Clinical trials: Wilson Disease
Hepatolenticular Degeneration; Wilson
Performance and Safety of MEX-CD1 Low-volume Continuous Veno-venous Haemodialysis Medical Device for Copper-extraction in Patients With Wilson's Disease
The goal of this clinical trial is to test the MEX-CD1 hemodialysis medical device in patients suffering from Wilson's Disease. The main questions it aims to answer are: Does the device work as expected by removing the excess of…
- Trial name
- MEXWILS (NCT05917327)
- Testing
- Low-volume continuous veno-venous haemodialysis
- Where
- 4 sites in France, Spain
- Volunteers
- 1 people
- Sponsor
- Mexbrain
Wilson Disease
Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
The study will evaluate the efficacy and safety of ALXN1840 (formerly called WTX101) administered for 48 weeks compared to standard of care (SoC) in Wilson Disease (WD) participants aged 12 and older in the Primary Evaluation…
- Trial name
- NCT03403205
- Testing
- ALXN1840, SoC Therapy
- Where
- 56 sites in United States, Australia and 20 more
- Volunteers
- 214 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Wilson Disease
Study of Tetrathiomolybdate in Patients With Wilson Disease
OBJECTIVES: Evaluate the safety and efficacy of ammonium tetrathiomolybdate alone and compared with trientine therapy as initial treatment in patients with Wilson disease presenting neurologically.
- Trial name
- NCT00004339
- Testing
- tetrathiomolybdate, trientine
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- National Center for Research Resources (NCRR)
Wilson Disease
French Wilson Disease Registry
This registry concerns adults and children with Wilson's disease. The collection of a large amount of data will allow a better understanding of the epidemiology of this rare disease, in particular the age of onset according to…
- Trial name
- WIL-FR (NCT05231876)
- Testing
- Recording of pathology-related information on the Wilson Register
- Where
- France
- Volunteers
- 1,000 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Wilson Disease
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
The main objective of the study is to evaluate the change in liver copper (Cu) concentration following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD) who have been previously treated for at…
- Trial name
- NCT04422431
- Testing
- Bis-Choline Tetrathiomolybdate
- Where
- 12 sites in United States, Canada and 6 more
- Volunteers
- 31 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Wilson Disease
Evaluation of Cognitive Dysfunction and Psychiatric Comorbidities
Primary objective Collection of patients with wilson disease either presented with neurological or hepatic symptoms Assessment of psychiatric and cognitive disorders in both groups by using specific scales Secondary objective…
- Trial name
- NCT06179394
- Volunteers
- 40 people
- Sponsor
- Assiut University
Wilson's Disease
International Wilson's Disease Patient Registry (iWilson Registry)
Longitudinal, observational, non-interventional, standard of care Registry. Data will be collected from the routinely scheduled WD clinic visits at approximately 6-12 month intervals.
- Trial name
- NCT05239858
- Where
- 16 sites in Belgium, France and 5 more
- Volunteers
- 500 people
- Sponsor
- Orphalan
Wilson Disease
The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment
This was a 24-month study to assess copper parameters in participants with Wilson disease (WD) treated with standard of care (SoC) medications.
- Trial name
- NCT02763215
- Testing
- Standard of Care Medications
- Where
- 10 sites in United States, Austria and 3 more
- Volunteers
- 64 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Wilson Disease
Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)
The goal of this clinical trial is to learn if GC310 (AAV5-ATP7B) gene therapy can treat Wilson's Disease (WD) in patients over the age of 18 years old.
- Trial name
- NCT07173933
- Testing
- GC310
- Where
- China
- Volunteers
- 15 people
- Sponsor
- GeneCradle Inc
Wilson Disease
An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease
The primary objective of this study is to evaluate the tolerability and safety of MWAV201 in patients with Wilson disease.
