The primary objective of this study is to evaluate the tolerability and safety of MWAV201 in patients with Wilson disease.
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;
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).
Single group
MWAV201
GeneticNonreplicating, recombinant gene transfer vector
Safety and tolerability profile [including adverse events (AEs) and serious adverse events (SAEs)]
AEs will be summarized based on incidence and the date of onset for the event.
A Laboratory Outcome Measure: Total serum copper
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
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
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.
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Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Lead sponsor
Mabwell (Shanghai) Bioscience Co., Ltd.
Collaborator
This trial is not recruiting at the moment. You can still explore other options:
