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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
Nonreplicating, recombinant gene transfer vector
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
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.
Time frame: At 1-Year post treatment
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.
Time frame: At 1-Year post treatment
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.
Time frame: At 1-Year post treatment
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.
Time frame: At 1-Year post treatment
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