chronic Obstructive Pulmonary Disease (COPD), Alpha1-Antitrypsin Deficiency","enrollment":""},{"nctId":"NCT03188601","phase":"","title":"α1-antitrypsin (AAT) Levels and Functions in Allogeneic Bone Marrow Transplantation and Throughout Progression Into GVHD","status":"COMPLETED","sponsor":"Rambam Health Care Campus","startDate":"2018-01-27","conditions":"GVHD, Bone Marrow Transplant Failure, Alpha 1-Antitrypsin","enrollment":27},{"nctId":"NCT04547140","phase":"PHASE2","title":"Study to Evaluate the Safety and Efficacy of Liquid Alpha1-Proteinase Inhibitor (Human) in Hospitalized Participants With Coronavirus Disease (COVID-19)","status":"TERMINATED","sponsor":"Grifols Therapeutics LLC","startDate":"2021-01-29","conditions":"COVID-19","enrollment":57},{"nctId":"NCT02915614","phase":"NA","title":"Effects of Pulmonary Rehabilitation on Skeletal Muscle in COPD Patients","status":"UNKNOWN","sponsor":"Schön Klinik Berchtesgadener Land","startDate":"2017-03-02","conditions":"Chronic Obstructive Pulmonary Disease","enrollment":40},{"nctId":"NCT05622838","phase":"","title":"Maternal alpha1 Antitrypsin as a Marker of Intrauterine Growth Restriction in Pre-eclamptic Women","status":"UNKNOWN","sponsor":"Assiut University","startDate":"2022-12","conditions":"alpha1 Anti-trypsin, Pre-Eclampsia, Intrauterine Growth Restriction","enrollment":160},{"nctId":"NCT03470857","phase":"","title":"Oxidoreductive Balance and Lysosomal Activity in Cancer Patients.","status":"COMPLETED","sponsor":"Nicolaus Copernicus University","startDate":"2017-06-19","conditions":"Neoplasms, Antioxidants, Oxidative Stress","enrollment":83},{"nctId":"NCT04443192","phase":"PHASE1","title":"A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiXZ Subjects","status":"TERMINATED","sponsor":"Z Factor Limited","startDate":"2020-08-03","conditions":"Alpha1 Anti-Trypsin Deficiency","enrollment":69},{"nctId":"NCT03815396","phase":"PHASE1","title":"Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency","status":"COMPLETED","sponsor":"Inhibrx Biosciences, Inc","startDate":"2019-07-19","conditions":"Alpha-1 Antitrypsin Deficiency, AATD","enrollment":31},{"nctId":"NCT01357460","phase":"NA","title":"Endoscopic Lung Volume Reduction in Patients With Advanced Emphysema Due to alpha1 Antitrypsin Deficiency","status":"TERMINATED","sponsor":"Heidelberg University","startDate":"2011-05","conditions":"Hereditary Emphysema (Alpha 1-antitrypsin Deficiency)","enrollment":18},{"nctId":"NCT04966221","phase":"","title":"Functional and Structural Lung Imaging in Chronic Obstructive Pulmonary Disease","status":"UNKNOWN","sponsor":"University of Nottingham","startDate":"2022-01-01","conditions":"Chronic Obstructive Pulmonary Disease, Alpha 1-Antitrypsin Deficiency","enrollment":75},{"nctId":"NCT02520076","phase":"PHASE1, PHASE2","title":"Aralast NP in Islet Transplant","status":"COMPLETED","sponsor":"University of Alberta","startDate":"2015-08","conditions":"Type 1 Diabetes Mellitus","enrollment":5},{"nctId":"NCT05335603","phase":"","title":"An Observational Study Evaluating Patients With Chronic Liver Diseases Associated With Hepatic Steatosis","status":"RECRUITING","sponsor":"University of Copenhagen","startDate":"2020-08-24","conditions":"Chronic Liver Disease","enrollment":335},{"nctId":"NCT03636347","phase":"PHASE2","title":"A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo.","status":"COMPLETED","sponsor":"Mereo BioPharma","startDate":"2018-10-29","conditions":"Alpha 1-Antitrypsin Deficiency, Emphysema, COPD","enrollment":99},{"nctId":"NCT02870348","phase":"PHASE1, PHASE2","title":"Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE)","status":"COMPLETED","sponsor":"Grifols Therapeutics LLC","startDate":"2016-07-29","conditions":"Alpha1-Antitrypsin Deficiency","enrollment":4},{"nctId":"NCT05178277","phase":"","title":"Czech AATD Registry","status":"RECRUITING","sponsor":"Thomayer University Hospital","startDate":"2018-01-01","conditions":"Alpha-1-antitrypsin Deficiency","enrollment":300},{"nctId":"NCT02870309","phase":"PHASE1, PHASE2","title":"Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participants With Alpha1-Antitrypsin Deficiency","status":"COMPLETED","sponsor":"Grifols Therapeutics LLC","startDate":"2016-07-29","conditions":"Alpha1-Antitrypsin Deficiency","enrollment":4},{"nctId":"NCT02525861","phase":"PHASE3","title":"GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study","status":"COMPLETED","sponsor":"Baxalta now part of Shire","startDate":"2016-03-08","conditions":"Alpha1-antitrypsin Deficiency","enrollment":34},{"nctId":"NCT01379469","phase":"PHASE2","title":"Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency","status":"TERMINATED","sponsor":"Washington University School of Medicine","startDate":"2012-01","conditions":"Alpha-1-antitrypsin Deficiency, Liver Cirrhosis","enrollment":20},{"nctId":"NCT01370018","phase":"PHASE2, PHASE3","title":"Therapy to Elevate CD4 Counts in HIV-1 Disease","status":"COMPLETED","sponsor":"Institute for Human Genetics and Biochemistry","startDate":"2006-12","conditions":"HIV Disease","enrollment":4},{"nctId":"NCT02614872","phase":"PHASE2","title":"Study