Study to Assess Safety and Effectiveness of LY-M003 Gene Therapy in Adults and Children With Wilson Disease

What this Trial Is About

Wilsons disease is a rare inherited disorder caused by mutations in the ATP7B gene, leading to copper buildup in organs like the liver, brain, and kidneys. Researchers are evaluating LY-M003 Injection, a gene therapy using an rAAV8 vector, to deliver the ATP7B gene to liver cells. This study aims to assess the safety, tolerability, effectiveness, immune response, and how the body processes this therapy in patients with Wilson disease. The study uses a single intravenous infusion of LY-M003 at one of four dose levels, ranging from 1.0 to 6.0 x 1013 vector genomes per kilogram. There are adult and pediatric groups, with dose escalation decisions based on safety and effectiveness data. Adult participants receive one of four dose levels, while pediatric dosing is determined after reviewing adult results. The study has a main phase and a long-term follow-up phase to monitor outcomes. Participants will be followed for 52 weeks after receiving the infusion. During this time, researchers will monitor adverse events, dose-limiting toxicities, changes in copper-related lab tests, liver function, neurological and psychiatric symptoms, and liver stiffness. They will also track reductions in standard Wilson disease medications. This comprehensive monitoring includes clinical assessments and laboratory tests to evaluate how participants respond to LY-M003 therapy over time.

A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease