3 completed, 1 not yet recruiting, 1 recruiting, 1 terminated. 4 Phase 1, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 4 | Completed 1, Not yet recruiting 1, Terminated 1, Withdrawn 1 |
| Phase 2 | 3 | Not yet recruiting 1, Terminated 1, Withdrawn 1 |
Follow this condition Literature Review of AATD trials Research Report on AATD
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 |
| 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Deficiency-Associated Lung Disease Biological NTLA-3001 - Lung Disease, Pulmonary Disease | Phase 1 / Phase 2 | Withdrawn | Intellia Therapeutics | 2024-11-20 | 2025-01-17 | |
| 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 |
| 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 | |
| Genomic Research in Alpha-1 Antitrypsin Deficiency Alpha 1 Antitrypsin Deficiency, AATD | Completed | University of Pittsburgh | 140 | 2013-05-01 | 2016-01-12 | |
| Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs History and physical exam., Blood draw., Pulmonary function testing. - Alpha-1 Antitrypsin Deficiency, AAT Deficiency | Recruiting | University of Florida | 220 | 2007-08-09 | 2025-11-06 |
alpha 1-Antitrypsin Deficiency (7) · Cystic Fibrosis (1) · Fibrosis (1) · Liver Cirrhosis (1) · Lung Diseases (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Digestive System Diseases (7) · Emphysema (7) · Genetic Diseases, Inborn (7) · Liver Diseases (7) · Pathologic Processes (7) · Pathological Conditions, Signs and Symptoms (7) · Respiratory Tract Diseases (7) · Subcutaneous Emphysema (7) · Lung Diseases (6) · Infant, Newborn, Diseases (1) · Pancreatic Diseases (1)
Blood Specimen Collection (2) · Health Records, Personal (2) · Phlebotomy (2) · Restraint, Physical (2) · Acetaminophen (1) · Albuterol (1) · Biopsy (1) · Fentanyl (1) · Lidocaine (1) · Lorazepam (1) · Midazolam (1) · Ondansetron (1) · Procaterol (1) · Respiratory Physiological Phenomena (1) · Surveys and Questionnaires (1)
University of Florida (2) · Inhibrx Biosciences, Inc (1) · Intellia Therapeutics (1) · Korro Bio, Inc. (1) · Prime Medicine, Inc. (1) · University of Pittsburgh (1)
United States (4) · New Zealand (3) · Australia (1) · United Kingdom (1)
Karina Serban, MD (1) · Mark Brantly, MD (1) · Michael Becich, MD, PhD (1) · Naftali Kaminski, MD (1) · Stephen Wisniewski, PhD (1) · Vasily Andrianov, MD (1)
U01HL112707 (1)