16 completed, 6 recruiting, 5 terminated, 2 not yet recruiting. 32 Phase 1, 15 Phase 2.
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 | Not yet recruiting | CRISPR Therapeutics | 68 | 2026-10-31 | 2026-09-30 |
| 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 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 | 2026-10-23 |
| 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 |
| 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Deficiency-Associated Lung Disease Biological NTLA-3001 - Lung Disease, Pulmonary Disease | Phase 1 / Phase 2 | Withdrawn | Intellia Therapeutics | 2024-11-20 | 2025-01-17 | |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 |
| 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 |
| A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency KB408 (Nebulization) - Alpha 1-Antitrypsin Deficiency | Phase 1 | Recruiting | Krystal Biotech, Inc. | 15 | 2024-02-15 | 2025-07-22 |
| 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 |
| A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiXZ Subjects ZF874, Placebo - Alpha1 Anti-Trypsin Deficiency | Phase 1 | Terminated | Z Factor Limited | 69 | 2020-08-03 | 2022-09-19 |
| 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 |
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alpha 1-Antitrypsin Deficiency (32) · Emphysema (4) · Lung Diseases (4) · Diabetes Mellitus (2) · Diabetes Mellitus, Type 1 (2) · Genetic Diseases, Inborn (2) · Liver Diseases (2) · Pathologic Processes (2) · Pulmonary Disease, Chronic Obstructive (2) · Respiratory Tract Diseases (2) · Congenital Abnormalities (1) · Digestive System Diseases (1) · Infant, Newborn, Diseases (1) · Inflammation (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (32) · Digestive System Diseases (31) · Pathological Conditions, Signs and Symptoms (31) · Genetic Diseases, Inborn (30) · Liver Diseases (30) · Pathologic Processes (30) · Respiratory Tract Diseases (30) · Emphysema (28) · Lung Diseases (28) · Chronic Disease (3) · Disease Attributes (3) · Autoimmune Diseases (2) · Endocrine System Diseases (2) · Glucose Metabolism Disorders (2)
alpha 1-Antitrypsin (4) · Mp alpha1 receptor (2) · 15-keto-17-phenyl-18,19,20-trinorprostaglandin F2 alpha-1-isopropyl ester (1) · Diphenhydramine (1) · Sodium Chloride (1)
Baxalta now part of Shire (3) · Grifols Therapeutics LLC (3) · Arrowhead Pharmaceuticals (2) · Vertex Pharmaceuticals Incorporated (2) · Wave Life Sciences USA, Inc. (2) · AIRNA Corporation (1) · Adverum Biotechnologies, Inc. (1) · Alnylam Pharmaceuticals (1) · Beam Therapeutics Inc. (1) · BioMarin Pharmaceutical (1) · CRISPR Therapeutics (1) · Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (1) · Inhibrx Biosciences, Inc (1) · Intellia Therapeutics (1) · Korro Bio, Inc. (1)
United States (16) · United Kingdom (10) · New Zealand (9) · Australia (7) · Netherlands (4) · Germany (3) · Sweden (3) · Japan (2) · Canada (1) · Finland (1) · Georgia (1) · Ireland (1) · Portugal (1) · Spain (1)
Charlton Strange, MD (1) · Cynthia Caracta, MD (1) · David Sweet, MD, PhD (1) · Friedrich Kueppers, MD (1) · Giuseppe Fiore, MSc MD (1) · Gordon Weir, MD (1) · Hongjun Wang, Ph.D (1) · Igor Barjakatarevic, MD (1) · Kyle Hogarth, MD (1) · Malcolm Boyce, BSc MD FRCP FFPM (1) · Mark Brantly, MD (1)
PMID 22634621, PMID 34256305, PMID 29572094, PMID 30416054, PMID 35970714, PMID 36001294, PMID 38659255
2015 (1), R01DK105183 (1), R01HL069877 (1), R61HL169190 (1)