Not applicableNot Yet RecruitingNCT06029543What this trial is testingPermian Health Women's Lung Health StudyWho this might be right forCOPDLung Function DecreasedAlpha 1-Antitrypsin Permian Health Lung Institute 350
Testing effectiveness (Phase 2)WithdrawnNCT06622668What this trial is testingNTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung DiseaseWho this might be right forLung DiseasePulmonary DiseaseAATD+2 more Intellia Therapeutics
Not applicableEnrolling By InvitationNCT06334666What this trial is testingThe Efficacy of Pedometer-motivated Physical Activity for the Management of Patients With MASLD.Who this might be right forDaily Step CountMASLDBMI+5 more Mahidol University 86
Not applicableStudy completedNCT04186260What this trial is testingA Gender and Culturally Specific Approach to Reduce NAFLD in Mexican-American MenWho this might be right forNAFLD University of Arizona 15
Post-approval studies (Phase 4)Study completedNCT01651351What this trial is testingGLASSIA Infusion Rate StudyWho this might be right forAlpha1-antitrypsin DeficiencyHealthy Volunteers Baxalta now part of Shire 30
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
Not applicableStudy completedNCT04277819What this trial is testingThe Use of Novel Diagnostic Tools to Increase Detection of Early Fibrosis in Cystic Fibrosis Related Liver Disease to Improve Clinical ManagementWho this might be right forCystic FibrosisLiver FibrosesCystic Fibrosis Liver Disease Manchester University NHS Foundation Trust 157
Testing effectiveness (Phase 2)Study completedNCT02595983What this trial is testingThe Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver TransplantWho this might be right forTransthyretin (TTR)-Mediated AmyloidosisFamilial Amyloidotic Polyneuropathy (FAP)ATTR Amyloidosis+1 more Alnylam Pharmaceuticals 12
Not applicableLooking for participantsNCT03131427What this trial is testingChina Registry for Genetic / Metabolic Liver DiseasesWho this might be right forGenetic/Metabolic Liver Diseases Beijing Friendship Hospital 20,000
Large-scale testing (Phase 3)Looking for participantsNCT07555483What this trial is testingA Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)Who this might be right forAlpha 1 Antitrypsin Deficiency Grifols Therapeutics LLC 40
Not applicableStudy completedNCT00005305What this trial is testingHepatitis Delta Infections in HemophiliacsWho this might be right forBlood DiseaseHepatitis, Viral, HumanLiver Diseases+1 more University of North Carolina, Chapel Hill 61
Testing effectiveness (Phase 2)Active Not RecruitingNCT02867592What this trial is testingCabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare TumorsWho this might be right forAdrenal Cortical CarcinomaAlveolar Soft Part SarcomaCentral Nervous System Neoplasm+41 more National Cancer Institute (NCI) 109
Not applicableActive Not RecruitingNCT05344599What this trial is testingEvaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia SyndromeWho this might be right forPostural Orthostatic Tachycardia SyndromeAcute Hepatic Porphyria Vanderbilt University Medical Center 70
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
Not applicableUnknownNCT02850692What this trial is testingPortal Hypertension and Systemic Endothelial FunctionWho this might be right forCystic FibrosisPortal Hypertension Hopital Foch 60
Not applicableEnrolling By InvitationNCT06892236What this trial is testingPreparation of IPSC for Cell Gene Editing for the Treatment of AATDWho this might be right forAlpha1-antitrypsin Deficiency Fondazione IRCCS Policlinico San Matteo di Pavia 3
Not applicableStudy completedNCT04965571What this trial is testingClinical Features and Outcome of Wilson's Disease With Generalized Epilepsy in Chinese PatientsWho this might be right forWilson DiseaseGeneralized Epilepsy Second Affiliated Hospital, School of Medicine, Zhejiang University 13
Not applicableUnknownNCT03802357What this trial is testingEffects of Different Exercise Training Modalities in Alpha-1 Antitrypsin Deficiency PatientsWho this might be right forChronic Obstructive Pulmonary DiseaseAlpha 1-Antitrypsin Deficiency Schön Klinik Berchtesgadener Land 30
Not applicableLooking for participantsNCT06694012What this trial is testingOsaka Cardiometabolic Epidemiological Study: Ohtori Study Part 2Who this might be right forType 2 Diabetes Mellitus (T2DM)Type 2 DiabetesDiabetes Mellitus+16 more Osaka Metropolitan University 2,000
Early research (Phase 1)Looking for participantsNCT06049082What this trial is testingKB408 for the Treatment of Alpha-1 Antitrypsin DeficiencyWho this might be right forAlpha 1-Antitrypsin Deficiency Krystal Biotech, Inc. 15