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Wilson's Disease clinical trials

View clinical trials related to Wilson's Disease.

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NCT ID: NCT06128954 Completed - Wilson's Disease Clinical Trials

Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily).

Start date: January 16, 2024
Phase: Phase 1
Study type: Interventional

A randomised, open-label study evaluating the pharmacokinetics, safety, and tolerability of a new once daily dose of 900mg of TETA 4HCL by comparing it against the current marketed Cuprior® formulation (450mg trientine base, twice daily) in healthy male and female participants.

NCT ID: NCT06051734 Not yet recruiting - Clinical trials for Cardiovascular Diseases

Early Detection of Cardiac Affection in Patients of Wilson's Disease

Wilson's
Start date: October 1, 2023
Phase:
Study type: Observational [Patient Registry]

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 liver and the brain being primarily affected

NCT ID: NCT05493605 Recruiting - Wilson's Disease Clinical Trials

Cardiac Involvement in Wilson's Disease

WIL-HEART
Start date: March 2, 2023
Phase: N/A
Study type: Interventional

Heart damage by copper accumulation has been reported in Wilson's Disease. However, the disease epidemiology is still poorly understood. A number of studies on pediatric populations have not shown any significant cardiac involvement apart from early dysautonomia. This could suggest that the clinical manifestations related to the copper accumulation in the heart appears with the duration of the disease. Case-control studies on adult populations have highlighted various electrocardiographic (ECG) abnormalities more frequent in patients with Wilson's Disease than in healthy volunteers, but all these studies involved small number of patients (maximum 60). The hypothesis is that there is cardiac involvement in Wilson's Disease, requiring screening, follow-up and appropriate support.

NCT ID: NCT05305872 Recruiting - Wilson's Disease Clinical Trials

Gandouling in the Treatment of Wilson's Disease

Start date: January 1, 2023
Phase: Phase 4
Study type: Interventional

To evaluate the efficacy and safety of gandouling tablet in the treatment of clinical symptoms of Wilson's diasease.

NCT ID: NCT05239858 Recruiting - Wilson's Disease Clinical Trials

International Wilson's Disease Patient Registry (iWilson Registry)

Start date: June 29, 2022
Phase:
Study type: Observational [Patient 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. At enrolment, in addition to data from the clinic visit, retrospective data will be collected from the diagnostic evaluation and any relevant past medical history and a summary of WD medication history.

NCT ID: NCT05183165 Recruiting - Wilson's Disease Clinical Trials

Description of the Copper Concentration in Breast Milk in Women Treated for Wilson's Disease

WILLACT
Start date: May 11, 2022
Phase: N/A
Study type: Interventional

Wilson's disease is a rare genetic disease, affecting less than 1,500 people in France. The transmission is autosomal recessive linked to an anomaly of the ATP7B gene on chromosome.This gene codes for an ATPase-type transmembrane protein involved in the transport of copper through the cell plasma member.This gene codes for an ATPase-type transmembrane protein involved in the transport of copper through the cell plasma member. If there is no mutation, this ATPase incorporates copper into apo-ceruloplasmin to be released into the blood serum. The mutation of the ATP7B gene results in a defective biliary excretion of copper, leading to its accumulation in the liver, but also in other organs such as the eye or the brain. Advances in treatment have dramatically changed the prognosis for Wilson's disease, making the desire for pregnancy more confident. The consensus is to maintain treatment during pregnancy, reducing the dosage to limit teratogenicity as well as the risk of fetal copper deficiency.The mammary gland is the primary site of copper metabolism in lactation, and ATPase 7B is the primary effector. It has been shown in a mouse model of Wilson's disease (ATP7B - / - mouse) with treatment, that mothers accumulate copper in the liver but also in the mammary gland. However, a recent study showed that the copper level in breast milk was normal in 18 Wilsonian patients treated with D-penicillamine, trientine salts or zinc salts, suggesting that breastfeeding is possible in these patients without risk to the development of the infants.The problem of breastfeeding newborns for patients with Wilson's disease is therefore associated with a risk of copper deficiency in the newborn due to insufficiently rich breast milk in copper due to drugs. In addition, the passage into breast milk of treatments is not sufficiently known. These factors make breastfeeding not currently recommended for Wilsonian mothers,However, many patients wish to breastfeed and some of them breastfeed their newborns despite the risk of breastfeeding

NCT ID: NCT04537377 Recruiting - Wilson's Disease Clinical Trials

A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

GATEWAY
Start date: September 3, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

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 patients with Wilson's Disease prior to and following background WD therapy withdrawal.

NCT ID: NCT04212195 Recruiting - Wilson's Disease Clinical Trials

Cohort Research on Wilson's Disease

CROWD
Start date: December 6, 2018
Phase:
Study type: Observational

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.

NCT ID: NCT04012658 Recruiting - Wilson's Disease Clinical Trials

A Registered Cohort Study on Wilson's Disease

Start date: July 1, 2019
Phase:
Study type: Observational

The aim of this study is to determine the clinical spectrum and natural progression of Wilson's Disease in a prospective multicenter natural history study, to assess the clinical, genetic, epigenetic features and biomarkers of patients with Wilson's Disease to optimize clinical management.

NCT ID: NCT03957720 Recruiting - Wilson's Disease Clinical Trials

The Individual Therapy for Patients With Wilson's Disease

Start date: March 15, 2019
Phase: Early Phase 1
Study type: Interventional

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 treatment regimens for each genotype, so as to further improve the prognosis of patients.