Clinical trials: Wilson's Disease
Cardiovascular Diseases, Wilson's Disease
Early Detection of Cardiac Affection in Patients of Wilson's Disease
Wilson disease (WD) is a rare autosomal recessive disorder caused by a genetic defect in ATP7B resulting in limited excretion of excess copper into the bile Pathological copper accumulation occurs in the entire body, with the…
- Trial name
- Wilson's (NCT06051734)
- Volunteers
- 50 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
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 Disease
Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
The main purpose of the study was to evaluate the efficacy of ALXN1840 (formerly WTX101) for 24 weeks on non-ceruloplasmin-bound copper (NCC) concentrations adjusted for molybdenum plasma concentration in participants newly…
- Trial name
- NCT02273596
- Testing
- ALXN1840
- Where
- 9 sites in United States, Austria and 3 more
- Volunteers
- 29 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Wilson's Disease
The Individual Therapy for Patients With Wilson's Disease
Based on the genotype characteristics and genotype-phenotype-treatment prognosis data of Chinese WD patients, this study intends to further optimize the treatment regimen of Chinese WD patients and formulate individualized…
- Trial name
- NCT03957720
- Testing
- DMPS, Penicillamine, DMSA
- Where
- China
- Volunteers
- 400 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Lysosomal Acid Lipase Deficiency
A Study to Identify and Characterize LAL-D Patients in High-risk Populations
The objective of this study is to determine the frequency of Lysosomal Acid Lipase Deficiency (LAL D) by lysosomal acid lipase (LAL) enzyme activity assay in patients who are considered to be at risk.
- Trial name
- NCT02345421
- Where
- 15 sites in United States
- Volunteers
- 640 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Wilson's Disease
Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease
This is a multicenter, open-label, single-arm, single-dose Phase I/II clinical study. It aims to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-M003…
- Trial name
- NCT07641140
- Testing
- LY-M003 Injection
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Lingyi Biotech Co., Ltd.
Wilson Disease, Liver Failure
Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease-related Liver Failure
This study is to investigate the clinical efficacy of artificial liver support system using combination of plasma exchange and continuous hemodiafiltration in treatment of Wilson's Disease - related liver failure.
- Trial name
- NCT03589820
- Testing
- artificial liver support system
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Third Affiliated Hospital, Sun Yat-Sen University
Wilson's Disease
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
Wilson's disease is an autosomal recessive genetically inherited disorder of copper metabolism, causing neurological, psychiatric and liver disease. The ATP7B gene on the 13th chromosome is responsible for the disease.
- Trial name
- NCT01378182
- Testing
- allogeneic mesenchymal stem cell transplantation
- Where
- Turkey (Türkiye)
- Volunteers
- 10 people
- Sponsor
- Murat Kantarcioglu
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
Movement Disorders, Essential Tremor
ExAblate Transcranial MRgFUS for the Management of Treatment-Refractory Movement Disorders
The proposed study is to evaluate the effectiveness of ExAblate Transcranial MRgFUS as a tool for creating a unilateral lesion in the Vim thalamus or the globus pallidus (GPi) in patients with treatment-refractory symptoms of…
- Conditions
- Movement Disorders, Essential Tremor, Holmes Tremor, Parkinson's Disease
- Trial name
- NCT02252380
- Testing
- Transcranial ExAblate System
- Where
- 2 sites in Canada
- Volunteers
- 10 people
- Sponsor
- InSightec
Wilson Disease
Early Access Program for ALXN1840 in Patients With Wilson Disease
This is an open-label, single-arm, multi-center EAP, designed to provide early access to ALXN1840 for eligible patients with WD.
- Trial name
- NCT05686564
- Testing
- ALXN1840
- Sponsor
- Alexion Pharmaceuticals, Inc.
Inherited Disease, Rare Diseases
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
General aim of the study is the improvement of the clinical knowledge of ultra-rare inherited metabolic and degenerative neurological diseases (prevalence less than 5:100,000) in adulthood through the systematic longitudinal…
- Conditions
- Inherited Disease, Rare Diseases, Metabolic Disease, Undiagnosed Disease
- Trial name
- NCT04880356
- Testing
- collection of data
- Where
- Italy
- Volunteers
- 100 people
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Wilson Disease
Study of Zinc for Wilson Disease
OBJECTIVES: I. Establish the safety and efficacy of extended maintenance zinc therapy in 200 patients with Wilson disease. II. Establish further the role of zinc in the prophylactic treatment of presymptomatic patients by…
- Trial name
- NCT00004338
- Testing
- zinc acetate
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- National Center for Research Resources (NCRR)
Wilson Disease
Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants
This is a Phase 1, randomized, 2-period, 2-sequence, cross-over study designed to determine the effect of ALXN1840 on the metabolism of bupropion, a sensitive cytochrome P450 2B6 (CYP2B6) substrate, in healthy male and female…
- Trial name
- NCT04526210
- Testing
- ALXN1840, Bupropion Hydrochloride
- Where
- United States
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Wilson's Disease
Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride
Decentralized study to assess patient reported treatment satisfaction comparing their current standard-of-care Wilson's Disease (WD) treatment with a new once-daily Trientine (TETA) 4HCl formulation.
- Trial name
- NCT07010575
- Testing
- New TETA 4HCl Formulation, Standard of Care
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- Orphalan
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
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
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Wilsons Disease
sCD163 and sMR in Wilsons Disease - Associations With Disease Severity and Fibrosis
The aim is to investigate macrophage activation markers and correlations to liver fibrosis in patients with Wilsons Disease. Researchers wish to investigate associations to neurologic and metabolic liver function.
- Trial name
- NCT02702765
- Testing
- Fibroscan, Ultrasound, Galactose
- Where
- Denmark
- Volunteers
- 33 people
- Sponsor
- University of Aarhus