alpha 1-antitrypsin deficiency
alpha 1-antitrypsin deficiency
Explore linked clinical trials. Standard reference: MONDO:001328234 results ยท Page 1 of 2
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Role of Genetic Factors in the Development of Lung Disease | Congenital 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 sclerosis | Recruiting | Not stated | 2026-10-08 | NCT00001532 | |
| 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 disorder | Recruiting | Phase 1 | 2026-10-06 | NCT07431112 | |
| 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 disorder | Recruiting | Phase 1 / Phase 2 | 2026-10-05 | NCT06389877 | |
| 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 disorder | Not yet recruiting | Phase 1 / Phase 2 | 2026-09-30 | NCT07850076 | |
| Gene Therapy for Alpha 1- Antitrypsin Deficiency | 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 disorder | Recruiting | Phase 1 | 2026-09-25 | NCT06996756 | |
| 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 disorder | Recruiting | Phase 1 / Phase 2 | 2026-09-24 | NCT07227207 | |
| Alpha-1 Research Registry | 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 disorder | Recruiting | Not stated | 2026-08-19 | NCT04157049 | |
| 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 disorder | Recruiting | Phase 3 | 2026-08-11 | NCT07555483 | |
| A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems | 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 disorder | Recruiting | Not stated | 2026-07-24 | NCT06512454 | |
| Development of a Prediction Score for the Occurrence of Death or Lung Transplantation in Patients With Emphysema Secondary to Alpha-1-anti-tripsin Deficiency | 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 disorder | Recruiting | Not stated | 2026-07-20 | NCT07715617 | |
| IBS-Oriented CDED Versus Low FODMAP Diet for Irritable Bowel Syndrome | Congenital, 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 disorder | Not yet recruiting | Not applicable | 2026-07-16 | NCT07707635 | |
| Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency | 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 disorder | Enrolling by invitation | Phase 3 | 2026-06-23 | NCT02796937 | |
| 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 disorder | Not yet recruiting | Not stated | 2026-06-10 | NCT07639996 | |
| 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 disorder | Not yet recruiting | Phase 4 | 2026-05-15 | NCT07326592 | |
| 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 | 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 disorder | Recruiting | Phase 3 | 2026-05-13 | NCT05677971 | |
| Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants | 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 disorder | Active, not recruiting | Phase 1 | 2026-05-06 | NCT06738017 | |
| Alpha-1 Antitrypsin Deficiency Adult Liver Study | Congenital, 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 disorder | Active, not recruiting | Not stated | 2026-04-27 | NCT02014415 | |
| A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ 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 disorder | Active, not recruiting | Phase 1 / Phase 2 | 2026-04-14 | NCT06405633 | |
| Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema | 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 disorder | Active, not recruiting | Phase 2 | 2026-04-13 | NCT05897424 | |
| 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 disorder | Recruiting | Phase 3 | 2026-03-05 | NCT06165341 | |
| 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 disorder | Recruiting | Not stated | 2026-01-16 | NCT06505603 | |
| Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of 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 disorder | Active, not recruiting | Not stated | 2026-01-16 | NCT05297812 | |
| Alpha-1 Foundation DNA and Tissue Bank | 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 disorder | Enrolling by invitation | Not stated | 2026-01-15 | NCT00884455 | |
| Prevalence of Alpha-1 Antitrypsin Deficiency in Non-Cirrhotic Liver Cancer | 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 disorder | Not yet recruiting | Not stated | 2026-01-05 | NCT07145385 | |
| The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study | 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 disorder | Recruiting | Not stated | 2025-11-24 | NCT00500123 |