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.
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
Enrollment
300
Yale University
New Haven, Connecticut, United States
RECRUITINGAdvent Health
Orlando, Florida, United States
RECRUITINGBaylor College of Medicine
Houston, Texas, United States
Create registry for Wilson disease
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.
Time frame: 5 Years
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Seattle Children's Hospital
Seattle, Washington, United States
RECRUITINGUniversitätsklinikum Heidelberg
Heidelberg, Germany
RECRUITINGRoyal Surrey Country Hospital
Guildford, Surrey, United Kingdom
RECRUITING