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

Shanghai Jiao Tong University

Status

Not yet enrolling

Conditions

Wilson Disease

Treatments

Genetic: MWAV201

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT06663878

2021YFC2700802 (Other Grant/Funding Number)

MWAV201-CP101

Details and patient eligibility

About

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

Full description

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.

Enrollment

9 estimated patients

Sex

All

Ages

18 to 65 years old

Volunteers

No Healthy Volunteers

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.