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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Role of Genetic Factors in the Development of Lung DiseaseCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Fibrosis, Hemic and Lymphatic Diseases, Hypersensitivity, Delayed, Hypersensitivity, Immediate, Infant, Newborn, Diseases, Malformations of Cortical Development, Group I, Neoplasms, Multiple Primary, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Respiratory Hypersensitivity, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, asthma, bronchial disorder, central nervous system malformation, cerebral cortical dysplasia, cystic fibrosis, hamartoma, hereditary disease, hereditary neoplastic syndrome, immune system disorder, inherited neurodegenerative disorder, interstitial lung disease, liver disorder, lung disorder, lymphatic system disorder, lymphoproliferative syndrome, neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, obstructive lung disease, pancreas disorder, pulmonary emphysema, pulmonary fibrosis, pulmonary sarcoidosis, respiratory system disorder, sarcoidosis, tuberous sclerosisRecruitingNot stated2026-10-08NCT00001532
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderRecruitingPhase 12026-10-06NCT07431112
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderRecruitingPhase 1 / Phase 22026-10-05NCT06389877
A Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460 in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderNot yet recruitingPhase 1 / Phase 22026-09-30NCT07850076
Gene Therapy for Alpha 1- Antitrypsin DeficiencyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderRecruitingPhase 12026-09-25NCT06996756
A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderRecruitingPhase 1 / Phase 22026-09-24NCT07227207
Alpha-1 Research RegistryChronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, chronic obstructive pulmonary disease, hereditary disease, liver disorder, lung disorder, obstructive lung disease, pulmonary emphysema, respiratory system disorderRecruitingNot stated2026-08-19NCT04157049
A 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)Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, chronic obstructive pulmonary disease, hereditary disease, liver disorder, lung disorder, obstructive lung disease, pulmonary emphysema, respiratory system disorderRecruitingPhase 32026-08-11NCT07555483
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver ProblemsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderRecruitingNot stated2026-07-24NCT06512454
Development of a Prediction Score for the Occurrence of Death or Lung Transplantation in Patients With Emphysema Secondary to Alpha-1-anti-tripsin DeficiencyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderRecruitingNot stated2026-07-20NCT07715617
IBS-Oriented CDED Versus Low FODMAP Diet for Irritable Bowel SyndromeCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, colonic disorder, digestive system disorder, functional colonic disease, hereditary disease, intestinal disorder, irritable bowel syndrome, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderNot yet recruitingNot applicable2026-07-16NCT07707635
Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin DeficiencyChronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, chronic obstructive pulmonary disease, hereditary disease, liver disorder, lung disorder, obstructive lung disease, pulmonary emphysema, respiratory system disorderEnrolling by invitationPhase 32026-06-23NCT02796937
Natural Course and Molecular Basis of Alpha 1- Antitrypsin Deficiency-associated Liver Disease.Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderNot yet recruitingNot stated2026-06-10NCT07639996
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)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderNot yet recruitingPhase 42026-05-15NCT07326592
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 ProteinCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderRecruitingPhase 32026-05-13NCT05677971
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult ParticipantsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderActive, not recruitingPhase 12026-05-06NCT06738017
Alpha-1 Antitrypsin Deficiency Adult Liver StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Fibrosis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderActive, not recruitingNot stated2026-04-27NCT02014415
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATDCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderActive, not recruitingPhase 1 / Phase 22026-04-14NCT06405633
Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency EmphysemaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderActive, not recruitingPhase 22026-04-13NCT05897424
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)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderRecruitingPhase 32026-03-05NCT06165341
PiMZ Longitudinal Cohort (PiMZ Logic)Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, chronic obstructive pulmonary disease, hereditary disease, liver disorder, lung disorder, obstructive lung disease, pulmonary emphysema, respiratory system disorderRecruitingNot stated2026-01-16NCT06505603
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderActive, not recruitingNot stated2026-01-16NCT05297812
Alpha-1 Foundation DNA and Tissue BankCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderEnrolling by invitationNot stated2026-01-15NCT00884455
Prevalence of Alpha-1 Antitrypsin Deficiency in Non-Cirrhotic Liver CancerCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderNot yet recruitingNot stated2026-01-05NCT07145385
The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderRecruitingNot stated2025-11-24NCT00500123