An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease

This is an open-label, dose escalation study to evaluate the tolerability, safety, and preliminary efficacy of MWAV201 in patients with Wilson disease. Participants will receive a single, peripheral intravenous (IV) infusion of MWAV201. The dose escalation plan and the number of dose levels may be adjusted during study period.

Inclusion criteria

• Male or female aged 18 and 65 years inclusive;
• Confirmed diagnosis of Wilson disease;
• Low copper diet and standardized medication for ≥ 1 year;
• Stable Wilson disease for ≥ 1 year;
• Able to understand and willing to follow study procedures.

Exclusion criteria

• Significant hepatic inflammation as evidenced by liver function test.
• Liver biopsy or liver stiffness measurement show progressive liver fibrosis.
• Laboratory tests or clinical symptoms indicate decreased liver reserve function.
• Other chronic liver disease (such as hepatitis B).
• Any signs of decompensated liver function (such as ascites).
• History of liver transplant or plan to receive liver transplant.
• Other diseases with clinical significance, such as cardiovascular and cerebrovascular diseases, kidney diseases, respiratory system diseases, neurological diseases, mental illnesses, active infections, etc.
• Body Mass Index ≥ 30 kg/m2.
• Other conditions that, in the Investigator's opinion, may not be suitable for the subject to be enrolled.