{"disease":{"id":"wilson-disease","name":"wilson disease"},"drugs":{"marketed":[{"drug_id":"trientine","indication_name":"Wilson's Disease","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Syprine","generic_name":"TRIENTINE","company_name":"Bausch Health","drug_phase":"marketed","molecular_target":"Carbonic anhydrase 14","drug_class":"Copper Chelator","quality_score":10,"revenue":null,"mechanism":""},{"drug_id":"trientine","indication_name":"Wilson's Disease","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Syprine","generic_name":"TRIENTINE","company_name":"Bausch Health","drug_phase":"marketed","molecular_target":"Carbonic anhydrase 14","drug_class":"Copper Chelator","quality_score":10,"revenue":null,"mechanism":""},{"drug_id":"trientine","indication_name":"Wilson's disease","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Syprine","generic_name":"TRIENTINE","company_name":"Bausch Health","drug_phase":"marketed","molecular_target":"Carbonic anhydrase 14","drug_class":"Copper Chelator","quality_score":10,"revenue":null,"mechanism":""},{"drug_id":"penicillamine","indication_name":"Wilson's disease","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Cuprimine","generic_name":"PENICILLAMINE","company_name":"Valeant Pharms Intl","drug_phase":"marketed","molecular_target":"","drug_class":"Antirheumatic Agent [EPC]","quality_score":45,"revenue":null,"mechanism":""},{"drug_id":"zinc-acetate","indication_name":"Wilson's disease","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Galzin","generic_name":"ZINC ACETATE","company_name":"Eton","drug_phase":"marketed","molecular_target":"","drug_class":"zinc acetate","quality_score":37,"revenue":null,"mechanism":""}],"pipeline":[],"offLabel":[],"totalMarketed":5,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT03655223","title":"Early Check: Expanded Screening in Newborns","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":30000,"lead_sponsor_name":"RTI International","has_results":false},{"nct_id":"NCT05687474","title":"Baby Detect : Genomic Newborn Screening","phase":"","overall_status":"COMPLETED","enrollment_count":6824,"lead_sponsor_name":"Centre Hospitalier Universitaire de Liege","has_results":false},{"nct_id":"NCT04965545","title":"Role for Biochemical Assays and Kayser-Fleischer Rings in Diagnosis of Wilson Disease","phase":"","overall_status":"RECRUITING","enrollment_count":1000,"lead_sponsor_name":"Second Affiliated Hospital, School of Medicine, Zhejiang University","has_results":false},{"nct_id":"NCT05231876","title":"French Wilson Disease Registry","phase":"","overall_status":"RECRUITING","enrollment_count":1000,"lead_sponsor_name":"Fondation Ophtalmologique Adolphe de Rothschild","has_results":false},{"nct_id":"NCT04212195","title":"Cohort Research on Wilson's Disease","phase":"","overall_status":"UNKNOWN","enrollment_count":500,"lead_sponsor_name":"University College, London","has_results":false},{"nct_id":"NCT03957720","title":"The Individual Therapy for Patients With Wilson's Disease","phase":"EARLY_PHASE1","overall_status":"UNKNOWN","enrollment_count":400,"lead_sponsor_name":"Second Affiliated Hospital, School of Medicine, Zhejiang University","has_results":false},{"nct_id":"NCT06573723","title":"Institutional Registry of Rare Diseases","phase":"","overall_status":"RECRUITING","enrollment_count":380,"lead_sponsor_name":"Hospital Italiano de Buenos Aires","has_results":false},{"nct_id":"NCT03334292","title":"Natural History of Wilson Disease","phase":"","overall_status":"RECRUITING","enrollment_count":300,"lead_sponsor_name":"Yale University","has_results":false},{"nct_id":"NCT05305872","title":"Gandouling in the Treatment of Wilson's Disease","phase":"PHASE4","overall_status":"UNKNOWN","enrollment_count":240,"lead_sponsor_name":"Jun Li","has_results":false},{"nct_id":"NCT03403205","title":"Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease","phase":"PHASE3","overall_status":"TERMINATED","enrollment_count":214,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT04408300","title":"Study