Gene therapy for Wilson disease study with PET scans
Part of Brain & nervous system, Digestive system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial uses a special PET scan with a copper tracer to see how well gene therapy is working in people with Wilson disease. It may help doctors understand if the treatment is helping your body handle copper properly.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have Wilson disease and received gene therapy (UX701) in an earlier study called NCT04884815.
- You are 18 years old or older.
- If you could become pregnant, you agree to use birth control (like an IUD or hormonal methods) during the PET scans.
- You have a negative pregnancy test on the day of the first PET scan.
- You are not allergic to copper or the radioactive tracer used in the PET scan.
- You are not pregnant, breastfeeding, or planning to become pregnant.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial is testing an experimental gene therapy called PM577 that aims to fix the faulty gene causing Wilson disease. It might offer a new treatment option that could reduce or replace the need for daily medications.
This study tests a new gene therapy injection for people with Wilson disease. It aims to see if it is safe and can help improve symptoms.
This study is looking at what happens to copper levels in the urine when people with Wilson disease briefly stop their medicine. It may help doctors understand how well your body controls copper without treatment.
This study tests an injection called LY-M003 for people with Wilson disease who have already been treated with standard medicines. The goal is to see if it is safe and works for them.
This trial is for people who already have a genetic diagnosis of Wilson disease. It looks at whether specific lab tests and a physical eye finding can help confirm and support the diagnosis.
This study follows people who already have Wilson disease to understand how the condition changes over time. It may help researchers better predict future symptoms and improve care.
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