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.
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.
Study Type
OBSERVATIONAL
Enrollment
5
Aarhus University Hospital
Aarhus, Aarhus N, Denmark
Ratio of liver SUV at 20 hours to liver SUV at 1 hours after injection of Cu64
The liver SUV is measured as the mean of five spherical VOIs (20 mm in diameter) placed in the right liver lobe, with distance to the organ borders and large blood vessels. The ratio is a measure of whether the tracer is retained in the liver (ratio \> 1) or is excreted from the liver (ratio \< 1).
Time frame: From first to last scan
Gallbladder SUV at 1 hour, 6 hours and 20 hours after injection of 64Cu
SUV in a VOI covering the entire gallbladder
Time frame: From first to last scan
Percentage of injected dose in the blood at 1 hour, 6 hours and 20 hours after injection of 64Cu
Radioactivity in the blood is measured by placing a cylindrical VOI in the descending aorta. From this VOI, the kilobecquerel/ml is found. The percentage of injected dose is found by correcting for decay and multiplying by the organ volume (estimated blood volume 5% of body weight, and the density of blood is 1 g/ml).
Time frame: From first to last scan
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