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

An Open-Label, Dose Escalation Study to Evaluate the Tolerability, Safety, and Preliminary Efficacy of MWAV201 in Subjects With Wilson Disease

The primary objective of this study is to evaluate the tolerability and safety of MWAV201 in patients 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.