Clinical Trial

Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT

Enrolling by Invitation
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Record status
This record was last updated September 8, 2025 (before its estimated January 2026 completion). Its status may not reflect the trial's current state.
Summary
The primary objective of this study is to investigate the effect of gene therapy (UX704) on copper distribution and excretion in Wilson disease patients. The effect is investigated using 64Cu positron emission tomography scans combined with a CT scan.
Trial Details
NCT Number NCT07159581
Lead Sponsor Thomas Damgaard Sandahl
Collaborators: Ultragenyx Pharmaceutical Inc
Conditions Wilson Disease
Enrollment 5 participants
Start Date 2025-07-08
Primary Completion 2026-01 (estimated)
Study Completion 2026-01 (estimated)
Updated on ClinicalTrials.gov 2025-09-08