Clinical trials: Wilson's Disease, open to volunteers
Wilson Disease, D-Penicillamine
Wilson's Disease Treated With D-Penicillamine: Characterization of Skin Damage Secondary to Treatment by Measuring Skin Elasticity
Wilson's disease is a genetic disorder, resulting from an anomaly present on the ATP7B gene located on chromosome 13, causing a progressive accumulation of copper in various organs such as the liver, nervous system and cornea…
- Conditions
- Wilson Disease, D-Penicillamine, Effect of D-penicilline on Cutaneous Elastity of Wilson's Patient
- Trial name
- WILDERME (NCT06945081)
- Testing
- Preparation of forearm molds with SILFLO® silicone
- Where
- 2 sites in France
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Wilson's Disease
Cardiac Involvement in Wilson's Disease
Heart damage by copper accumulation has been reported in Wilson's Disease. However, the disease epidemiology is still poorly understood.
- Trial name
- WIL-HEART (NCT05493605)
- Testing
- Delivery of a long-term Holter 21 days Or placement of an implantable holter from the outset so syncope
- Where
- France
- Volunteers
- 150 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Wilson Disease
Spanish Wilson Disease Registry
The main objective and purpose of the Registry is to know the current status of Wilson Disease in Spain. As secondary objectives, the prevalence and incidence of the disease will be analysed.
- Trial name
- NCT06466291
- Where
- Spain
- Volunteers
- 600 people
- Sponsor
- Asociación Española para el Estudio del Hígado
Wilson's Disease
Multifaceted Assessment of Patients With Wilson's Disease in a Low-Resource Setting in Upper Egypt: Service Integration, Psychosocial Burden, Dietary Practices, and the Geo-Spatial Disease Map
Wilson's disease (WD) is a rare autosomal recessive disorder of copper metabolism, caused by mutations in the *ATP7B* gene, leading to impaired copper metabolism and toxic accumulation in many organs including the liver, brain…
- Trial name
- NCT07241832
- Testing
- KAB-pB Scores
- Volunteers
- 66 people
- Sponsor
- Assiut University
Wilson Disease
Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT
The primary objective of this study is to investigate the effect of gene therapy (UX704) on copper distribution and excretion in Wilson disease patients.
- Trial name
- NCT07159581
- Where
- Denmark
- Volunteers
- 5 people
- Sponsor
- Thomas Damgaard Sandahl
Wilson's Disease
Description of the Copper Concentration in Breast Milk in Women Treated for Wilson's Disease
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…
- Trial name
- WILLACT (NCT05183165)
- Testing
- Patients with Wilson's disease declaring pregnancy,
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
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
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'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.
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
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
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.
Acute Liver Failure, Wilson Disease
Daily Versus Alternate Day Plasma Exchange in Wilson Disease With Acute Liver Failure in Children
Wilson disease in children has a varied presentation. Wilson disease with acute liver failure is associated with very high mortality and morbidity.
- Trial name
- NCT06698991
- Testing
- Plasma Exchange, Standard Medical Treatment
- Where
- India
- Volunteers
- 20 people
- Sponsor
- Institute of Liver and Biliary Sciences, India
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
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 Disease
Oral Health and Wilson's Disease: SOMAWI
Patients with Wilson disease have poorer dental and periodontal health and a have lower oral quality of life than control patients.
- Trial name
- SOMAWI (NCT05444127)
- Testing
- Saliva samples, Questionnaire OHIP-14, Thorough dental examination
- Where
- 2 sites in France
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Wilson Disease
A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
Wilson's disease (WD), also known as Wilson's disease, is a rare autosomal recessive metabolic disorder caused by a mutation of the copper transport ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3).
- Trial name
- WD (NCT06650319)
- Testing
- LY-M003
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Chaohui Yu
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
Clinical Features and Outcome
Clinical Features and Outcome in Patients With Osseomuscular Type of Wilson's Disease
The investigators aimed to observe the clinical features and outcome in patients with osseomuscular type of Wilson's disease from a large cohort during long-term follow-up
- Trial name
- NCT04965558
- Testing
- No intervention
- Where
- China
- Volunteers
- 1,000 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine