16 completed. 16 Phase 1, 7 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 16 | Completed 16 |
| Phase 2 | 7 | Completed 7 |
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 Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1) WVE-006 - Alpha-1 Antitrypsin Deficiency (AATD) | Phase 1 | Completed | Wave Life Sciences USA, Inc. | 47 | 2023-11-14 | 2026-03-23 |
| 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 |
| A Phase 1, First-in-human Study of VX-634 VX-634, Placebo - Alpha 1-Antitrypsin Deficiency | Phase 1 | Completed | Vertex Pharmaceuticals Incorporated | 127 | 2022-10-13 | 2024-03-12 |
| 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 |
| Study of DCR-A1AT in Healthy Adult Volunteers belcesiran, Placebo - Alpha 1-Antitrypsin Deficiency | Phase 1 | Completed | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 30 | 2019-10-24 | 2024-11-06 |
| 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 |
| Study of ARO-AAT in Normal Adult Volunteers ARO-AAT Injection, Sterile Normal Saline (0.9% NaCl) - Alpha 1-Antitrypsin Deficiency | Phase 1 | Completed | Arrowhead Pharmaceuticals | 45 | 2018-03-12 | 2025-12-24 |
| Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency ADVM-043 - Alpha 1-Antitrypsin Deficiency | Phase 1 / Phase 2 | Completed | Adverum Biotechnologies, Inc. | 6 | 2017-11-28 | 2023-10-05 |
| Alpha-1 Antitrypsin (AAT) Enhances Islet Autograft Survival in Patients With Chronic Pancreatitis Prolastin-C, Placebo - Chronic Pancreatitis | Phase 1 / Phase 2 | Completed | Medical University of South Carolina | 48 | 2017-01-11 | 2023-06-29 |
| Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE) Alpha-1 MP - Alpha1-Antitrypsin Deficiency | Phase 1 / Phase 2 | Completed | Grifols Therapeutics LLC | 4 | 2016-07-29 | 2022-03-31 |
| Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participants With Alpha1-Antitrypsin Deficiency Alpha-1 MP - Alpha1-Antitrypsin Deficiency | Phase 1 / Phase 2 | Completed | Grifols Therapeutics LLC | 4 | 2016-07-29 | 2021-10-28 |
| The Effects of Alpha-1 Antitrypsin (AAT) on the Progression of Type 1 Diabetes Alpha 1-Antitrypsin (AAT, Aralast NP) - Diabetes, Type 1 Diabetes | Phase 1 | Completed | University of Colorado, Denver | 12 | 2010-10-01 | 2017-03-24 |
| Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency rAAV1-CB-hAAT - Alpha 1-Antitrypsin Deficiency | Phase 1 | Completed | University of Massachusetts, Worcester | 9 | 2006-02-01 | 2016-12-15 |
| Pharmacokinetic Study of ARALAST (Human Alpha1- PI) Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor, Dose of 60 mg/kg alpha1-proteinase inhibitor - Alpha 1-Antitrypsin Deficiency | Phase 1 | Completed | Baxalta now part of Shire | 25 | 2005-12-20 | 2021-05-13 |
| Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency Aerosolized, Recombinant Alpha 1-Antitrypsin - Alpha1-antitrypsin Deficiency | Phase 1 / Phase 2 | Completed | Baxalta now part of Shire | 15 | 2004-03-01 | 2021-05-05 |
| Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency Aerosolized, Recombinant Alpha 1-Antitrypsin - Alpha1-antitrypsin Deficiency | Phase 1 / Phase 2 | Completed | Baxalta now part of Shire | 40 | 2003-01-07 | 2021-05-05 |
alpha 1-Antitrypsin Deficiency (16) · Emphysema (2) · Lung Diseases (2) · Pulmonary Disease, Chronic Obstructive (2) · Diabetes Mellitus (1) · Diabetes Mellitus, Type 1 (1) · Digestive System Diseases (1) · Genetic Diseases, Inborn (1) · Liver Diseases (1) · Lung Diseases, Obstructive (1) · Pancreatitis, Chronic (1) · Pathologic Processes (1) · Pulmonary Emphysema (1) · Respiratory Tract Diseases (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Pathological Conditions, Signs and Symptoms (16) · Digestive System Diseases (15) · Genetic Diseases, Inborn (15) · Liver Diseases (15) · Pathologic Processes (15) · Respiratory Tract Diseases (15) · Emphysema (14) · Lung Diseases (14) · Chronic Disease (3) · Disease Attributes (3) · Autoimmune Diseases (1) · Endocrine System Diseases (1) · Glucose Metabolism Disorders (1)
Mp alpha1 receptor (2) · alpha 1-Antitrypsin (2) · 15-keto-17-phenyl-18,19,20-trinorprostaglandin F2 alpha-1-isopropyl ester (1) · Sodium Chloride (1)
Baxalta now part of Shire (3) · Grifols Therapeutics LLC (3) · Vertex Pharmaceuticals Incorporated (2) · Adverum Biotechnologies, Inc. (1) · Arrowhead Pharmaceuticals (1) · Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (1) · Inhibrx Biosciences, Inc (1) · Medical University of South Carolina (1) · University of Colorado, Denver (1) · University of Massachusetts, Worcester (1) · Wave Life Sciences USA, Inc. (1)
United States (10) · New Zealand (4) · Japan (2) · United Kingdom (2) · Australia (1) · Sweden (1)
Charlton Strange, MD (1) · Cynthia Caracta, MD (1) · Friedrich Kueppers, MD (1) · Hongjun Wang, Ph.D (1) · Igor Barjakatarevic, MD (1) · Kyle Hogarth, MD (1) · Mark Brantly, MD (1) · Mark L Brantly, MD (1) · Peter A Gottlieb, MD (1) · Terence R. Flotte, MD (1) · Thomas Bowman, MD (1) · Vasily Andrianov, MD (1)
PMID 34256305, PMID 22634621, PMID 30416054, PMID 35970714, PMID 36001294, PMID 38659255
R01DK105183 (1), R01HL069877 (1)