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Enrolling by Invitation

NCT Number: NCT07159581

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

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.

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Key information

Conditions

Wilson Disease Basal Ganglia Diseases Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Hepatolenticular Degeneration Heredodegenerative Disorders, Nervous System Liver Diseases Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Movement Disorders Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Aarhus University Hospital

Aarhus, Aarhus N, 8200, Denmark

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients with Wilson disease who have received gene therapy (UX701) as part of NCT04884815
  • Age above 18 years
  • Women of childbearing potential must accept to use safe contraception at the time of the PET scans: Spiral or hormonal contraception (oral pills, implants, transdermal patches, vaginal rings, or depot injections)
  • At the day of the first PET scan, a negative urine pregnancy test is required for women of childbearing potential
  • Before the first PET scan, a signed informed consent must be completed

Exclusion criteria

  • Known hypersensitivity to Cu-64, copper in general, or other components of the radiotracer formulation
  • Claustrophobia
  • Pregnancy, breastfeeding, or plans to become pregnant

Treatment and study plan

Primary outcomes

  1. Ratio of liver SUV at 20 hours to liver SUV at 1 hours after injection of Cu64

    Time frame: From first to last scan

    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).

Secondary outcomes

  1. Gallbladder SUV at 1 hour, 6 hours and 20 hours after injection of 64Cu

    Time frame: From first to last scan

    SUV in a VOI covering the entire gallbladder

  2. Percentage of injected dose in the blood at 1 hour, 6 hours and 20 hours after injection of 64Cu

    Time frame: From first to last scan

    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).

Sponsors and collaborators

Lead sponsor

Thomas Damgaard Sandahl

Other

Collaborators

  • Ultragenyx Pharmaceutical Inc

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Sep 8, 2025
Registry last updated
Sep 8, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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