- Trial name
- NCT06663878
- Testing
- MWAV201
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Wilson Disease
A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease
The assumption is that in some of the carriers, the increase in enzymes reflects tissue damage due to excess copper. The reduction of the amount of copper absorbed will decrease excess copper in the liver, which will result in a…
- Trial name
- NCT03659331
- Testing
- Zinc
- Volunteers
- 50 people
- Sponsor
- Prof. Elon Pras
Wilson's Disease and COVID-19
Impact of the Lockdown Due to the COVID Pandemic in French Wilson's Disease Patients
Wilson's disease is a rare genetic disorder that causes copper to build up in the body. This overload is initially localized in the liver and the brain, but can spread throughout the body and cause systemic damage if copper…
- Trial name
- WILL-Ecco (NCT05004701)
- Where
- France
- Volunteers
- 120 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Wilson's Disease
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
The objectives of this clinical trial are to assess, for up to 5 years, the safety, tolerability and pharmacological activity of a single ascending doses of VTX-801, a gene therapy, administered intravenously (IV) to adult…
- Trial name
- GATEWAY (NCT04537377)
- Testing
- VTX-801
- Where
- 10 sites in United States, Denmark and 2 more
- Volunteers
- 4 people
- Sponsor
- Vivet Therapeutics SAS
Wilson's Disease, Myocardial Dysfunction
Subclinical Myocardial Dysfunction in Children With Wilson's Disease
Wilson's disease (WD) is one of the most common metabolic liver diseases in older children. The most frequent clinical presentation is liver disease. However, Wilson's disease (WD) is a multisystem disorder.
- Conditions
- Wilson's Disease, Myocardial Dysfunction, Children, Wilson Disease
- Trial name
- NCT07765472
- Testing
- Echocardiography, Electrocardiography, N-terminal pro b- type natriuretic peptide
- Where
- 2 sites in Egypt
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Hebatullah Fawzy
Wilson Disease
Description of Renal Involvement in Wilson's Disease
Wilson's disease (WD) is a rare genetic disorder that leads to copper accumulation in various tissues, including the liver, nervous system, heart, and kidneys.
- Trial name
- WILKID (NCT07075393)
- Testing
- full renal workup
- Where
- France
- Volunteers
- 150 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Wilson Disease
Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial
The aim of this study is to inform and improve future clinical trials in Wilson Disease (WD) by better understanding how patients with WD are living with and managing the disease, and by identifying key factors that shape their…
- Trial name
- NCT07226622
- Where
- 7 sites in United States
- Volunteers
- 30 people
- Sponsor
- Prime Medicine, Inc.
Wilson Disease
Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease
Hypothesis: The investigators postulate that patients with Wilson disease who are asymptomatic or who have been effectively treated for their symptoms and are in a maintenance phase therapy can be safely and effectively treated…
- Trial name
- NCT01472874
- Testing
- Once a day Trientine
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Yale University
Wilson Disease, Movement Disorders
Inhibitory rTMS in Dystonic Wilson Patients
Wilson disease is a genetic disorder resulting in copper accumulation in liver, brain and eye. The neurologic complications include dystonic syndrome, which is a prolonged and excessive muscle activation responsible for abnormal…
- Conditions
- Wilson Disease, Movement Disorders, Repetitive Transcranial Magnetic Stimulation
- Trial name
- WILSTIM (NCT01980433)
- Testing
- Repetitive Transcranial Magnetic Stimulation (rTMS), pre and post-rTMS electroencephalogram, WCRS
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Wilson Disease
Real World Evidence Study in Subjects With Wilson's Disease
This non-interventional Real-World Evidence (RWE) study aims to describe non-ceruloplasmin copper values obtained using a new NCC Speciation assay by taking a small (up to 10mLs) volume of additional blood from patients with…
- Trial name
- REASON (NCT05783687)
- Testing
- NCC-Sp Assay
- Where
- 9 sites in United States
- Volunteers
- 50 people
- Sponsor
- Orphalan
Hepatolenticular Degeneration; Wilson
Clinical Value of DWI-ADC Matching in the Short-term Prognosis of Wilson's Disease
In earlier studies, it was found that patients of Wilson disease with new diagnosed who only has neurological symptoms often had DWI hyper-intensity in brain MRI, which was more common in putamen and midbrain, indicating that the…
- Trial name
- NCT06196931
- Where
- China
- Volunteers
- 200 people
- Sponsor
- Hospital Affiliated to the Institute of Neurology, Anhui University of Chinese Medicine