Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Wilson Disease. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Denmark
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Evaluation of the Effect of Gene Therapy for Wilson Disease With 64CuCl₂ PET Imaging
Overview
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.
Primary outcome measures
- Ratio of liver SUV at 20 hours to liver SUV at 1 hours after injection of Cu64 [Time frame: From first to last scan]
Secondary outcome measures (2)
- Gallbladder SUV at 1 hour, 6 hours and 20 hours after injection of 64Cu [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 [Time frame: From first to last scan]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Denmark · 1 center
- Aarhus University Hospital — Aarhus
Publications
- Kirk FT, Munk DE, Swenson ES, Quicquaro AM, Vendelbo MH, Larsen A, Schilsky ML, Ott P, Sandahl TD. Effects of tetrathiomolybdate on copper metabolism in healthy volunteers and in patients with Wilson disease. J Hepatol. 2024 Apr;80(4):586-595. doi: 10.1016/j.jhep.2023.11.023. Epub 2023 Dec 10. PMID 38081365
- Sandahl TD, Gormsen LC, Kjaergaard K, Vendelbo MH, Munk DE, Munk OL, Bender D, Keiding S, Vase KH, Frisch K, Vilstrup H, Ott P. The pathophysiology of Wilson's disease visualized: A human 64 Cu PET study. Hepatology. 2022 Jun;75(6):1461-1470. doi: 10.1002/hep.32238. Epub 2022 Jan 10. PMID 34773664
Identifiers
NCT: NCT07159581 · 1-10-72-146-24