Clinical trials: Wilson 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 Disease
Establishment of Human Cellular Disease Models for Wilson Disease
Establishment of human cellular disease models for Wilson disease for an individualized therapy develop-ment having the capacity to address both hepatic and neurologic forms of the disease
- Trial name
- IPSWILSON (NCT03867526)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
Rare Diseases, Amyloidosis
Institutional Registry of Rare Diseases
The goal of this observational study is to create a single macro registry system with data collection on common clinical features, grouping the different rare diseases (RD).
- Conditions
- Rare Diseases, Amyloidosis, Sarcoidosis, Phacomatosis
- Trial name
- NCT06573723
- Where
- Argentina
- Volunteers
- 380 people
- Sponsor
- Hospital Italiano de Buenos Aires
Wilson's Disease
Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.
The goal of this clinical trial is to learn if a new trientine tetrahydrochloride (TETA 4HCl) formulation administered once a day compared to d-Penicillamine (DPA) as a first line treatment for people living with Wilson's disease…
- Trial name
- TRADITiONAL (NCT07465718)
- Testing
- TETA 4HCl formulation, D-Penicillamine
- Where
- 10 sites in United States, China and 2 more
- Volunteers
- 38 people
- Sponsor
- Orphalan
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Trientine Treatment for Wilson's Disease
A Retrospective Study to Assess the Clinical Efficacy and Safety of Trientine in Wilson's Disease Patients
This is a retrospective study to assess the clinical efficacy and safety of trientine in Wilson's disease patients
- Trial name
- NCT03299829
- Testing
- Trientine
- Where
- Taiwan
- Volunteers
- 48 people
- Sponsor
- Excelsior
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's Disease
Exchangeable and Relative Exchangeable Copper as an Alternative to 24-Hour Urinary Copper in Wilson's Disease Monitoring
Serum exchangeable copper (EC) and relative exchangeable copper (REC) are blood tests developed to improve the assessment of copper levels in patients with Wilson's disease.
- Trial name
- NCT07462026
- Where
- Turkey (Türkiye)
- Volunteers
- 81 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Wilson's Disease
Gandouling in the Treatment of Wilson's Disease
To evaluate the efficacy and safety of gandouling tablet in the treatment of clinical symptoms of Wilson's diasease.
- Trial name
- NCT05305872
- Testing
- Gandouling
- Where
- China
- Volunteers
- 240 people
- Sponsor
- Jun Li
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 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'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
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
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 Clinical Study of Botulinum Toxin Type A Injection in the Treatment of Wilson Disease
In order to expand the treatment of Wilson disease and improve the quality of life of patients with Wilson's disease, the clinical effect of botulinum toxin type A injection on Wilson's disease was discussed.
- Trial name
- WD (NCT06762509)
- Testing
- Botulinum toxin type A
- Where
- China
- Volunteers
- 60 people
- Sponsor
- The First Affiliated Hospital of Anhui University of Traditional Chinese Medicine
Wilson's Disease
Endocrine Dysfunction in Pediatric Wilson's Disease
This cross-sectional study investigates endocrine changes in children diagnosed with Wilson's disease, aiming to characterize hormonal dysfunctions affecting pituitary, thyroid, adrenal, and gonadal axes.
- Trial name
- NCT07208565
- Volunteers
- 30 people
- Sponsor
- Assiut University
Wilson Disease, Wilson's Disease
A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)
The purpose of this study is to evaluate the safety, tolerability, biological activity, and initial efficacy of PM577a, an investigational Prime Editing therapy, in adults and adolescents with Wilson disease (WD).
- Conditions
- Wilson Disease, Wilson's Disease, Wilsons Disease
- Trial name
- PM577a (NCT07748403)
- Testing
- PM577a
- Where
- 4 sites in United States, New Zealand
- Volunteers
- 42 people
- Sponsor
- Prime Medicine, Inc.
Wilson Disease, Generalized Epilepsy
Clinical Features and Outcome of Wilson's Disease With Generalized Epilepsy in Chinese Patients
Generalized epilepsy is rarely reported in patients with Wilson disease (WD) and lacks experience in clinical practice. We aim to provide better experience for the diagnosis and treatment for WD patients with epilepsy in the…
- Trial name
- NCT04965571
- Testing
- genetic test
- Where
- China
- Volunteers
- 13 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Wilson Disease
Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
This study is being conducted to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease (WD).
- Trial name
- NCT05047523
- Testing
- ALXN1840, Standard of Care
- Where
- 20 sites in Australia, France and 5 more
- Volunteers
- 40 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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