Natural History of Wilson Disease

Recruiting

• Conditions: Wilson Disease

• Registration Number: NCT03334292
• Lead Sponsor: Yale University

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 Timeline

• Study First Submitted: 2017-10-31
• Study First Submitted QC: 2017-11-02
• Study First Posted: 2017-11-07
• Study Start: 2017-12-18
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-31
• Last Update Posted: 2024-08-01
• Primary Completion: 2029-11-15
• Study Completion: 2029-11-15

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 300

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 Institutional Review Board (IRB) regulation)

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Exclusion Criteria

• Diagnosis of WD has been excluded
• Unwilling to provide informed consent or assent

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Create registry for Wilson disease	5 Years	This outcome is a binary 'yes/no' outcome as to whether or not this study can successfully create a repository with the intent to store data and specimens to support the conduct of future research on Wilson disease.

Trial Locations

Locations (6)

Baylor College of Medicine; 🇺🇸 Houston, Texas, United States

Yale University; 🇺🇸 New Haven, Connecticut, United States

Royal Surrey Country Hospital; 🇬🇧 Guildford, Surrey, United Kingdom

Seattle Children's Hospital; 🇺🇸 Seattle, Washington, United States

Advent Health; 🇺🇸 Orlando, Florida, United States

Universitätsklinikum Heidelberg; 🇩🇪 Heidelberg, Germany