21 completed, 12 recruiting, 5 active not recruiting, 4 unknown. 10 Not applicable, 6 Phase 1, 6 Phase 2, 4 Phase 3, 2 Phase 4.
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 |
| Description of Renal Involvement in Wilson's Disease full renal workup - Wilson Disease | Recruiting | Fondation Ophtalmologique Adolphe de Rothschild | 150 | 2026-07-22 | 2026-10-01 | |
| Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study Wilson Disease | Recruiting | Yale University | 30 | 2026-01-08 | 2026-03-05 | |
| Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial Wilson Disease | Active not recruiting | Prime Medicine, Inc. | 30 | 2025-12-29 | 2026-05-12 | |
| Wilson's Disease Treated With D-Penicillamine: Characterization of Skin Damage Secondary to Treatment by Measuring Skin Elasticity Preparation of forearm molds with SILFLO® silicone - Wilson Disease, D-Penicillamine | Not applicable | Recruiting | Centre Hospitalier Universitaire de Saint Etienne | 120 | 2025-11-28 | 2026-01-23 |
| Subclinical Myocardial Dysfunction in Children With Wilson's Disease Echocardiography, Electrocardiography, N-terminal pro b- type natriuretic peptide - Wilson's Disease, Myocardial Dysfunction | Active not recruiting | Hebatullah Fawzy | 72 | 2025-10-01 | 2026-08-14 | |
| 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 |
| Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT Wilson Disease | Enrolling by invitation | Thomas Damgaard Sandahl | 5 | 2025-07-08 | 2025-09-08 | |
| Circadian Variation of Urinary Copper Excretion in Wilson Disease Patients urine and blood test - Wilson Disease | Recruiting | Hospices Civils de Lyon | 30 | 2025-01-10 | 2026-01-15 | |
| Daily Versus Alternate Day Plasma Exchange in Wilson Disease With Acute Liver Failure in Children Plasma Exchange, Standard Medical Treatment - Acute Liver Failure, Wilson Disease | Not applicable | Not yet recruiting | Institute of Liver and Biliary Sciences, India | 20 | 2024-11-10 | 2024-11-21 |
| An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease MWAV201 - Wilson Disease | Not applicable | Active not recruiting | Xinhua Hospital, Shanghai Jiao Tong University School of Medicine | 9 | 2024-10-28 | 2026-08-12 |
| A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease LY-M003 - Wilson Disease | Early Phase 1 | Recruiting | Chaohui Yu | 18 | 2024-09-24 | 2026-01-23 |
| The Exploratory Handheld Optical Coherence Tomography Study in Wilson's Disease Diagnostic procedures - Wilson Disease | Not applicable | Completed | Royal Surrey County Hospital NHS Foundation Trust | 10 | 2024-08-02 | 2025-07-01 |
| Institutional Registry of Rare Diseases Rare Diseases, Amyloidosis | Recruiting | Hospital Italiano de Buenos Aires | 380 | 2024-07-01 | 2026-01-14 | |
| Evaluation of Cognitive Dysfunction and Psychiatric Comorbidities Wilson Disease | Unknown | Assiut University | 40 | 2023-12-15 | 2023-12-21 | |
| Real World Evidence Study in Subjects With Wilson's Disease NCC-Sp Assay - Wilson Disease | Completed | Orphalan | 50 | 2023-06-28 | 2026-04-06 | |
| Oral Health and Wilson's Disease: SOMAWI Saliva samples, Questionnaire OHIP-14, Thorough dental examination - Wilson Disease | Recruiting | Fondation Ophtalmologique Adolphe de Rothschild | 150 | 2023-05-17 | 2026-10-06 | |
| rTMS in Wilson Disease Dysarthria rTMS, Sham stimulation - Wilson Disease | Not applicable | Completed | Assistance Publique - Hôpitaux de Paris | 18 | 2023-01-23 | 2024-02-28 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Spanish Wilson Disease Registry Wilson Disease | Recruiting | Asociación Española para el Estudio del Hígado | 600 | 2021-12-02 | 2024-06-20 |
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Hepatolenticular Degeneration (49) · Metabolism, Inborn Errors (4) · Ornithine Carbamoyltransferase Deficiency Disease (3) · Rare Diseases (3) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (2) · Acidemia, isovaleric (2) · Adrenal Hyperplasia, Congenital (2) · Anemia, Sickle Cell (2) · Argininosuccinic Aciduria (2) · Ataxia with vitamin E deficiency (2) · Basal ganglia disease, biotin-responsive (2) · Beta ketothiolase deficiency (2) · Biotinidase Deficiency (2) · Brown-Vialetto-Van Laere syndrome (2) · Carbamoyl-Phosphate Synthase I Deficiency Disease (2)
Basal Ganglia Diseases (49) · Brain Diseases (49) · Brain Diseases, Metabolic (49) · Brain Diseases, Metabolic, Inborn (49) · Central Nervous System Diseases (49) · Digestive System Diseases (49) · Heredodegenerative Disorders, Nervous System (49) · Nervous System Diseases (49) · Neurodegenerative Diseases (49) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (48) · Liver Diseases (48) · Metabolic Diseases (48) · Metal Metabolism, Inborn Errors (48) · Movement Disorders (48) · Nutritional and Metabolic Diseases (48)
tetrathiomolybdate (5) · Trientine (3) · Hematologic Tests (2) · Standard of Care (2) · Botulinum Toxins, Type A (1) · Bupropion (1) · Celecoxib (1) · Diagnostic Techniques and Procedures (1) · Echocardiography (1) · Electrocardiography (1) · Genetic Testing (1) · Lead (1) · Penicillamine (1) · Plasma Exchange (1) · Transcranial Magnetic Stimulation (1)
Alexion Pharmaceuticals, Inc. (10) · Fondation Ophtalmologique Adolphe de Rothschild (4) · Ultragenyx Pharmaceutical Inc (3) · Yale University (3) · Assistance Publique - Hôpitaux de Paris (2) · National Center for Research Resources (NCRR) (2) · Orphalan (2) · Prime Medicine, Inc. (2) · Second Affiliated Hospital, Zhejiang University, School of Medicine (2) · Asociación Española para el Estudio del Hígado (1) · Assiut University (1) · CENTOGENE GmbH Rostock (1) · Centre Hospitalier Universitaire de Liege (1) · Centre Hospitalier Universitaire de Saint Etienne (1) · Chaohui Yu (1)
United States (20) · United Kingdom (12) · France (11) · Germany (8) · China (7) · Spain (6) · Poland (5) · Denmark (4) · New Zealand (4) · Austria (3) · Canada (3) · Australia (2) · Belgium (2) · Brazil (2) · Italy (2)
Eugene S. Swenson, MD, PhD (3) · George J. Brewer (2) · Adrian Gadano, MD (1) · Arndt Rolfs, Prof. Dr. (1) · Chaohui Yu, PhD (1) · Curt Scharfe, MD, PhD (1) · Eduardo Jorge Premoli, MD (1) · Elon Pras, Prof (1) · Eugene S. Swenson, M.D., Ph.D. (1) · Eugene Swenson, MD, PhD (1) · Hebatullah I Fawzy, Msc Student (1)
PMID 22340672, PMID 25605552, PMID 28753182, PMID 28988934, PMID 30691535, PMID 31315600, PMID 31947878, PMID 34162411, PMID 34286869, PMID 34572285, PMID 35142618, PMID 35482910, PMID 36183738, PMID 38397079, PMID 38660122, PMID 39139457, PMID 40089450, PMID 40104015, PMID 41831608
2017 (1), 2018 (1), 2019 (1), 2025 (1), R01DK137861 (1)