Natural History of Wilson Disease

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Wilson disease
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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

Germany

Location Countries

Germany

Administrative Informations

NCT ID

NCT03334292

Organization ID

1609018429

Responsible Party

Sponsor

Study Sponsor

Yale University

Study Sponsor

, ,

Verification Date

July 2021