Wilson's Disease Clinical Trial
Official title:
Cohort Research On Wilson's Disease: Genetic Determinants and Biomarker Discovery for Neurological Involvement
Wilson's disease (WD) is an inherited disorder that causes abnormal copper accumulation in
the brain and/or liver. Some people develop neurological or psychiatric symptoms whereas
other develop liver disease. The reasons for this are unclear but genetic factors are likely
to contribute. Current treatment, using copper-binding medications, is required lifelong.
Some respond well but others suffer debilitating side-effects or deteriorate despite
treatment, leading to disability or the need for liver transplantation.
In the first part of this study the main aim is to identify genetic factors that determine
whether someone with a diagnosis of WD will develop neurological involvement or not. The
investigators will invite 500 adults with WD across the UK to take part. Participants will be
asked to complete an online questionnaire and provide a saliva sample for genetic testing
using a collection kit sent via post. Identifying these genetic factors would significantly
advance our understanding of the disease and may provide new targets for drug discovery or
help guide more personalised approaches to treatment.
In the second part of this study the main aim is to develop new ways to monitor the effect of
WD on the brain using tests. Copper levels in blood and urine, currently used to monitor the
disease, are unreliable and do not necessarily reflect ongoing brain damage. The role of MRI
scans, cerebrospinal fluid tests or other measures of brain damage, commonly used in other
neurological disorders, is unclear. The investigators will therefore follow a group of 40
patients using clinical assessments and a combination of neurological tests, including novel
imaging and laboratory techniques, over 24 months. Developing new approaches to monitoring
the effect of WD on the brain will enable better prevention of neurological disability and be
essential for demonstrating the effectiveness of new treatments, such as gene therapy, in
clinical trials in the future.
n/a
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT04537377 -
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
|
Phase 1/Phase 2 | |
| Not yet recruiting |
NCT06051734 -
Early Detection of Cardiac Affection in Patients of Wilson's Disease
|
||
| Active, not recruiting |
NCT02252380 -
ExAblate Transcranial MRgFUS for the Management of Treatment-Refractory Movement Disorders
|
N/A | |
| Completed |
NCT01874028 -
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 | |
| Recruiting |
NCT05183165 -
Description of the Copper Concentration in Breast Milk in Women Treated for Wilson's Disease
|
N/A | |
| Completed |
NCT00212355 -
Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson's Disease in Japan.
|
Phase 3 | |
| Recruiting |
NCT05493605 -
Cardiac Involvement in Wilson's Disease
|
N/A | |
| Recruiting |
NCT03957720 -
The Individual Therapy for Patients With Wilson's Disease
|
Early Phase 1 | |
| Recruiting |
NCT05239858 -
International Wilson's Disease Patient Registry (iWilson Registry)
|
||
| Recruiting |
NCT04012658 -
A Registered Cohort Study on Wilson's Disease
|
||
| Completed |
NCT02552628 -
WILSTIM - DBS (WILson STIMulation - Deep Brain Stimulation)
|
N/A | |
| Completed |
NCT06128954 -
Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily).
|
Phase 1 | |
| Recruiting |
NCT05305872 -
Gandouling in the Treatment of Wilson's Disease
|
Phase 4 | |
| Completed |
NCT01378182 -
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
|
N/A |