Tiny trial uses PET scans to peek at gene therapy for wilson disease
NCT ID NCT07159581
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This study will use special PET scans to see how gene therapy (UX704) changes copper distribution in the bodies of 5 people with Wilson disease. The goal is to understand if the therapy helps the liver handle copper better. It is an early, observational imaging study, not a test of whether the therapy cures the disease.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- UX704 gene therapy
- What this could lead to
- If successful, this could show that gene therapy helps Wilson disease patients process copper better, pointing toward a more effective treatment.
- What could go wrong
- This is a very small study (5 people) that only looks at imaging results, not long-term health outcomes. It may not prove the therapy works or is safe.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Aarhus University Hospital
Aarhus, Aarhus N, 8200, Denmark
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Newborn screening study aims to catch rare diseases at birth
- Building a database to unlock wilson disease mysteries
- New Free-Breathing MRI could make liver scans easier for kids