to Evaluate the Safety and Efficacy of Intravenous Glassia® Treatment in Lung Transplantation","status":"COMPLETED","sponsor":"Kamada, Ltd.","startDate":"2016-07-26","conditions":"Transplantation, Lung Rejection","enrollment":30},{"nctId":"NCT03459040","phase":"PHASE2","title":"A Proof of Concept Pilot Trial of Alpha-1-Antitrypsin for Pre-Emption Of Steroid-Refractory Acute GVHD","status":"COMPLETED","sponsor":"John Levine","startDate":"2018-08-17","conditions":"Graft-versus-host-disease, GVHD","enrollment":30},{"nctId":"NCT04495101","phase":"PHASE2","title":"Study to Evaluate the Safety and Efficacy of Prolastin in Hospitalized Subjects With COVID-19","status":"COMPLETED","sponsor":"Instituto Grifols, S.A.","startDate":"2020-07-29","conditions":"COVID-19","enrollment":100},{"nctId":"NCT00313144","phase":"PHASE4","title":"Aralast alpha1-proteinase Inhibitor Surveillance Study","status":"TERMINATED","sponsor":"Baxalta now part of Shire","startDate":"2006-06-09","conditions":"Alpha1-antitrypsin Deficiency","enrollment":127},{"nctId":"NCT01651351","phase":"PHASE4","title":"GLASSIA Infusion Rate Study","status":"COMPLETED","sponsor":"Baxalta now part of Shire","startDate":"2012-07-31","conditions":"Alpha1-antitrypsin Deficiency, Healthy Volunteers","enrollment":30},{"nctId":"NCT02722304","phase":"PHASE3","title":"Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency","status":"TERMINATED","sponsor":"Baxalta now part of Shire","startDate":"2016-11-02","conditions":"Chronic Obstructive Pulmonary Disease, Alpha1-antitrypsin Deficiency","enrollment":7},{"nctId":"NCT00396006","phase":"PHASE4","title":"Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor)","status":"COMPLETED","sponsor":"Baxalta now part of Shire","startDate":"2006-10-27","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":21},{"nctId":"NCT00242385","phase":"PHASE1","title":"Pharmacokinetic Study of ARALAST (Human Alpha1- PI)","status":"COMPLETED","sponsor":"Baxalta now part of Shire","startDate":"2005-12-20","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":25},{"nctId":"NCT00161707","phase":"PHASE1, PHASE2","title":"Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency","status":"COMPLETED","sponsor":"Baxalta now part of Shire","startDate":"2003-01-07","conditions":"Alpha1-antitrypsin Deficiency","enrollment":40},{"nctId":"NCT00157092","phase":"PHASE1, PHASE2","title":"Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency","status":"COMPLETED","sponsor":"Baxalta now part of Shire","startDate":"2004-03-01","conditions":"Alpha1-antitrypsin Deficiency","enrollment":15},{"nctId":"NCT03767829","phase":"PHASE1, PHASE2","title":"A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease","status":"TERMINATED","sponsor":"Alnylam Pharmaceuticals","startDate":"2018-12-05","conditions":"ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease","enrollment":32},{"nctId":"NCT04785235","phase":"","title":"Neutrophil Elastase, Elastase Alpha-1 Proteinase, Periodontitis, Gingival Crevicular Fluid and Acute Coronary Syndrome","status":"COMPLETED","sponsor":"Baskent University","startDate":"2007-10-15","conditions":"Acute Coronary Syndrome, Periodontitis","enrollment":43},{"nctId":"NCT00499941","phase":"","title":"Alpha-1 Foundation Research Registry","status":"COMPLETED","sponsor":"Medical University of South Carolina","startDate":"2000-04","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":6655},{"nctId":"NCT03008915","phase":"PHASE2","title":"Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency","status":"COMPLETED","sponsor":"Columbia University","startDate":"2017-01","conditions":"Alpha-1 Antitrypsin Deficiency, Emphysema, Chronic Obstructive Pulmonary Disease","enrollment":15},{"nctId":"NCT02956122","phase":"PHASE2, PHASE3","title":"A Phase 2/3 Study of GLASSIA for the Treatment of Acute GvHD","status":"TERMINATED","sponsor":"Baxalta now part of Shire","startDate":"2017-04-26","conditions":"Graft Versus Host Disease","enrollment":1}],"_emaApprovals":[],"_faersSignals":[{"count":143,"reaction":"PNEUMONIA"},{"count":137,"reaction":"DYSPNOEA"},{"count":132,"reaction":"PRODUCT DOSE OMISSION ISSUE"},{"count":90,"reaction":"COVID-19"},{"count":83,"reaction":"DEATH"},{"count":81,"reaction":"COUGH"},{"count":65,"reaction":"FALL"},{"count":62,"reaction":"FATIGUE"},{"count":60,"reaction":"RESPIRATORY TRACT INFECTION"},{"count":55,"reaction":"SINUSITIS"}],"_approvalHistory":[],"publicationCount":1194,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Zemaira","Prolastin"],"phase":"marketed","status":"active","brandName":"alpha-1-Proteinase Inhibitor","genericName":"alpha-1-Proteinase Inhibitor","companyName":"Institute for Human Genetics and Biochemistry","companyId":"institute-for-human-genetics-and-biochemistry","modality":"Biologic","firstApprovalDate":"","aiSummary":"Alpha-1-proteinase inhibitor (AAT) replaces or augments deficient alpha-1 antitrypsin to prevent elastin degradation in the lungs and other tissues. Used for Alpha-1 antitrypsin deficiency with emphysema, Alpha-1 antitrypsin deficiency with progressive lung disease.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}