A Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular Dystrophy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 36
- 试验地点
- 11
- 主要终点
- Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)
研究概览
简要总结
The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner.
详细描述
Duchenne Muscular Dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in 3,500 lives male births. DMD patients suffer from a relentless decline in muscle strength that impairs the ability of walking and breathing, resulting in their lives with wheelchairs and then loss of upper body function. The main objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with DMD in an exploratory manner. The objective of this study is also to evaluate the safety, the dose-response and the urinary excretion of pharmacodynamic (PD) marker after 24-week repeated oral doses of TAS-205 in DMD patients.
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
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Able to give an informed consent. If applicable, able to give an informed assent.
- •Phenotypic evidence of DMD.
- •Male and ≧5 years of age.
- •Bodyweight ≧7.5 kg and <60 kg.
- •Able to complete the 6MWD test with a distance of at least 75 m.
- •Able to take tablets.
- •If taking oral glucocorticoids no significant change in the total daily or dosing 6 months before enrollment.
排除标准
- •Any serious drug allergy.
- •A forced vital capacity (FVC) of <50% of predicted value.
- •Wearing a respirator continuously (except for the use during sleep).
- •A left ventricular ejection fraction (EF) of <40% or fractional shortening (FS) of <25% on echocardiogram.
- •Clinically significant cardiac failure and respiratory failure.
- •Ongoing immunosuppressive therapy (other than corticosteroids) .
- •Surgical history or plan for surgery that may affect muscular strength or motor function.
- •Any injury that may affect muscular strength or motor function.
- •With any systemic allergic disease or any chronic inflammatory disease.
- •Previous gene therapy (exon skipping, or stop codon read through therapy), cell-based therapy, or any other investigational agents.
研究组 & 干预措施
TAS-205（Low dose group）
Low dose group：Oral administration of tablets for 24 weeks, bis in die (BID) after meal The number of tablets of the study drug corresponding to the dosage (6.67-13.33 mg/kg/dose) by body weight within 14 days before enrollment was to be administered within 30 minutes after breakfast and dinner.
干预措施: TAS-205 (Drug)
TAS-205（High dose group）
High dose group: Oral administration of tablets for 24 weeks, bis in die (BID) after meal The number of tablets of the study drug corresponding to the dosage (13.33-26.67 mg/kg/dose) by body weight within 14 days before enrollment was to be administered within 30 minutes after breakfast and dinner.
干预措施: TAS-205 (Drug)
Placebo
Placebo group: Oral administration of tablets for 24 weeks, BID after meal
干预措施: Placebo (Drug)
结局指标
主要结局
Mean Change From Baseline to 24 Weeks in the 6-minute Walk Distance (6MWD)
时间窗: baseline, 24 weeks
The distance the subject can walk as fast as possible in 6 minutes will be evaluated.
次要结局
- Mean Change From Baseline in Time to Rise From the Floor(baseline, and 24 weeks)
- Mean Change From Baseline in Time to Walk/Run for 10meters(baseline, and 24 weeks)
- Mean Change From Baseline in Time to up and go (TUG)(baseline, and 24 weeks)