Natural History of Wilson Disease
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03334292|
Recruitment Status : Recruiting
First Posted : November 7, 2017
Last Update Posted : May 24, 2019
|Condition or disease|
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]|
|Estimated Enrollment :||300 participants|
|Target Follow-Up Duration:||5 Years|
|Official Title:||Natural History of Wilson Disease: Registry for Patients With Wilson Disease|
|Actual Study Start Date :||December 18, 2017|
|Estimated Primary Completion Date :||November 15, 2022|
|Estimated Study Completion Date :||November 15, 2027|
- Create registry for Wilson disease [ Time Frame: 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.
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03334292
|Contact: Ricarda Tomlin||(203) firstname.lastname@example.org|
|United States, Connecticut|
|New Haven, Connecticut, United States, 06520|
|Contact: Michelle Camarata, MD 203-737-3422 email@example.com|
|Principal Investigator: Michael Schilsky, MD|
|United States, Texas|
|Baylor College of Medicine||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Reza Amerinia|