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