| Not Yet Recruiting | Description of Renal Involvement in Wilson's Disease NCT07075393 | Fondation Ophtalmologique Adolphe de Rothschild | — |
| Recruiting | Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study NCT07301216 | Yale University | — |
| Recruiting | Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial NCT07226622 | Prime Medicine, Inc. | — |
| Recruiting | Wilson's Disease Treated With D-Penicillamine: Characterization of Skin Damage Secondary to Treatment by Measu NCT06945081 | Centre Hospitalier Universitaire de Saint Etienne | N/A |
| Not Yet Recruiting | Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients Wi NCT07173933 | GeneCradle Inc | Phase 1 / Phase 2 |
| Enrolling By Invitation | Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT NCT07159581 | Thomas Damgaard Sandahl | — |
| Recruiting | Circadian Variation of Urinary Copper Excretion in Wilson Disease Patients NCT06430359 | Hospices Civils de Lyon | — |
| Not Yet Recruiting | Daily Versus Alternate Day Plasma Exchange in Wilson Disease With Acute Liver Failure in Children NCT06698991 | Institute of Liver and Biliary Sciences, India | N/A |
| Not Yet Recruiting | An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease NCT06663878 | Xinhua Hospital, Shanghai Jiao Tong University School of Medicine | N/A |
| Recruiting | A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease NCT06650319 | Chaohui Yu | EARLY_Phase 1 |
| Completed | The Exploratory Handheld Optical Coherence Tomography Study in Wilson's Disease NCT07046507 | Royal Surrey County Hospital NHS Foundation Trust | N/A |
| Recruiting | Institutional Registry of Rare Diseases NCT06573723 | Hospital Italiano de Buenos Aires | — |
| Unknown | Evaluation of Cognitive Dysfunction and Psychiatric Comorbidities NCT06179394 | Assiut University | — |
| Completed | Real World Evidence Study in Subjects With Wilson's Disease NCT05783687 | Orphalan | — |
| Recruiting | Oral Health and Wilson's Disease: SOMAWI NCT05444127 | Fondation Ophtalmologique Adolphe de Rothschild | — |
| Completed | rTMS in Wilson Disease Dysarthria NCT04910581 | Assistance Publique - Hôpitaux de Paris | N/A |
| Completed | Baby Detect : Genomic Newborn Screening NCT05687474 | Centre Hospitalier Universitaire de Liege | — |
| Recruiting | Spanish Wilson Disease Registry NCT06466291 | Asociación Española para el Estudio del Hígado | — |
| Active Not Recruiting | A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease NCT04884815 | Ultragenyx Pharmaceutical Inc | Phase 1 / Phase 2 |
| Terminated | Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease NCT05047523 | Alexion Pharmaceuticals, Inc. | Phase 3 |
| Terminated | Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease NCT04909346 | Ultragenyx Pharmaceutical Inc | — |
| Completed | Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease NCT04531189 | Ultragenyx Pharmaceutical Inc | — |
| Completed | Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated Wit NCT04422431 | Alexion Pharmaceuticals, Inc. | Phase 2 |
| Completed | Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants NCT04526210 | Alexion Pharmaceuticals, Inc. | Phase 1 |
| Completed | Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 NCT04573309 | Alexion Pharmaceuticals, Inc. | Phase 2 |
| Completed | Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants. NCT04526197 | Alexion Pharmaceuticals, Inc. | Phase 1 |
| Completed | Study of Retinal Vascular Parameters in Patients With Wilson's Disease NCT04408300 | Fondation Ophtalmologique Adolphe de Rothschild | N/A |
| Completed | Pharmacokinetic Study of Oral ALXN1840 in Japanese and Non-Japanese Adult Healthy Participants NCT05641311 | Alexion Pharmaceuticals, Inc. | Phase 1 |
| Enrolling By Invitation | Early Check: Expanded Screening in Newborns NCT03655223 | RTI International | — |
| Completed | Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease NCT03539952 | Orphalan | Phase 3 |
| Unknown | A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease NCT03659331 | Prof. Elon Pras | N/A |
| Completed | Establishment of Human Cellular Disease Models for Wilson Disease NCT03867526 | CENTOGENE GmbH Rostock | — |
| Unknown | Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease-related Liver Failure NCT03589820 | Third Affiliated Hospital, Sun Yat-Sen University | N/A |
| Terminated | Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson NCT03403205 | Alexion Pharmaceuticals, Inc. | Phase 3 |
| Recruiting | Natural History of Wilson Disease NCT03334292 | Yale University | — |
| Completed | The Clinical Study of Botulinum Toxin Type A Injection in the Treatment of Wilson Disease NCT06762509 | The First Affiliated Hospital of Anhui University of Traditional Chinese Medicine | — |
| Completed | The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment NCT02763215 | Alexion Pharmaceuticals, Inc. | — |
| Unknown | Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penic NCT02426905 | Univar BV | Phase 4 |
| Completed | Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients NCT02273596 | Alexion Pharmaceuticals, Inc. | Phase 2 |
| Completed | Inhibitory rTMS in Dystonic Wilson Patients NCT01980433 | Assistance Publique - Hôpitaux de Paris | N/A |
| Completed | Clinical Features and Outcome of Wilson's Disease With Generalized Epilepsy in Chinese Patients NCT04965571 | Second Affiliated Hospital, School of Medicine, Zhejiang University | — |
| Completed | Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease NCT01472874 | Yale University | N/A |
| Recruiting | French Wilson Disease Registry NCT05231876 | Fondation Ophtalmologique Adolphe de Rothschild | — |
| Recruiting | Role for Biochemical Assays and Kayser-Fleischer Rings in Diagnosis of Wilson Disease NCT04965545 | Second Affiliated Hospital, School of Medicine, Zhejiang University | — |
| Completed | Study of Tetrathiomolybdate in Patients With Wilson Disease NCT00004339 | National Center for Research Resources (NCRR) | Phase 3 |
| Completed | Study of Zinc for Wilson Disease NCT00004338 | National Center for Research Resources (NCRR) | Phase 4 |
| No Longer Available | Early Access Program for ALXN1840 in Patients With Wilson Disease NCT05686564 | Alexion Pharmaceuticals, Inc. | — |