Clinical trials: Wilson Disease
Genetic/Metabolic Liver Diseases
China Registry for Genetic / Metabolic Liver Diseases
CR-GMLD registry started on June 13, 2015 to collect cases of genetic/metabolic liver diseases from tertiary or secondary hospitals in mainland China.
- Trial name
- NCT03131427
- Testing
- Standard of care
- Where
- 12 sites in China
- Volunteers
- 20,000 people
- Sponsor
- Beijing Friendship Hospital
Wilson Disease
Natural History of Wilson Disease
The purpose of the registry/repository is to provide a mechanism to store data and specimens to support the conduct of future research about Wilson disease (WD).
- Trial name
- NCT03334292
- Where
- 6 sites in United States, Germany and 1 more
- Volunteers
- 300 people
- Sponsor
- Yale University
Wilson Disease
Study of Zinc for Wilson Disease
OBJECTIVES: I. Establish the safety and efficacy of extended maintenance zinc therapy in 200 patients with Wilson disease. II. Establish further the role of zinc in the prophylactic treatment of presymptomatic patients by…
- Trial name
- NCT00004338
- Testing
- zinc acetate
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- National Center for Research Resources (NCRR)
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
Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine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 Disease
Establishment of Human Cellular Disease Models for Wilson Disease
Establishment of human cellular disease models for Wilson disease for an individualized therapy develop-ment having the capacity to address both hepatic and neurologic forms of the disease
- Trial name
- IPSWILSON (NCT03867526)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
Wilson Disease
Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease
The primary objective of the study is to determine the relevance and appropriateness of outcome assessments, including biomarkers, within the Wilson disease population to inform study design and endpoint selection for future…
- Trial name
- NCT04531189
- Where
- 6 sites in United States, Germany
- Volunteers
- 16 people
- Sponsor
- Ultragenyx Pharmaceutical 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's Disease
Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson's Disease in Japan.
The purpose of this long-term study is to determine whether Zinc Acetate is effective and safe in the treatment of Wilson's disease among Japanese.
- Trial name
- NCT00212355
- Testing
- NPC-02
- Volunteers
- 37 people
- Sponsor
- Nobelpharma
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.
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'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
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, 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 Disease
Early Access Program for ALXN1840 in Patients With Wilson Disease
This is an open-label, single-arm, multi-center EAP, designed to provide early access to ALXN1840 for eligible patients with WD.
- Trial name
- NCT05686564
- Testing
- ALXN1840
- Sponsor
- Alexion Pharmaceuticals, Inc.
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 Disease
Study of Tetrathiomolybdate in Patients With Wilson Disease
OBJECTIVES: Evaluate the safety and efficacy of ammonium tetrathiomolybdate alone and compared with trientine therapy as initial treatment in patients with Wilson disease presenting neurologically.
- Trial name
- NCT00004339
- Testing
- tetrathiomolybdate, trientine
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- National Center for Research Resources (NCRR)
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
Wilson's Disease
Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.
The goal of this clinical trial is to learn if a new trientine tetrahydrochloride (TETA 4HCl) formulation administered once a day compared to d-Penicillamine (DPA) as a first line treatment for people living with Wilson's disease…
- Trial name
- TRADITiONAL (NCT07465718)
- Testing
- TETA 4HCl formulation, D-Penicillamine
- Where
- 10 sites in United States, China and 2 more
- Volunteers
- 38 people
- Sponsor
- Orphalan
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege