Gene therapy for Wilson's disease
Part of Brain & nervous system, Digestive system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests a new gene therapy called LY-M003 that may help people with Wilson's disease by fixing the faulty gene that causes copper buildup. It's for adults who are already on standard treatment and have a confirmed genetic diagnosis.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of Wilson's disease, including a genetic test showing the ATP7B gene mutation.
- You must have been on standard treatment (like D-penicillamine or zinc acetate) for at least 6 months before joining.
- You must have been following a low-copper diet for at least 6 months and be willing to continue it during the study.
- You must agree not to donate blood, organs, tissues, or cells after treatment.
- If you are of childbearing age, you must have a negative pregnancy test and use effective birth control during the study and for 6 months after.
- You must not have severe liver disease, active bleeding, or certain infections (like hepatitis B, 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 tests an experimental mRNA treatment (DSL101) for adults with Wilson disease. It's for people whose condition is stable on current therapy and who have certain genetic markers. The goal is to see if the new treatment can help the body process copper better and reduce the need for standard medicines.
This study follows people who have the genetic cause of Wilson disease, including people who feel well and family members. Researchers want to better understand Wilson disease and what to watch for over time.
This study measures how much copper is present in breast milk in people with Wilson’s disease. It aims to provide safer guidance for breastfeeding during treatment, including during pregnancy.
This trial tests a once-daily trientine pill as the first treatment for Wilson's disease, a condition where copper builds up in the body. It may help if you've never been treated for Wilson's disease or have only had zinc therapy briefly.
This trial is a patient registry that collects health information from people with Wilson’s disease, including those without symptoms yet and those taking any type of treatment. It helps researchers understand the disease and how it progresses, which can support future studies and care improvements.
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