An Open-Label, Dose Escalation Study to Evaluate the Tolerability, Safety, and Preliminary Efficacy of MWAV201 in Subjects With Wilson Disease
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Safety and tolerability profile [including adverse events (AEs) and serious adverse events (SAEs)]
研究概览
简要总结
The primary objective of this study is to evaluate the tolerability and safety of MWAV201 in patients with Wilson disease.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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/m
- •Other conditions that, in the Investigator's opinion, may not be suitable for the subject to be enrolled.
研究组 & 干预措施
MWAV201
All subjects will receive a single intravenous (IV) infusion of MWAV201.
干预措施: MWAV201 (Genetic)
结局指标
主要结局
Safety and tolerability profile [including adverse events (AEs) and serious adverse events (SAEs)]
时间窗: At 1-Year post treatment
AEs will be summarized based on incidence and the date of onset for the event.
次要结局
- A Laboratory Outcome Measure: Total serum copper(At 1-Year post treatment)
- A Laboratory Outcome Measure: 24-hour urinary copper(At 1-Year post treatment)
- A Laboratory Outcome Measure: Aspartate aminotransferase to platelet ratio index(At 1-Year post treatment)