Natural History of Wilson Disease

Status Recruiting

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.

Clinical Trial 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 Design

Related Conditions & MeSH terms

Wilson Disease

Clinical Trial Details

NCT number	NCT03334292
Study type	Observational [Patient Registry]
Source	Yale University
Contact	Ricarda Tomlin
Phone	(203) 785-2073
Email	ricarda.tomlin@yale.edu
Status	Recruiting
Phase
Start date	December 18, 2017
Completion date	November 15, 2024

View Details

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