Gene therapy for Wilson disease
Part of Brain & nervous system, Digestive system, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 or older.
- You must have a confirmed diagnosis of Wilson disease using specific tests (like low copper protein in blood or certain genetic changes).
- You cannot have had any gene therapy before.
- Your liver must not be severely damaged (no advanced liver disease or high risk of liver failure).
- You cannot be pregnant, breastfeeding, or planning to conceive within a year.
- You must not have serious infections like hepatitis B, hepatitis C, or HIV.
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 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.
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 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 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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