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

Not Applicable

Not yet recruiting

• Conditions: Wilson Disease

• Registration Number: NCT06663878
• Lead Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Brief Summary

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

Detailed 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.

Study Timeline

• Status Verified: 2024-10
• Study Start: 2024-10
• Study First Submitted: 2024-10-10
• Study First Submitted QC: 2024-10-27
• Last Update Submitted: 2024-10-27
• Study First Posted: 2024-10-29
• Last Update Posted: 2024-10-29
• Primary Completion: 2026-03
• Study Completion: 2031-05

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 9

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Safety and tolerability profile [including adverse events (AEs) and serious adverse events (SAEs)]	At 1-Year post treatment	AEs will be summarized based on incidence and the date of onset for the event.

Secondary Outcome Measures

Name	Time	Method
A Laboratory Outcome Measure: Total serum copper	At 1-Year post treatment	Total serum copper will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values, changes from baseline and percent change from baseline.
A Laboratory Outcome Measure: 24-hour urinary copper	At 1-Year post treatment	24-hour urinary copper will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values, changes from baseline and percent change from baseline.
A Laboratory Outcome Measure: Aspartate aminotransferase to platelet ratio index	At 1-Year post treatment	Aspartate aminotransferase to platelet ratio index will be summarized descriptively for all patients by dose cohort and planned visit, for absolute values, changes from baseline and percent change from baseline.

Trial Locations

Locations (1)

Xinhua Hospital, Shanghai Jiao Tong University School of Medicine; 🇨🇳 Shanghai, Shanghai, China