Gene therapy for Wilson disease
Part of Brain & nervous system, Digestive system, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of Wilson disease
- Your genetic test must show the H1069Q mutation in at least one of your two genes
- You must have been on the same Wilson disease medication for 6 months without needing dose changes
- Your copper levels must be controlled at screening
- You must be willing to avoid alcohol until 3 months after the infusion and limit it afterward
- You must agree to be followed for about 15 years after the treatment
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 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 early-stage trial tests LY-M003, a new gene therapy treatment for Wilson's disease—a rare genetic condition where copper builds up in the body. The study aims to see if this injection is safe and whether it can help patients who are already on standard copper-lowering medicines.
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 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 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 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.
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