66 completed, 20 recruiting, 18 terminated, 10 unknown. 42 Phase 2, 32 Phase 1, 13 Phase 3, 12 Not applicable, 9 Phase 4.
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 Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460 in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD) CTX460 - Alpha-1 Antitrypsin Deficiency (AATD) | Phase 1 / Phase 2 | Not yet recruiting | CRISPR Therapeutics | 68 | 2026-10-31 | 2026-09-30 |
| Natural Course and Molecular Basis of Alpha 1- Antitrypsin Deficiency-associated Liver Disease. Multi-omic spatial analysis - Alpha 1-antitrypsin Deficiency (AATD) | Not yet recruiting | Assiut University | 45 | 2026-10-01 | 2026-06-10 | |
| 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 |
| 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) CE1226 - Alpha1 Antitrypsin Deficiency, Alpha1-Proteinase Inhibitor Deficiency | Phase 4 | Recruiting | CSL Behring | 270 | 2026-09-29 | 2026-10-16 |
| IBS-Oriented CDED Versus Low FODMAP Diet for Irritable Bowel Syndrome Modified Crohn's Disease Exclusion Diet (CDED), Low FODMAP Diet - Irritable Bowel Syndrome (IBS) | Not applicable | Not yet recruiting | Ariel University | 150 | 2026-07-07 | 2026-07-16 |
| 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) Alpha-1 15%, Liquid Alpha1-PI - Alpha 1 Antitrypsin Deficiency | Phase 3 | Recruiting | Grifols Therapeutics LLC | 40 | 2026-05-05 | 2026-10-22 |
| A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD) TSRA-196 - Alpha-1 Antitrypsin Deficiency (AATD) | Phase 1 / Phase 2 | Recruiting | Tessera Therapeutics, Inc. | 72 | 2026-04-21 | 2026-09-24 |
| A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD) AIR-001 SAD dose level 1, AIR-001 SAD dose level 2, AIR-001 SAD dose level 3 - Alpha 1 Antitrypsin Deficiency | Phase 1 | Recruiting | AIRNA Corporation | 54 | 2026-03-17 | 2026-10-06 |
| Development of a Prediction Score for the Occurrence of Death or Lung Transplantation in Patients With Emphysema Secondary to Alpha-1-anti-tripsin Deficiency Alpha-1-antitrypsin Deficiency, Emphysema | Recruiting | University Hospital, Bordeaux | 230 | 2026-02-16 | 2026-07-20 | |
| Prevalence of Alpha-1 Antitrypsin Deficiency in Non-Cirrhotic Liver Cancer multiplex digital PCR - Retrospective Cohort of Patients Diagnosed With HCC on Non-Cirrhotic Liver | Not yet recruiting | University Hospital, Bordeaux | 71 | 2026-01-01 | 2026-01-05 | |
| Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency 20-valent pneumococcal conjugate vaccine - Alpha 1-Antitrypsin, COPD | Phase 4 | Recruiting | University of Alabama at Birmingham | 30 | 2025-09-03 | 2025-11-10 |
| Screening for Alpha-1 Antitrypsin Deficiency in Patients With Airway Obstruction Blood Sampling - Alpha 1-antitrypsin Deficiency (AATD) | Recruiting | Muğla Sıtkı Koçman University | 734 | 2025-08-06 | 2025-09-10 | |
| Study of YOLT-202 in the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) YOLT-202 - Alpha-1 Antitrypsin Deficiency (AATD) | Early Phase 1 | Active not recruiting | RenJi Hospital | 18 | 2025-06-18 | 2025-09-26 |
| PiMZ Longitudinal Cohort (PiMZ Logic) Alpha 1-Antitrypsin Deficiency, Emphysema or COPD | Recruiting | Columbia University | 80 | 2025-05-30 | 2026-01-16 | |
| Gene Therapy for Alpha 1- Antitrypsin Deficiency AAV8hAAT(AVL) - Alpha 1-Antitrypsin Deficiency | Phase 1 | Recruiting | Weill Medical College of Cornell University | 16 | 2025-02-26 | 2026-10-16 |
| Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants BMN 349, Placebo - Alpha 1-Antitrypsin Deficiency | Phase 1 | Active not recruiting | BioMarin Pharmaceutical | 6 | 2025-02-21 | 2026-05-06 |
| Preparation of IPSC for Cell Gene Editing for the Treatment of AATD iPSC generation - Alpha1-antitrypsin Deficiency | Not applicable | Enrolling by invitation | Fondazione IRCCS Policlinico San Matteo di Pavia | 3 | 2025-01-15 | 2025-03-24 |
| A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110 KRRO-110 - Alpha-1 Anti-trypsin Deficiency, AATD | Phase 1 / Phase 2 | Terminated | Korro Bio, Inc. | 42 | 2025-01-13 | 2026-04-17 |
| 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 | |
| Effect Of Pulmonary Rehabilitation in Patients With Alpha-1 Antitrypsin Deficiency Pulmonary Rehabilitation - Alpha-1 Antitrypsin Deficiency (AATD) | Completed | University of Parma | 50 | 2024-11-15 | 2025-11-21 |
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Pulmonary Disease, Chronic Obstructive (30) · Emphysema (29) · Lung Diseases (10) · Genetic Diseases, Inborn (8) · Cystic Fibrosis (7) · Pulmonary Emphysema (7) · Liver Diseases (5) · Pulmonary Fibrosis (5) · Bronchiectasis (4) · Diabetes Mellitus, Type 1 (4) · Diabetes Mellitus (3) · Inflammation (3) · Liver Cirrhosis (3) · Lung Diseases, Obstructive (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (139) · Digestive System Diseases (138) · Pathological Conditions, Signs and Symptoms (138) · Pathologic Processes (136) · Respiratory Tract Diseases (136) · Subcutaneous Emphysema (136) · Liver Diseases (134) · Genetic Diseases, Inborn (131) · Lung Diseases (129) · Emphysema (110) · Disease Attributes (38) · Chronic Disease (37) · Lung Diseases, Obstructive (34) · Immune System Diseases (11) · Infant, Newborn, Diseases (8)
alpha 1-Antitrypsin (26) · Mp alpha1 receptor (7) · Blood Specimen Collection (3) · Inhalation (3) · Phlebotomy (3) · Respreeza (3) · Sodium Chloride (3) · 15-keto-17-phenyl-18,19,20-trinorprostaglandin F2 alpha-1-isopropyl ester (2) · Exercise (2) · Health Records, Personal (2) · Restraint, Physical (2) · 4-phenylbutylamine (1) · Acetaminophen (1) · Albumins (1) · Albuterol (1)
Grifols Therapeutics LLC (12) · Baxalta now part of Shire (8) · University of Florida (6) · Arrowhead Pharmaceuticals (5) · Takeda (5) · Vertex Pharmaceuticals Incorporated (5) · CSL Behring (4) · Columbia University (4) · Kamada, Ltd. (4) · Medical University of South Carolina (4) · National Heart, Lung, and Blood Institute (NHLBI) (4) · Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (3) · Schön Klinik Berchtesgadener Land (3) · Adverum Biotechnologies, Inc. (2) · Alpha-1 Foundation (2)
United States (80) · United Kingdom (26) · Germany (24) · Canada (19) · Sweden (18) · Australia (17) · New Zealand (16) · Ireland (15) · Denmark (13) · Spain (12) · Poland (11) · Netherlands (10) · France (9) · Portugal (7) · Austria (6)
Mark Brantly, MD (5) · Charlie Strange, MD (4) · Terence R. Flotte, MD (3) · Charlton Strange, MD (2) · Ed Doo, MD (2) · Friedrich Kueppers, MD (2) · Gordon Weir, MD (2) · John Magee, MD (2) · Karina Serban, MD (2) · Kim Hanna, MSc (2) · Lisa Henn, PhD (2) · Mark L Brantly, MD (2)
PMID 34256305, PMID 21424325, PMID 22634621, PMID 26026936, PMID 30237305, PMID 36001294, PMID 10680096, PMID 10988213, PMID 14760160, PMID 14985567, PMID 15266208, PMID 15822045, PMID 16403617, PMID 16651497, PMID 16760357, PMID 17291167, PMID 19196813, PMID 19251783, PMID 19483042, PMID 19678952, PMID 20054436, PMID 20920295, PMID 20920370, PMID 21609134, PMID 21617168, PMID 2185272, PMID 22672739, PMID 22835581, PMID 23226015, PMID 23261311, PMID 23664631, PMID 23822603, PMID 24231351, PMID 24525449, PMID 25651489, PMID 25727857, PMID 25938292, PMID 26153726, PMID 27916480, PMID 28890438, PMID 2890373, PMID 28945476, PMID 29572094, PMID 30138687, PMID 30254761, PMID 30291925, PMID 30416054, PMID 30965011, PMID 31121167, PMID 31467115
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