Clinical trials: Wilson's Disease
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's Disease
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
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…
- Trial name
- GATEWAY (NCT04537377)
- Testing
- VTX-801
- Where
- 10 sites in United States, Denmark and 2 more
- Volunteers
- 4 people
- Sponsor
- Vivet Therapeutics SAS
Wilson Disease
Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.
This was a Phase 1, randomized, 2-period, 2-sequence, cross-over study designed to determine the effect of ALXN1840 on the metabolism of celecoxib, a sensitive cytochrome P450 2C9 (CYP2C9) substrate, in healthy male and female…
- Trial name
- NCT04526197
- Testing
- ALXN1840, Celecoxib
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, 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
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
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
Ornithine Transcarbamylase Deficiency, Wilson Disease
Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
The objective of this observational study is to evaluate the seroprevalence of anti-AAV antibodies in subjects with Ornithine Transcarbamylase (OTC) deficiency, Glycogen Storage Disease Type Ia (GSDIa), and Wilson Disease
- Conditions
- Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
- Trial name
- NCT04909346
- Where
- 5 sites in United States, Brazil and 3 more
- Volunteers
- 51 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
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
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
Wilson's Disease, Myocardial Dysfunction
Subclinical Myocardial Dysfunction in Children With Wilson's Disease
Wilson's disease (WD) is one of the most common metabolic liver diseases in older children. The most frequent clinical presentation is liver disease. However, Wilson's disease (WD) is a multisystem disorder.
- Conditions
- Wilson's Disease, Myocardial Dysfunction, Children, Wilson Disease
- Trial name
- NCT07765472
- Testing
- Echocardiography, Electrocardiography, N-terminal pro b- type natriuretic peptide
- Where
- 2 sites in Egypt
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Hebatullah Fawzy
Wilson Disease
Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease
This is a multicenter, randomized, open-label study with an active standard-of-care comparator (penicillamine)
- Trial name
- NCT03539952
- Testing
- Penicillamine (D1-W12), TETA 4HCL (W12-60), Penicillamine (W12-W60)
- Where
- 15 sites in United States, Belgium and 7 more
- Volunteers
- 77 people
- Sponsor
- Orphalan
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 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.
Clinical Outcome in WILSON Disease
Clinical Profile and Out Come of Children With Wilson's Disease
Clinical profile and out come of Children with Wilson's disease :A single tertiary center study
- Trial name
- NCT05792319
- Testing
- 24 hour copper in urine and liver function test
- Volunteers
- 20 people
- Sponsor
- Assiut University
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
Wison's Disease
Efficacy and Safety Study of Zinc Acetate to Treat Wilson's Disease in Japan.
The purpose of this study is to determine whether Zinc Acetate is effective and safe in the treatment of Wilson's disease among Japanese.
- Trial name
- NCT00212368
- Testing
- Zinc acetate
- Sponsor
- Nobelpharma
Wilson Disease
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
The primary objectives of this study are to evaluate the safety of single IV doses of UX701 in patients with Wilson disease, to select the UX701 dose with the best benefit/risk profile based on the totality of safety and efficacy…
- Trial name
- NCT04884815
- Testing
- UX701, Standard of Care (SOC)
- Where
- 16 sites in United States, Portugal and 2 more
- Volunteers
- 82 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Wilson Disease
The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment
This was a 24-month study to assess copper parameters in participants with Wilson disease (WD) treated with standard of care (SoC) medications.
- Trial name
- NCT02763215
- Testing
- Standard of Care Medications
- Where
- 10 sites in United States, Austria and 3 more
- Volunteers
- 64 people
- Sponsor
- Alexion Pharmaceuticals, Inc.