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.
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 Type
OBSERVATIONAL
Enrollment
16
Yale University School of Medicine
New Haven, Connecticut, United States
Jackson Memorial Hospital Miami Transplant Unit University of Miami Miller School of Medicine
Miami, Florida, United States
Northwestern University Feinberg School of Medicine
Chicago, Illinois, United States
University of Michigan Michigan Medicine Hepatology Clinic Taubman Center
Clinical manifestation of Wilson Disease under study: demographics
Time frame: 30 days
Clinical manifestation of Wilson Disease under study: general medical history, Wilson Disease history and treatments
Time frame: 30 days
Clinical manifestation of Wilson Disease under study: patient reported outcomes and clinician reported outcomes
Time frame: 30 days
Clinical manifestation of Wilson Disease under study: activity monitoring
Time frame: 30 days
Clinical manifestation of Wilson Disease under study: motor function
Time frame: 30 days
Clinical manifestation of Wilson Disease under study: joint pain
Time frame: 30 days
Clinical manifestation of Wilson Disease under study: serum copper biomarker assessments
Time frame: 30 days
Clinical manifestation of Wilson Disease under study: 24-hour urinary copper concentration
Time frame: 30 days
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Ann Arbor, Michigan, United States
Seattle Children's Hospital
Seattle, Washington, United States
Universitätsklinikum Heidelberg
Heidelberg, Baden-Wurttemberg, Germany