14 completed, 4 terminated, 3 recruiting, 2 withdrawn. 8 Phase 2, 6 Phase 1, 3 Not applicable.
Follow Alpha-1 Antitrypsin Deficiency: new trials by email Literature Review of Alpha-1 Antitrypsin Deficiency trials Research Report on Alpha-1 Antitrypsin Deficiency
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin Deficiency PM647a - Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease | Phase 1 / Phase 2 | Not yet recruiting | Prime Medicine, Inc. | 54 | 2026-10-01 | 2026-10-21 |
| NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease Biological NTLA-3001 - Lung Disease, Pulmonary Disease | Phase 1 / Phase 2 | Withdrawn | Intellia Therapeutics | 2024-11-20 | 2025-01-17 | |
| A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD WVE-006 - Alpha-1 Antitrypsin Deficiency | Phase 1 / Phase 2 | Recruiting | Wave Life Sciences USA, Inc. | 48 | 2024-07-19 | 2026-10-19 |
| A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype VX-864 - Alpha-1 Antitrypsin Deficiency | Phase 2 | Terminated | Vertex Pharmaceuticals Incorporated | 14 | 2023-02-23 | 2025-09-08 |
| A Phase 1, First-in-human Study of VX-668 VX-668, Placebo - Alpha-1 Antitrypsin Deficiency | Phase 1 | Completed | Vertex Pharmaceuticals Incorporated | 114 | 2023-02-08 | 2024-03-15 |
| Respreeza® Self-administration and Learning Program (AmAREtTI Study) Alpha-1 Antitrypsin Deficiency | Completed | CSL Behring | 15 | 2019-10-18 | 2025-05-28 | |
| Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency INBRX-101/rhAAT-Fc - Alpha-1 Antitrypsin Deficiency, AATD | Phase 1 | Completed | Inhibrx Biosciences, Inc | 31 | 2019-07-19 | 2022-09-13 |
| Long-Term Follow-up Study of ADVM-043 No Intervention - Alpha-1 Antitrypsin Deficiency | Completed | Adverum Biotechnologies, Inc. | 6 | 2018-12-27 | 2024-09-20 | |
| Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency Aspirin, Placebo, Withdrawal from alpha1 antitrypsin replacement therapy - Alpha-1 Antitrypsin Deficiency, Emphysema | Phase 2 | Completed | Columbia University | 15 | 2017-01-01 | 2021-02-17 |
| Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels ARC-AAT Injection - Alpha-1 Antitrypsin Deficiency | Phase 2 | Withdrawn | Arrowhead Pharmaceuticals | 2016-10-01 | 2025-12-18 | |
| Environment Effect on Six-Minute Walk Test Performance six-minute walk study indoors, six-minute walk study outdoors - Chronic Obstructive Pulmonary Disease, Alpha-1 Antitrypsin Deficiency | Not applicable | Terminated | University of Florida | 27 | 2015-10-01 | 2019-08-22 |
| Microbioma in Sputa From COPD With Alpha-1 Antitrypsin Deficiency collection of sputum - Alpha-1 Antitrypsin Deficiency | Completed | Fondazione Salvatore Maugeri | 100 | 2015-09-01 | 2018-08-01 | |
| A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD) ARC-AAT Injection, Placebo, Diphenhydramine - Alpha-1 Antitrypsin Deficiency | Phase 1 | Terminated | Arrowhead Pharmaceuticals | 65 | 2015-02-01 | 2026-01-12 |
| Alpha-1 Carrier Genomics Study Genetic Sequencing - Alpha-1 Antitrypsin Deficiency, Emphysema | Completed | Medical University of South Carolina | 117 | 2015-02-01 | 2019-07-22 | |
| Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation" Kamada-AAT for Inhalation, 80mg, Placebo, Kamada-AAT for Inhalation, 160mg - Alpha-1 Antitrypsin Deficiency | Phase 2 | Completed | Kamada, Ltd. | 36 | 2014-04-01 | 2020-01-18 |
| Alpha-1 Antitrypsin Deficiency Adult Liver Study Liver Biopsy (Biopsy Group Only) - Alpha-1 Antitrypsin Deficiency | Active not recruiting | St. Louis University | 120 | 2013-12-01 | 2026-04-27 | |
| Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD) Abdominal ultrasound, History and physical, Intravenous catheter - Liver Fibrosis, Alpha-1 Antitrypsin Deficiency | Completed | University of Florida | 109 | 2013-10-01 | 2023-08-21 | |
| Ex-vivo Perfusion and Ventilation of Lungs Recovered From Non-Heart-Beating Donors to Assess Transplant Suitability Transplantation of lungs obtained from Non-Heart-Beating Donors (NHBDs) after ex-vivo perfusion w/ STEEN Solution™, STEEN Solution™ - Emphysema, Chronic Obstructive Pulmonary Disease (COPD) | Not applicable | Completed | University of North Carolina, Chapel Hill | 24 | 2013-09-01 | 2018-06-08 |
| Phase 2 Study of Ex-vivo Perfusion and Ventilation of Lungs to Assess Transplant Suitability Steen Solution™, Conventional Lung Transplant - Emphysema, Chronic Obstructive Pulmonary Disease (COPD) | Not applicable | Terminated | University of North Carolina, Chapel Hill | 11 | 2010-12-01 | 2018-07-13 |
| Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency rAAV1-CB-hAAT - Alpha-1 Antitrypsin Deficiency | Phase 2 | Completed | Beacon Therapeutics | 9 | 2010-06-01 | 2019-03-28 |
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alpha 1-Antitrypsin Deficiency (25) · Pulmonary Disease, Chronic Obstructive (9) · Emphysema (6) · Cystic Fibrosis (3) · Lung Diseases (3) · Bronchiectasis (2) · Fibrosis (2) · Hypertension, Pulmonary (2) · Pulmonary Fibrosis (2) · Sarcoidosis (2) · Liver Cirrhosis (1) · Smoking (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (25) · Digestive System Diseases (25) · Genetic Diseases, Inborn (25) · Liver Diseases (25) · Pathologic Processes (25) · Pathological Conditions, Signs and Symptoms (25) · Respiratory Tract Diseases (25) · Subcutaneous Emphysema (25) · Lung Diseases (22) · Emphysema (19) · Chronic Disease (9) · Disease Attributes (9) · Lung Diseases, Obstructive (9) · Infant, Newborn, Diseases (3) · Pancreatic Diseases (3)
Blood Specimen Collection (2) · Health Records, Personal (2) · Phlebotomy (2) · Restraint, Physical (2) · Acetaminophen (1) · Albuterol (1) · Aspirin (1) · Biopsy (1) · Diphenhydramine (1) · Fentanyl (1) · Inhalation (1) · Lidocaine (1) · Lorazepam (1) · Midazolam (1) · Ondansetron (1)
University of Florida (4) · Arrowhead Pharmaceuticals (2) · Medical University of South Carolina (2) · University of North Carolina, Chapel Hill (2) · Vertex Pharmaceuticals Incorporated (2) · Adverum Biotechnologies, Inc. (1) · Alpha-1 Foundation (1) · Beacon Therapeutics (1) · CSL Behring (1) · Columbia University (1) · Fondazione Salvatore Maugeri (1) · Inhibrx Biosciences, Inc (1) · Intellia Therapeutics (1) · Kamada, Ltd. (1) · National Heart, Lung, and Blood Institute (NHLBI) (1)
United States (17) · United Kingdom (4) · Germany (3) · Ireland (3) · New Zealand (3) · Australia (2) · Canada (2) · Finland (1) · France (1) · Italy (1) · Netherlands (1) · Puerto Rico (1) · Sweden (1)
Mark Brantly, MD (3) · Charlie Strange, MD (2) · Thomas M. Egan, MD, MSc. (2) · Andrew A Wilson, MD (1) · Bruce C. Trapnell, MD (1) · Carrie Aaron, MD (1) · Charlton Strange, MD (1) · David A. Brenner, MD (1) · Jeffrey Teckman, MD (1) · Karina Serban, M.D. (1) · Karina Serban, MD (1) · Mark Schluchter (1) · Noel G. McElvaney, MB, BCh, BAO (1) · Pantaleo Giannuzzi, M.D. (1)
PMID 15266208, PMID 16651497, PMID 17291167, PMID 21424325, PMID 21609134, PMID 24231351, PMID 28945476, PMID 29572094, PMID 30138687, PMID 30254761, PMID 34256305, PMID 39263815
1UM1HL113115-01A1 (1), 2009 (1), 2015 (1), 2016 (1), R01HL069877 (1), U54RR019498 (1)