Clinical trials: Wilson's Disease
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.
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'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
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
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.
Lysosomal Acid Lipase Deficiency
A Study to Identify and Characterize LAL-D Patients in High-risk Populations
The objective of this study is to determine the frequency of Lysosomal Acid Lipase Deficiency (LAL D) by lysosomal acid lipase (LAL) enzyme activity assay in patients who are considered to be at risk.
- Trial name
- NCT02345421
- Where
- 15 sites in United States
- Volunteers
- 640 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Wilsons Disease
A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)
This study adopted an open, single-arm, non-randomized, dose-escalation research design, aiming to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetic and immunogenicity characteristics of single and multiple…
- Trial name
- NCT07240896
- Testing
- Group 1: DSL101 Low dose, Group 2: DSL101 Medium dose, Group 3: DSL101 High dose
- Where
- China
- Volunteers
- 18 people
- Sponsor
- DSciLab Co., Ltd.
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.
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
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
The main objective of the study is to evaluate the change in liver copper (Cu) concentration following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD) who have been previously treated for at…
- Trial name
- NCT04422431
- Testing
- Bis-Choline Tetrathiomolybdate
- Where
- 12 sites in United States, Canada and 6 more
- Volunteers
- 31 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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.
Movement Disorders, Essential Tremor
ExAblate Transcranial MRgFUS for the Management of Treatment-Refractory Movement Disorders
The proposed study is to evaluate the effectiveness of ExAblate Transcranial MRgFUS as a tool for creating a unilateral lesion in the Vim thalamus or the globus pallidus (GPi) in patients with treatment-refractory symptoms of…
- Conditions
- Movement Disorders, Essential Tremor, Holmes Tremor, Parkinson's Disease
- Trial name
- NCT02252380
- Testing
- Transcranial ExAblate System
- Where
- 2 sites in Canada
- Volunteers
- 10 people
- Sponsor
- InSightec
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
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's Disease
Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily).
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…
- Trial name
- NCT06128954
- Testing
- 900mg TETA 4HCl Once Daily Formulation, 900mg TETA 4HCl Cuprior®
- Where
- United Kingdom
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Orphalan
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
Healthy
A Study of the Cardiac Effects of ALXN1840 in Healthy Adults
This study will evaluate the effect of a supratherapeutic dose of ALXN1840 on the heart rate (HR)-corrected QT interval (QTc) in healthy adult participants. Moxifloxacin will be used as the active control.
- Trial name
- NCT04560816
- Testing
- ALXN1840, Moxifloxacin
- Where
- United States
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Wilson's Disease
Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride
Decentralized study to assess patient reported treatment satisfaction comparing their current standard-of-care Wilson's Disease (WD) treatment with a new once-daily Trientine (TETA) 4HCl formulation.
- Trial name
- NCT07010575
- Testing
- New TETA 4HCl Formulation, Standard of Care
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- Orphalan
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 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.