An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: MWAV201.
- Who it may be relevant to
- Registry conditions: Wilson Disease. Basic parameters: 18 years — 65 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Open-Label, Dose Escalation Study to Evaluate the Tolerability, Safety, and Preliminary Efficacy of MWAV201 in Subjects With Wilson Disease
Overview
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
- Genetic MWAV201
Nonreplicating, recombinant gene transfer vector
Primary outcome measures
- Safety and tolerability profile [including adverse events (AEs) and serious adverse events (SAEs)] [Time frame: At 1-Year post treatment]
Secondary outcome measures (3)
- A Laboratory Outcome Measure: Total serum copper [Time frame: At 1-Year post treatment]
- A Laboratory Outcome Measure: 24-hour urinary copper [Time frame: At 1-Year post treatment]
- A Laboratory Outcome Measure: Aspartate aminotransferase to platelet ratio index [Time frame: At 1-Year post treatment]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine — Shanghai
Identifiers
NCT: NCT06663878 · MWAV201-CP101 · 2021YFC2700802