Natural History of Wilson Disease

Last updated: May 21, 2025
Sponsor: Yale University
Overall Status: Active - Recruiting

Phase

N/A

Condition

Wilson's Disease

Liver Disease

Kidney Failure

Treatment

N/A

Clinical Study ID

NCT03334292
1609018429
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Known diagnosis of WD

  • Able and willing to provide informed consent for adults (Parental/guardianpermission (informed consent) and if appropriate, child assent for participants <18 (or per local Institutional Review Board (IRB) regulation)

Exclusion

Exclusion Criteria:

  • Diagnosis of WD has been excluded

  • Unwilling to provide informed consent or assent

Study Design

Total Participants: 300
Study Start date:
December 18, 2017
Estimated Completion Date:
November 15, 2029

Study 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.

Connect with a study center

  • Universitätsklinikum Heidelberg

    Heidelberg, 69120
    Germany

    Active - Recruiting

  • Royal Surrey Country Hospital

    Guildford, Surrey GU2
    United Kingdom

    Active - Recruiting

  • Yale University

    New Haven, Connecticut 06520
    United States

    Active - Recruiting

  • Advent Health

    Orlando, Florida 32803
    United States

    Active - Recruiting

  • Baylor College of Medicine

    Houston, Texas 77030
    United States

    Active - Recruiting

  • Seattle Children's Hospital

    Seattle, Washington 98105
    United States

    Active - Recruiting

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