3 completed, 1 active not recruiting, 1 not yet recruiting, 1 recruiting. 6 Phase 2, 3 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 6 | Completed 3, Active not recruiting 1, Not yet recruiting 1, Recruiting 1 |
| Phase 1 | 3 | Active not recruiting 1, Not yet recruiting 1, Recruiting 1 |
Follow Wilson Disease: new trials by email Literature Review of Wilson Disease trials Research Report on Wilson Disease
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 (PM577) in Participants With Wilson Disease (WD) PM577a - Wilson Disease, Wilson's Disease | Phase 1 / Phase 2 | Recruiting | Prime Medicine, Inc. | 42 | 2026-09-01 | 2026-10-01 |
| Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD) GC310 - Wilson Disease | Phase 1 / Phase 2 | Not yet recruiting | GeneCradle Inc | 15 | 2025-10-01 | 2025-09-15 |
| A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease UX701, Standard of Care (SOC) - Wilson Disease | Phase 1 / Phase 2 | Active not recruiting | Ultragenyx Pharmaceutical Inc | 82 | 2021-09-27 | 2026-09-25 |
| Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840 Bis-Choline Tetrathiomolybdate - Wilson Disease | Phase 2 | Completed | Alexion Pharmaceuticals, Inc. | 31 | 2020-12-02 | 2024-10-18 |
| Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 ALXN1840 - Wilson Disease | Phase 2 | Completed | Alexion Pharmaceuticals, Inc. | 9 | 2020-09-07 | 2024-06-24 |
| Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients ALXN1840 - Wilson Disease | Phase 2 | Completed | Alexion Pharmaceuticals, Inc. | 29 | 2014-11-24 | 2021-09-29 |
Hepatolenticular Degeneration (6)
Basal Ganglia Diseases (6) · Brain Diseases (6) · Brain Diseases, Metabolic (6) · Brain Diseases, Metabolic, Inborn (6) · Central Nervous System Diseases (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Digestive System Diseases (6) · Genetic Diseases, Inborn (6) · Heredodegenerative Disorders, Nervous System (6) · Liver Diseases (6) · Metabolic Diseases (6) · Metabolism, Inborn Errors (6) · Metal Metabolism, Inborn Errors (6) · Movement Disorders (6) · Nervous System Diseases (6)
Standard of Care (1) · tetrathiomolybdate (1)
Alexion Pharmaceuticals, Inc. (3) · GeneCradle Inc (1) · Prime Medicine, Inc. (1) · Ultragenyx Pharmaceutical Inc (1)
United States (5) · United Kingdom (4) · New Zealand (3) · Spain (2) · Austria (1) · Canada (1) · China (1) · Denmark (1) · Germany (1) · Poland (1) · Portugal (1) · Russia (1) · Singapore (1)
Eugene S. Swenson, MD, PhD (2) · Eugene Swenson, MD, PhD (1) · Peter Ksenuk, MD (1)
2019 (1), 2025 (1)