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Enrolling By InvitationNCT07159581

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

Evaluation of the Effect of Gene Therapy for Wilson Disease With 64CuCl₂ PET Imaging

Status
Enrolling By Invitation
Phase
Study type
Observational
Enrollment
5 (estimated)
Sponsor
Thomas Damgaard Sandahl · Academic / Other
Sex
All
Age
18 Years
Healthy volunteers
Not accepted

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.

Detailed description

Patients entering this study are included from the ongoing clinical study "A Phase 1/ 2/ 3 Study of UX701 Gene Therapy in Adults With Wilson Disease" (NCT04884815). The patients will have received gene therapy as part of the clinical study. This study is an extension study that aims to specifically monitor the effect of gene therapy on copper distribution and excretion using 64Cu PET/CT. Patients will receive 40-80 MBq 64CuCl2 intravenously and will undergo three PET/CT scans during the next 20 hours.

Conditions

Timeline

Start date
2025-07-08
Primary completion
2026-01-01
Completion
2026-01-01
First posted
2025-09-08
Last updated
2025-09-08

Locations

1 site across 1 country: Denmark

Source: ClinicalTrials.gov record NCT07159581. Inclusion in this directory is not an endorsement.