Gene Therapy for Wilson's Disease
Part of Brain & nervous system, Digestive system, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have been diagnosed with Wilson's disease and have a confirmed ATP7B gene mutation
- You've been on standard Wilson's disease treatment (like D-penicillamine or zinc) for at least 6 months
- You've followed a strict low-copper diet for at least 6 months and can continue it throughout the study
- Your liver disease is stable (no severe cirrhosis, bleeding, or organ failure)
- You cannot be pregnant, planning pregnancy, or breastfeeding; effective contraception required for 6 months after treatment
- You have not received any previous gene therapy or cell therapy
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 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.
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.
Hear when a new Wilson's Disease trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.