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NCT03334292 Clinical Trial

Natural History of Wilson Disease

Wilson Disease Clinical Trial

Official title:
Natural History of Wilson Disease: Registry for Patients With Wilson Disease

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT03334292
Other study ID # 1609018429
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date December 18, 2017
Est. completion date November 15, 2024

Study information
Verified date August 2023
Source Yale University
Contact Ricarda Tomlin
Phone (203) 785-2073
Email ricarda.tomlin@yale.edu
Is FDA regulated No
Health authority
Study type Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility
Status Recruiting
Enrollment 300
Est. completion date November 15, 2024
Est. primary completion date November 15, 2024
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility 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) Exclusion Criteria: - Diagnosis of WD has been excluded - Unwilling to provide informed consent or assent

Study Design

Related Conditions & MeSH terms

Locations
Country Name City State
Germany Universitätsklinikum Heidelberg Heidelberg
United Kingdom Royal Surrey Country Hospital Guildford Surrey
United States Baylor College of Medicine Houston Texas
United States Yale University New Haven Connecticut
United States Advent Health Orlando Florida
United States Seattle Children's Hospital Seattle Washington

Sponsors (2)
Lead Sponsor Collaborator
Yale University Wilson Disease Association

Countries where clinical trial is conducted

United States,  Germany,  United Kingdom, 

Outcome
Type Measure Description Time frame Safety issue
Primary 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. 5 Years
See also
  Status Clinical Trial Phase
Completed NCT04573309 - Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 Phase 2
Completed NCT03539952 - Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease Phase 3
Active, not recruiting NCT04884815 - Study of UX701 Gene Transfer for the Treatment of Wilson Disease Phase 1/Phase 2
Not yet recruiting NCT03659331 - A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease N/A
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Completed NCT04965571 - Clinical Features and Outcome of Wilson's Disease With Generalized Epilepsy in Chinese Patients
Terminated NCT05047523 - Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease Phase 3
Completed NCT04526210 - Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants Phase 1
Completed NCT00004338 - Study of Zinc for Wilson Disease Phase 4
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Completed NCT02273596 - Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients Phase 2
Completed NCT02763215 - The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment
Recruiting NCT05444127 - Oral Health and Wilson's Disease: SOMAWI
Completed NCT04408300 - Study of Retinal Vascular Parameters in Patients With Wilson's Disease N/A
Active, not recruiting NCT05783687 - Real World Evidence Study in Subjects With Wilson's Disease
Terminated NCT04909346 - Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
Completed NCT03867526 - Establishment of Human Cellular Disease Models for Wilson Disease
Enrolling by invitation NCT03589820 - Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease-related Liver Failure N/A
Completed NCT04526197 - Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants. Phase 1
Not yet recruiting NCT06430359 - Circadian Variation of Urinary Copper Excretion in Wilson Disease Patients