Trials / Completed
CompletedNCT04531189
Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 16 (actual)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- 12 Years
- Healthy volunteers
- Not accepted
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.
Detailed 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.
Conditions
Timeline
- Start date
- 2020-12-11
- Primary completion
- 2022-03-25
- Completion
- 2022-03-25
- First posted
- 2020-08-28
- Last updated
- 2022-04-29
Locations
6 sites across 2 countries: United States, Germany
Source: ClinicalTrials.gov record NCT04531189. Inclusion in this directory is not an endorsement.