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Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease

Wilson Disease Clinical Trial

Official title:
Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease

Clinical Trial Summary

The primary objective of the study is to determine the relevance and appropriateness of outcome assessments, including biomarkers, within the Wilson disease population to inform study design and endpoint selection for future clinical studies.

Clinical Trial Description

Given the limited information on the frequency and spectrum of disease manifestations and clinical course of Wilson disease, the UX701-CL001 study aims to assess the utility and feasibility of various assessments and biomarkers to inform endpoint selection for future clinical studies, better understand the relationship between biomarkers and potential clinical outcomes, and characterize the clinical presentation of Wilson disease. UX701-CL001 is a clinical survey study. Subjects will complete assessments at the study site and at home to evaluate the clinical manifestations of Wilson disease in clinical and real-world environments. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04531189
Study type Observational
Source Ultragenyx Pharmaceutical Inc
Contact
Status Completed
Phase
Start date December 11, 2020
Completion date March 25, 2022
View Details
See also
  Status Clinical Trial Phase
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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
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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