Phase III Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar Degeneration
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 142
- 试验地点
- 2
- 主要终点
- Change from baseline in SARA total score at 24 weeks of treatment period
研究概览
简要总结
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, phase III study to evaluate the efficacy and safety of KPS-0373 given once daily after breakfast in 142 patients with spinocerebellar degeneration . This study consists of the 4-week screening period, 24-week treatment period, and 4-week follow-up period.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Japanese SCD patients with mild to moderate ataxia
排除标准
- •Patients with secondary ataxia
- •Patients with clinically significant hepatic, renal, or cardiovascular dysfunction
研究组 & 干预措施
Placebo group
One placebo tablet is orally administered once daily after breakfast.
干预措施: Placebo (Drug)
KPS-0373 group
One KPS-0373 2.4 mg tablet is orally administered once daily after breakfast.
干预措施: KPS-0373 (Drug)
结局指标
主要结局
Change from baseline in SARA total score at 24 weeks of treatment period
时间窗: Up to 24 weeks
The SARA is a scale that assesses cerebellar ataxia. An evaluator observes the subject's ability to perform motor activities across in 8 individual items: ("gait: 0-8 points," "stance: 0-6 points," "sitting: 0-4 points," "speech disturbance: 0-6 points," "finger chase: 0-4 points," "nose-finger test: 0-4 points," "fast alternating hand movements: 0-4 points," and "heel-shin slide: 0-4 points." The total SARA score ranges from 0 (no ataxia) to 40 (severe ataxia).
次要结局
- Change from baseline in SARA total score(Up to 24 weeks)
- Symptom Maintenance Ratio (Ratio of Improvement and Stability Cases) based on SARA total score(Up to 24 weeks)
- Change from baseline in SARA individual scores(Up to 24 weeks)
- Change from baseline in SF-8(Up to 24 weeks)