A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Study of MSA-01 in Patients With Multiple System Atrophy
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 140
- 试验地点
- 12
- 主要终点
- Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 score
研究概览
简要总结
The purpose of this study is to evaluate whether MSA-01 slows the progression of multiple system atrophy (MSA) and to assess its safety.
The primary question is:
• Does MSA-01 slow the progression of motor impairment as measured by UMSARS Part 2 score?
Participants will be randomly assigned to receive MSA-01 or placebo for 12 months. They will attend regular clinic visits for safety and efficacy assessments and record their medication use and any side effects in a diary.
详细描述
Multiple system atrophy (MSA) is a progressive neurodegenerative disorder with no established disease-modifying treatment. Evidence suggests that coenzyme Q10 (CoQ10) deficiency may contribute to MSA pathophysiology. MSA-01 (ubiquinol), a highly bioavailable form of CoQ10, demonstrated acceptable safety and potential efficacy in a prior phase II trial.
This is a multicenter, randomized, double-blind, placebo-controlled phase III study evaluating the efficacy and safety of MSA-01 in patients with MSA. Approximately 140 participants will be randomized 1:1 to receive oral MSA-01 or placebo for 52 weeks.
The primary endpoint is the change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 score. Secondary endpoints include additional clinical scales and safety assessments. Efficacy will be analyzed using a mixed-effects model for repeated measures. The study aims to determine whether MSA-01 slows clinical progression compared with placebo while maintaining an acceptable safety profile.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 30 Years 至 79 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •At the time of informed consent
- •Patients diagnosed as 'clinically established' or 'clinically probable' MSA based on the revised MSA diagnostic criteria of the Movement Disorder Society (MDS).
- •Patients who are able to walk independently or with the use of assistive devices.
- •Patients who are able to attend outpatient visits at the participating study site.
- •At the start of study drug administration
- •Patients who are able to discontinue the use of CoQ10 supplements.
排除标准
- •Patients with severe neurological disorders, other progressive movement disorders, or cognitive impairment.
- •Patients with severe liver disease.
- •Patients with a known history of hypersensitivity to any component of the investigational drug.
- •Pregnant women, breastfeeding women, or women who may be pregnant.
- •Patients who have previously participated in a clinical trial of MSA-01.
研究组 & 干预措施
Placebo
干预措施: Placebo (Drug)
MSA-01
干预措施: MSA-01 (Drug)
结局指标
主要结局
Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 score
时间窗: Baseline to Week 52
次要结局
- Change from baseline to Week 52 in the Barthel Index(Baseline to Week 52)
- Change from baseline to Week 52 in the Scale for the Assessment and Rating of Ataxia (SARA) score(Baseline to Week 52)
- Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 1 score(Baseline to Week 52)
研究者
Jun Mitsui
Project Associate Professor
Tokyo University