Wilson Disease
Conditions
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.
Interventions
GENETICMWAV201
Nonreplicating, recombinant gene transfer vector
Sponsors
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Mabwell (Shanghai) Bioscience Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Participants will receive a single, peripheral intravenous (IV) infusion of MWAV201.
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
China
Contacts
PRINCIPAL_INVESTIGATORJian-Gao Fan, doctor
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Outcome results
None listed