Wilson Disease
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CLINICAL_TRIALS trial_result · 2026-04-06Clinical Trial Update: Real World Evidence Study in Subjects With Wilson's Disease
This clinical trial update reports the completion of a real-world evidence study in subjects with Wilson's disease, sponsored by Orphalan.
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CLINICAL_TRIALS trial_result · 2026-04-06Clinical Trial Update: Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride [Phase 2]
A Phase 2 patient-preference study comparing standard of care versus once-daily trientine tetrahydrochloride for Wilson's disease has comple…
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CLINICAL_TRIALS trial_result · 2026-03-10Clinical Trial Update: Exchangeable and Relative Exchangeable Copper as an Alternative to 24-Hour Urinary Copper in Wilson's Disease Monitoring
A completed clinical trial evaluates exchangeable and relative exchangeable copper as alternatives to 24-hour urinary copper for monitoring …
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CLINICAL_TRIALS trial_result · 2026-01-28Clinical Trial Update: A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease [Phase 1, Phase 2]
A Phase I/II trial update for VTX-801 in adults with Wilson's disease, with results posted January 28, 2026.
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CLINICAL_TRIALS trial_result · 2025-12-05Clinical Trial Update: Performance and Safety of MEX-CD1 Low-volume Continuous Veno-venous Haemodialysis Medical Device for Copper-extraction in Patients With Wilson's Disease [NA]
A clinical trial evaluating the MEX-CD1 low-volume continuous venovenous haemodialysis device for copper extraction in Wilson disease has be…
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CLINICAL_TRIALS trial_result · 2025-09-15Clinical Trial Update: Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD) [Phase 1, Phase 2]
A Phase I/II clinical trial is planned to evaluate GC310 Injection in Wilson disease. Recruitment has not yet begun.
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CLINICAL_TRIALS trial_result · 2025-08-22Clinical Trial Update: WILSTIM - DBS (WILson STIMulation - Deep Brain Stimulation) [NA]
A completed study evaluating Wilson STIMulation Deep Brain Stimulation (WILSTIM DBS) for severe dystonia in Wilson's disease, sponsored by H…
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CLINICAL_TRIALS trial_result · 2025-08-15Clinical Trial Update: Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily). [Phase 1]
A Phase 1 trial comparing a once-daily 900 mg dose of TETA 4HCl to Cuprior (trientine) in Wilson's disease has completed and posted results.
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CLINICAL_TRIALS trial_result · 2025-07-01Clinical Trial Update: The Exploratory Handheld Optical Coherence Tomography Study in Wilson's Disease [NA]
The document reports completion of an exploratory handheld optical coherence tomography study in Wilson disease.
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CLINICAL_TRIALS trial_result · 2025-01-15Clinical Trial Update: The Clinical Study of Botulinum Toxin Type A Injection in the Treatment of Wilson Disease
A completed clinical trial evaluating botulinum toxin type A injections for Wilson disease.
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CLINICAL_TRIALS trial_result · 2024-10-15Clinical Trial Update: Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease [Phase 3]
A Phase 3 trial evaluating ALXN1840 versus standard of care in pediatric Wilson disease participants was terminated, and trial results have …
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CLINICAL_TRIALS trial_result · 2024-02-09Clinical Trial Update: Gandouling in the Treatment of Wilson's Disease [Phase 4]
Phase 4 clinical trial update for Gandouling in Wilson's Disease; current status is unknown.
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CLINICAL_TRIALS trial_result · 2023-11-07Clinical Trial Update: Pharmacokinetic Study of Oral ALXN1840 in Japanese and Non-Japanese Adult Healthy Participants [Phase 1]
Phase 1 pharmacokinetic study of oral ALXN1840 in Japanese and non-Japanese healthy adults completed; results posted.
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CLINICAL_TRIALS trial_result · 2023-10-27Clinical Trial Update: Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 [Phase 2]
Phase 2 results from a trial of ALXN1840 in Wilson disease report copper and molybdenum balance in participants. Results were posted on 2023…
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CLINICAL_TRIALS trial_result · 2023-10-10Clinical Trial Update: Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease [Phase 3]
A Phase 3 trial comparing ALXN1840 to standard of care in Wilson disease was terminated, and results were posted on 2023-10-10.
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CLINICAL_TRIALS trial_result · 2023-08-31Clinical Trial Update: Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants [Phase 1]
Phase 1 study evaluating ALXN1840's effect on metabolism of a CYP2B6 substrate in healthy participants, completed with results posted.
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CLINICAL_TRIALS trial_result · 2023-08-21Clinical Trial Update: Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants. [Phase 1]
Phase 1 study evaluating ALXN1840's effect on metabolism of a CYP2C9 substrate in healthy participants completed with results posted.
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CLINICAL_TRIALS trial_result · 2023-07-03Clinical Trial Update: Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840 [Phase 2]
Phase 2 clinical trial results evaluating ALXN1840 for Wilson disease, including copper concentration and liver histopathologic changes; res…
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CLINICAL_TRIALS trial_result · 2023-04-20Clinical Trial Update: Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease [Phase 3]
Phase 3 trial results for trientine tetrahydrochloride in Wilson disease have been completed, with results posted in April 2023.
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CLINICAL_TRIALS trial_result · 2021-09-29Clinical Trial Update: Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients [Phase 2]
Phase 2 trial assessing efficacy and safety of WTX101 (ALXN1840) in adults with Wilson disease has completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-10-26Clinical Trial Update: The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment
A completed clinical trial evaluating copper parameter assessment in Wilson disease participants receiving standard of care treatment; resul…
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CLINICAL_TRIALS trial_result · 2018-07-18Clinical Trial Update: Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease-related Liver Failure [NA]
This clinical trial update describes the use of plasma exchange and continuous hemodiafiltration for Wilson disease-related liver failure, w…
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CLINICAL_TRIALS trial_result · 2017-08-23Clinical Trial Update: Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine [Phase 4]
Phase 4 trial assessing the long-term outcomes of trientine in Wilson disease patients who were withdrawn from d-penicillamine therapy.
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CLINICAL_TRIALS trial_result · 2014-12-31Clinical Trial Update: Efficacy of Invitro Expanded Bone Marrow Derived Allogeneic Mesenchymal Stem Cell Transplantation Via Portal Vein or Hepatic Artery or Peripheral Vein in Patients With Wilson Cirrhosis [NA]
A completed clinical trial evaluating the efficacy of Invitro-expanded allogeneic bone marrow-derived mesenchymal stem cell transplantation …
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CLINICAL_TRIALS trial_result · 2014-09-19Clinical Trial Update: A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease Patients [Phase 1]
This completed Phase 1 clinical trial evaluated the pharmacokinetics and tolerability of a single dose of trientine dihydrochloride in patie…