of Retinal Vascular Parameters in Patients With Wilson's Disease","phase":"NA","overall_status":"COMPLETED","enrollment_count":124,"lead_sponsor_name":"Fondation Ophtalmologique Adolphe de Rothschild","has_results":false},{"nct_id":"NCT05004701","title":"Impact of the Lockdown Due to the COVID Pandemic in French Wilson's Disease Patients","phase":"","overall_status":"TERMINATED","enrollment_count":120,"lead_sponsor_name":"Fondation Ophtalmologique Adolphe de Rothschild","has_results":false},{"nct_id":"NCT07462026","title":"Exchangeable and Relative Exchangeable Copper as an Alternative to 24-Hour Urinary Copper in Wilson's Disease Monitoring","phase":"","overall_status":"COMPLETED","enrollment_count":81,"lead_sponsor_name":"Hacettepe University","has_results":false},{"nct_id":"NCT03539952","title":"Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":77,"lead_sponsor_name":"Orphalan","has_results":true},{"nct_id":"NCT07241832","title":"Multifaceted Assessment of Patients With Wilson's Disease in a Low-Resource Setting in Upper Egypt: Service Integration, Psychosocial Burden, Dietary Practices, and the Geo-Spatial Disease Map","phase":"","overall_status":"NOT_YET_RECRUITING","enrollment_count":66,"lead_sponsor_name":"Assiut University","has_results":false},{"nct_id":"NCT02763215","title":"The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment","phase":"","overall_status":"COMPLETED","enrollment_count":64,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT06762509","title":"The Clinical Study of Botulinum Toxin Type A Injection in the Treatment of Wilson Disease","phase":"","overall_status":"COMPLETED","enrollment_count":60,"lead_sponsor_name":"The First Affiliated Hospital of Anhui University of Traditional Chinese Medicine","has_results":false},{"nct_id":"NCT03589820","title":"Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease-related Liver Failure","phase":"NA","overall_status":"UNKNOWN","enrollment_count":60,"lead_sponsor_name":"Third Affiliated Hospital, Sun Yat-Sen University","has_results":false},{"nct_id":"NCT04526210","title":"Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":54,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT04909346","title":"Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease","phase":"","overall_status":"TERMINATED","enrollment_count":51,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":false},{"nct_id":"NCT06051734","title":"Early Detection of Cardiac Affection in Patients of Wilson's Disease","phase":"","overall_status":"NOT_YET_RECRUITING","enrollment_count":50,"lead_sponsor_name":"Assiut University","has_results":false},{"nct_id":"NCT05783687","title":"Real World Evidence Study in Subjects With Wilson's Disease","phase":"","overall_status":"COMPLETED","enrollment_count":50,"lead_sponsor_name":"Orphalan","has_results":false},{"nct_id":"NCT03299829","title":"A Retrospective Study to Assess the Clinical Efficacy and Safety of Trientine in Wilson's Disease Patients","phase":"","overall_status":"COMPLETED","enrollment_count":48,"lead_sponsor_name":"Excelsior","has_results":false},{"nct_id":"NCT03867526","title":"Establishment of Human Cellular Disease Models for Wilson Disease","phase":"","overall_status":"COMPLETED","enrollment_count":40,"lead_sponsor_name":"CENTOGENE GmbH Rostock","has_results":false},{"nct_id":"NCT05047523","title":"Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease","phase":"PHASE3","overall_status":"TERMINATED","enrollment_count":40,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT06179394","title":"Evaluation of Cognitive Dysfunction and Psychiatric Comorbidities","phase":"","overall_status":"UNKNOWN","enrollment_count":40,"lead_sponsor_name":"Assiut University","has_results":false},{"nct_id":"NCT04526197","title":"Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":36,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT04422431","title":"Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":31,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT07208565","title":"Endocrine Dysfunction in Pediatric Wilson's Disease","phase":"","overall_status":"NOT_YET_RECRUITING","enrollment_count":30,"lead_sponsor_name":"Assiut University","has_results":false},{"nct_id":"NCT07301216","title":"Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study","phase":"","overall_status":"RECRUITING","enrollment_count":30,"lead_sponsor_name":"Yale University","has_results":false},{"nct_id":"NCT06430359","title":"Circadian Variation of Urinary Copper Excretion in Wilson Disease Patients","phase":"","overall_status":"RECRUITING","enrollment_count":30,"lead_sponsor_name":"Hospices Civils de Lyon","has_results":false},{"nct_id":"NCT02273596","title":"Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":29,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT06128954","title":"Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily).","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":26,"lead_sponsor_name":"Orphalan","has_results":true},{"nct_id":"NCT07476417","title":"Oral Health, Dento-facial Condition and OHRQoL in Subjects With Mowat-Wilson Syndrome: an Epidemiologic Study.","phase":"","overall_status":"NOT_YET_RECRUITING","enrollment_count":25,"lead_sponsor_name":"University of Milan","has_results":false},{"nct_id":"NCT05641311","title":"Pharmacokinetic Study of Oral ALXN1840 in Japanese and Non-Japanese Adult Healthy Participants","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":24,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT05792319","title":"Clinical Profile and Out Come of Children With Wilson's Disease","phase":"","overall_status":"UNKNOWN","enrollment_count":20,"lead_sponsor_name":"Assiut University","has_results":false},{"nct_id":"NCT06698991","title":"Daily Versus Alternate Day Plasma Exchange in Wilson Disease With Acute Liver Failure in Children","phase":"NA","overall_status":"NOT_YET_RECRUITING","enrollment_count":20,"lead_sponsor_name":"Institute of Liver and Biliary Sciences, India","has_results":false},{"nct_id":"NCT06650319","title":"A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease","phase":"EARLY_PHASE1","overall_status":"RECRUITING","enrollment_count":18,"lead_sponsor_name":"Chaohui Yu","has_results":false},{"nct_id":"NCT04910581","title":"rTMS in Wilson Disease Dysarthria","phase":"NA","overall_status":"COMPLETED","enrollment_count":18,"lead_sponsor_name":"Assistance Publique - Hôpitaux de Paris","has_results":false},{"nct_id":"NCT04531189","title":"Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease","phase":"","overall_status":"COMPLETED","enrollment_count":16,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":false},{"nct_id":"NCT04965571","title":"Clinical Features and Outcome of Wilson's Disease With Generalized Epilepsy in Chinese Patients","phase":"","overall_status":"COMPLETED","enrollment_count":13,"lead_sponsor_name":"Second Affiliated Hospital, School of Medicine, Zhejiang University","has_results":false},{"nct_id":"NCT07010575","title":"Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":10,"lead_sponsor_name":"Orphalan","has_results":false},{"nct_id":"NCT07046507","title":"The Exploratory Handheld Optical Coherence Tomography Study in Wilson's Disease","phase":"NA","overall_status":"COMPLETED","enrollment_count":10,"lead_sponsor_name":"Royal Surrey County Hospital NHS Foundation Trust","has_results":false},{"nct_id":"NCT04573309","title":"Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":9,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT04537377","title":"A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease","phase":"PHASE1, PHASE2","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":4,"lead_sponsor_name":"Vivet Therapeutics SAS","has_results":true},{"nct_id":"NCT05686564","title":"Early Access Program for ALXN1840 in Patients With Wilson Disease","phase":"","overall_status":"NO_LONGER_AVAILABLE","enrollment_count":null,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":false}],"total":46},"guidelines":[],"source":"Drug Landscape verified database"}