Natural History of Wilson Disease

checkorphan
Learn more about:
Wilson disease
Related Clinical Trial
Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 A Phase I/II Study of VTX-801 in Adult Patients With Wilson’s Disease Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants. Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease Copper Concentration and Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840 Study of Retinal Vascular Parameters in Patients With Wilson’s Disease Evaluation of Patients With Liver Disease ExAblate Transcranial MRgFUS for the Management of Treatment-Refractory Movement Disorders The Assessment of Copper Parameters in Wilson Disease Subjects on Standard of Care Treatment Establishment of Human Cellular Disease Models for Wilson Disease Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson’s Disease in Japan. Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson’s Disease-related Liver Failure The Individual Therapy for Patients With Wilson’s Disease Study of Zinc for Wilson Disease Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson’s Disease Study of Tetrathiomolybdate in Patients With Wilson Disease Efficacy and Safety of ALXN1840 (Formerly Named WTX101) Administered for 48 Weeks Versus Standard of Care in Patients With Wilson Disease With an Extension Period of up to 60 Months WILSTIM – DBS (WILson STIMulation – Deep Brain Stimulation) Efficacy and Safety Study of Zinc Acetate to Treat Wilson’s Disease in Japan. Efficacy of Invitro Expanded Bone Marrow Derived Allogeneic Mesenchymal Stem Cell Transplantation Via Portal Vein or Hepatic Artery or Peripheral Vein in Patients With Wilson Cirrhosis Inhibitory rTMS in Dystonic Wilson Patients Natural History of Wilson Disease A Registered Cohort Study on Wilson’s Disease sCD163 and sMR in Wilsons Disease – Associations With Disease Severity and Fibrosis Cohort Research on Wilson’s Disease Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson’s Disease Patients Efficacy and Safety Study of WTX101 in Adult Wilson Disease Patients

Brief Title

Natural History of Wilson Disease

Official Title

Natural History of Wilson Disease: Registry for Patients With Wilson Disease

Brief Summary

      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). The overall aim is to
      determine the optimal testing for diagnosis and parameters for monitoring treatment of WD
      that will aid product utilization and development.

Detailed Description

      There are three aims outlined as part of this research study.

      Aim 1 is to study the natural history of a carefully characterized cohort of patients with WD
      followed longitudinally at Centers of Excellence for WD in the United States and in the
      United Kingdom.

      Aim 2 seeks to evaluate parameters for diagnosis and treatment monitoring for patients on
      chelation therapy and zinc treatment for their WD. Data gathered in Specific aim 1 will be
      used for analyzing the components of the diagnostic scores for patients.

      Aim 3 is intended to determine whether a composite index or a biomarker can be used as
      surrogate marker for treatment monitoring for current patients on therapy that can be used
      for future patient treatment trials.

Study Type

Observational [Patient Registry]

Primary Outcome

Create registry for Wilson disease

Condition

Wilson Disease

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Estimated Enrollment

300

Start Date

December 18, 2017

Completion Date

November 15, 2027

Primary Completion Date

November 15, 2022

Eligibility Criteria

        Inclusion Criteria:

          -  Known diagnosis of WD

          -  Able and willing to provide informed consent for adults (Parental/guardian permission
             (informed consent) and if appropriate, child assent for participants <18 (or per local
             IRB regulation)

        Exclusion Criteria:

          -  Diagnosis of WD has been excluded

          -  Unwilling to provide informed consent or assent

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

, (203) 785-2073, [email protected]

Location Countries

United Kingdom

Location Countries

United Kingdom

Administrative Informations

NCT ID

NCT03334292

Organization ID

1609018429

Responsible Party

Sponsor

Study Sponsor

Yale University

Study Sponsor

, ,

Verification Date

July 2020