24 completed, 6 terminated, 5 withdrawn, 4 recruiting. 42 Phase 2, 15 Phase 1, 2 Phase 3.
Follow Subcutaneous Emphysema: new trials by email Literature Review of Subcutaneous Emphysema trials Research Report on Subcutaneous Emphysema
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 | Recruiting | CRISPR Therapeutics | 68 | 2026-12-01 | 2026-11-13 |
| 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 |
| 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 | 2026-11-12 |
| 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Deficiency-Associated Lung Disease Biological NTLA-3001 - Lung Disease, Pulmonary Disease | Phase 1 / Phase 2 | Withdrawn | Intellia Therapeutics | 2024-11-20 | 2025-01-17 | |
| 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 |
| Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema SAR447537 - Alpha 1-Antitrypsin Deficiency, Emphysema | Phase 2 | Active not recruiting | Sanofi | 185 | 2024-06-20 | 2026-04-13 |
| A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) BEAM-302 - Alpha 1-Antitrypsin Deficiency | Phase 1 / Phase 2 | Recruiting | Beam Therapeutics Inc. | 192 | 2024-06-19 | 2026-10-05 |
| Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema SAR447537, Zemaira - Alpha 1-Antitrypsin Deficiency, Emphysema | Phase 2 | Completed | Sanofi | 99 | 2023-10-12 | 2025-08-11 |
| 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 Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency Alpha-1 15%, Liquid Alpha1-Proteinase Inhibitor (Human) - Alpha1-Antitrypsin Deficiency | Phase 1 / Phase 2 | Completed | Grifols Therapeutics LLC | 17 | 2021-12-16 | 2026-10-08 |
| An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD) Belcesiran - Alpha 1-Antitrypsin Deficiency | Phase 2 | Withdrawn | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 2021-12-01 | 2024-09-19 | |
| A Study of Belcesiran in Patients With AATLD Belcesiran, Placebo - Alpha 1-Antitrypsin Deficiency | Phase 2 | Terminated | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 16 | 2021-02-12 | 2026-04-07 |
| Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype VX-864, Placebo - Alpha1-Antitrypsin Deficiency | Phase 2 | Completed | Vertex Pharmaceuticals Incorporated | 44 | 2020-07-24 | 2022-05-26 |
| Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype VX-814, Placebo - Alpha 1-Antitrypsin Deficiency | Phase 2 | Terminated | Vertex Pharmaceuticals Incorporated | 48 | 2020-01-13 | 2022-02-02 |
| Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) ARO-AAT - Alpha 1-Antitrypsin Deficiency | Phase 2 | Completed | Arrowhead Pharmaceuticals | 16 | 2019-10-31 | 2025-10-15 |
| Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT) Fazisiran Injection, Placebo - Alpha 1-Antitrypsin Deficiency | Phase 2 | Completed | Arrowhead Pharmaceuticals | 40 | 2019-08-07 | 2025-11-04 |
| Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency Alvelestat (MPH966), Placebo - Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations | Phase 2 | Completed | University of Alabama at Birmingham | 63 | 2019-04-08 | 2024-08-20 |
| A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo. Placebo Oral Tablet, Alvelestat oral tablet - dose 1, Alvelestat oral tablet - dose 2 - Alpha 1-Antitrypsin Deficiency, Emphysema | Phase 2 | Completed | Mereo BioPharma | 99 | 2018-10-29 | 2022-04-14 |
| Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency OctaAlpha1, Glassia - Alpha 1-Antitrypsin Deficiency | Phase 2 | Withdrawn | Octapharma | 2018-07-01 | 2018-04-10 |
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alpha 1-Antitrypsin Deficiency (42) · Emphysema (10) · Pulmonary Disease, Chronic Obstructive (6) · Lung Diseases (5) · Liver Diseases (3) · Diabetes Mellitus, Type 1 (2) · Pulmonary Emphysema (2) · Congenital lobar emphysema (1) · Cystic Fibrosis (1) · Diabetes Mellitus (1) · Digestive System Diseases (1) · Genetic Diseases, Inborn (1) · Inflammation (1) · Liver Cirrhosis (1) · Lung Diseases, Obstructive (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (42) · Pathological Conditions, Signs and Symptoms (42) · Digestive System Diseases (41) · Genetic Diseases, Inborn (41) · Pathologic Processes (41) · Respiratory Tract Diseases (41) · Liver Diseases (39) · Lung Diseases (37) · Emphysema (32) · Chronic Disease (8) · Disease Attributes (8) · Lung Diseases, Obstructive (6) · Autoimmune Diseases (2) · Endocrine System Diseases (2)
alpha 1-Antitrypsin (10) · Mp alpha1 receptor (4) · Inhalation (2) · 15-keto-17-phenyl-18,19,20-trinorprostaglandin F2 alpha-1-isopropyl ester (1) · 4-phenylbutylamine (1) · Albumins (1) · Aspirin (1) · Carbamazepine (1) · Respreeza (1) · Sodium Chloride (1)
Grifols Therapeutics LLC (7) · Arrowhead Pharmaceuticals (3) · Kamada, Ltd. (3) · Vertex Pharmaceuticals Incorporated (3) · Baxalta now part of Shire (2) · Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (2) · Sanofi (2) · Adverum Biotechnologies, Inc. (1) · Beacon Therapeutics (1) · Beam Therapeutics Inc. (1) · CRISPR Therapeutics (1) · Columbia University (1) · Gerard Turino (1) · Intellia Therapeutics (1) · Korro Bio, Inc. (1)
United States (28) · United Kingdom (12) · Ireland (9) · Sweden (9) · Australia (8) · Canada (8) · Germany (8) · New Zealand (8) · Denmark (5) · Netherlands (5) · Spain (5) · Poland (3) · Austria (2) · Belgium (2) · Italy (2)
Friedrich Kueppers, MD (2) · Mark Brantly, MD (2) · Anne-Sophie Sejling, MD (1) · Asger Dirksen, MD PHD (1) · Bruce C. Trapnell, MD (1) · Carrie Aaron, MD (1) · Charlie Strange, MD (1) · Charlton Strange, MD (1) · David H. Perlmutter, M.D. (1) · Gerard M Turino, MD (1) · Gordon Weir, MD (1) · Hongjun Wang, Ph.D (1)
PMID 34256305, PMID 19196813, PMID 19251783, PMID 19483042, PMID 19678952, PMID 20920370, PMID 21609134, PMID 21617168, PMID 23226015, PMID 23664631, PMID 23822603, PMID 24231351, PMID 30416054, PMID 30965011, PMID 31467115, PMID 33244872, PMID 35748699, PMID 35970714, PMID 38659255, PMID 38964420
2018 (2), 2019 (2), 2023 (2), 2009 (1), 2016 (1), 4UH3TR002450-02 (1), R01DK105183 (1), R01HL069877 (1), R21DK092567 (1)