Clinical trials: Wilson Disease
Wilson Disease
Pharmacokinetic Study of Oral ALXN1840 in Japanese and Non-Japanese Adult Healthy Participants
The main purpose of the study is to confirm how long ALXN1840 stays in the body of Japanese and non-Japanese healthy participants (that is, pharmacokinetic [PK] profile).
- Trial name
- NCT05641311
- Testing
- ALXN1840
- Where
- United Kingdom
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Wilson Disease
Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot Study
This is a prospective study that will determine the optimal timing for 24-hour urinary copper excretion (UCE) measurement after temporary discontinuation of standard therapies in Wilson Disease (WD) patients.
- Trial name
- NCT07301216
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Yale University
Wilson Disease
The Exploratory Handheld Optical Coherence Tomography Study in Wilson's Disease
This is an exploratory study using anterior segment optical coherence tomography (OCT) devices to measure copper in the eye. The investigators hope to use this to diagnosis kayser flesicher rings, an important diagnostic criteria…
- Trial name
- NCT07046507
- Testing
- Diagnostic procedures
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- Royal Surrey County Hospital NHS Foundation Trust
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 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 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 Disease
Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.
- Trial name
- NCT02426905
- Testing
- trientine dihydrochloride
- Where
- 4 sites in Germany, Greece and 2 more
- Volunteers
- 90 people
- Sponsor
- Univar BV
Wilson's Disease
A Registered Cohort Study on Wilson's Disease
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…
- Trial name
- NCT04012658
- Testing
- No intervention
- Where
- China
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Wan-Jin Chen
Wilson Disease
Study of Retinal Vascular Parameters in Patients With Wilson's Disease
The rare disease reference center " Wilson disease and other rare copper-related diseases" of the Rothschild Foundation follows a large number of patients with Wilson's with varying degrees of impairment and located at different…
- Trial name
- WILLY (NCT04408300)
- Testing
- Ophthalmological exam
- Where
- France
- Volunteers
- 124 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
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'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 Disease
Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease
Hypothesis: The investigators postulate that patients with Wilson disease who are asymptomatic or who have been effectively treated for their symptoms and are in a maintenance phase therapy can be safely and effectively treated…
- Trial name
- NCT01472874
- Testing
- Once a day Trientine
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Yale University
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
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
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
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
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
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
Wilson Disease
Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
The study will evaluate the efficacy and safety of ALXN1840 (formerly called WTX101) administered for 48 weeks compared to standard of care (SoC) in Wilson Disease (WD) participants aged 12 and older in the Primary Evaluation…
- Trial name
- NCT03403205
- Testing
- ALXN1840, SoC Therapy
- Where
- 56 sites in United States, Australia and 20 more
- Volunteers
- 214 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Severe Dystonia, Wilson's Disease
WILSTIM - DBS (WILson STIMulation - Deep Brain Stimulation)
Dystonia in Wilson's disease represent a major issue. The persistence of disabling motor symptoms despite medical treatments justifies conducting a study on deep brain stimulation (DBS) in Wilson's disease (WD).
- Trial name
- WILSTIM DBS (NCT02552628)
- Testing
- Medtronic, Activa® PC "on", Medtronic, Activa® PC "off"
- Where
- 3 sites in France
- Volunteers
- 5 people
- Sponsor
- Hospices Civils de Lyon