Not applicableStudy completedNCT03804021What this trial is testingLong-Term Follow-up Study of ADVM-043Who this might be right forAlpha-1 Antitrypsin Deficiency Adverum Biotechnologies, Inc. 6
Testing effectiveness (Phase 2)Ended earlyNCT04764448What this trial is testingBelcesiran in Patients With AATLDWho this might be right forAlpha 1-Antitrypsin Deficiency Dicerna Pharmaceuticals, Inc., a Novo Nordisk company 16
Post-approval studies (Phase 4)Study completedNCT00670007What this trial is testingExtension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.Who this might be right forEmphysemaAlpha 1-proteinase Inhibitor Deficiency CSL Behring 140
Early research (Phase 1)Study completedNCT03362242What this trial is testingStudy of ARO-AAT in Normal Adult VolunteersWho this might be right forAlpha 1-Antitrypsin Deficiency Arrowhead Pharmaceuticals 45
Not applicableStudy completedNCT01676688What this trial is testingSafety Study of Alfalastin (Human Alpha-1 Antitrypsin) Administered at HomeWho this might be right forAlpha 1-Antitrypsin Deficiency Laboratoire français de Fractionnement et de Biotechnologies 17
Testing effectiveness (Phase 2)Study completedNCT03114020What this trial is testingEfficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin DeficiencyWho this might be right forEmphysemaAlpha 1-Antitrypsin Deficiency Gerard Turino 27
Not applicableLooking for participantsNCT00001532What this trial is testingRole of Genetic Factors in the Development of Lung DiseaseWho this might be right forCystic FibrosisPulmonary FibrosisTuberous Sclerosis+2 more National Heart, Lung, and Blood Institute (NHLBI) 3,500
Not applicableStudy completedNCT03383939What this trial is testingInhaled A1AT in Adult Stable BronchiectasisWho this might be right forBronchiectasis Adult Hospital Universitari Vall d'Hebron Research Institute 20
Not applicableLooking for participantsNCT06512454What this trial is testingA Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver ProblemsWho this might be right forAlpha1-Antitrypsin Deficiency Takeda 500
Post-approval studies (Phase 4)Study completedNCT00396006What this trial is testingEfficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor)Who this might be right forAlpha 1-Antitrypsin Deficiency Baxalta now part of Shire 21
Large-scale testing (Phase 3)Active Not RecruitingNCT05899673What this trial is testingAn Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver DiseaseWho this might be right forAlpha1-Antitrypsin Deficiency Takeda 31
Large-scale testing (Phase 3)Ended earlyNCT02722304What this trial is testingStage 1 Study of ARALAST NP and GLASSIA in A1PI DeficiencyWho this might be right forChronic Obstructive Pulmonary DiseaseAlpha1-antitrypsin Deficiency Baxalta now part of Shire 7
Early research (Phase 1)Ended earlyNCT04443192What this trial is testingA Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiXZ SubjectsWho this might be right forAlpha1 Anti-Trypsin Deficiency Z Factor Limited 69
Large-scale testing (Phase 3)Looking for participantsNCT06165341What this trial is testingStudy to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)Who this might be right forAlpha1-Antitrypsin Deficiency Takeda 50
Early research (Phase 1)Ended earlyNCT02503683What this trial is testingAn Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver DiseaseWho this might be right forAntitrypsin Deficiency Liver Disease Alnylam Pharmaceuticals 26
Testing effectiveness (Phase 2)Ended earlyNCT04167345What this trial is testingEvaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ GenotypeWho this might be right forAlpha 1-Antitrypsin Deficiency Vertex Pharmaceuticals Incorporated 48
Testing effectiveness (Phase 2)Study completedNCT03008915What this trial is testingTargeting Pulmonary Perfusion in Alpha-1 Antitrypsin DeficiencyWho this might be right forAlpha-1 Antitrypsin DeficiencyEmphysemaChronic Obstructive Pulmonary Disease Columbia University 15
Testing effectiveness (Phase 2)Ended earlyNCT02093221What this trial is testingStudy of Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes MellitusWho this might be right forType 1 Diabetes Mellitus Grifols Therapeutics LLC 76
Early research (Phase 1)Study completedNCT05579431What this trial is testingA Phase 1, First-in-human Study of VX-634Who this might be right forAlpha 1-Antitrypsin Deficiency Vertex Pharmaceuticals Incorporated 127
Large-scale testing (Phase 3)Study completedNCT00295061What this trial is testingComparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient AdultsWho this might be right forAlpha 1-Antitrypsin Deficiency Grifols Therapeutics LLC 24