A Phase I Study of Single and Multiple Doses of TAS-205 in Patients With Duchenne Muscular Dystrophy
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 23
- 试验地点
- 1
- 主要终点
- Incidence of Adverse Events
研究概览
简要总结
The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.
详细描述
Duchenne Muscular Dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3500 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 loss of upper body function. The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 after single and multiple doses in DMD patients. It is also evaluated if TAS-205 affects the urinary excretion of pharmacodynamic (PD) marker 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 15 Years（Child）
- 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.
- •Male and >= 5 years and < 16 years of age.
- •Bodyweight of >= 15.0 kg and < 75.0 kg.
- •Phenotypic evidence of DMD.
- •Able to take tablets.
- •If taking oral glucocorticosteroids no significant change in total daily dosage or dosing regimen after enrollment.
- •Confirmed the urinary PD marker over its criteria.
- •Able to follow the study protocol.
排除标准
- •Current diagnosis or history of any drug allergy.
- •A forced vital capacity (FVC) < 50% of predicted value.
- •A left ventricular ejection fraction (EF) < 50% or fractional shortening (FS) < 25% based on echocardiogram (ECHO).
- •Ongoing immunosuppressive therapy (other than corticosteroids).
- •With severe disease such as hepatic disease, kidney disease and others.
- •With any systemic allergic disease or any chronic inflammatory disease.
- •Treated with any other investigational agents within 90 days.
- •Positive reaction in hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test.
研究组 & 干预措施
Placebo
干预措施: Placebo (Drug)
TAS-205 low dose
干预措施: TAS-205 (Drug)
TAS-205 middle dose
干预措施: TAS-205 (Drug)
TAS-205 high dose
干预措施: TAS-205 (Drug)
结局指标
主要结局
Incidence of Adverse Events
时间窗: From the first administration day to the end of the observation period (ie. single-dose phase: 8 days, multiple-doses phase: 14 days)
Source Vocabulary Name for Table Default: CTCAE (4.03)
次要结局
- Peak Plasma Concentration (Cmax) of TAS-205(Single-dose phase: immediately before dosing, 0, 0.5, 1, 2, 4, 8, 24, 48 hours post-dose, Multiple-dose phase: Days 1 and 7, immediately before morning dose, 0.5, 1, 2, 4, and 8 hours post-dose and Day 4, immediately before morning dose.)
- Area Under the Plasma Concentration Versus Time Curve (AUC) of TAS-205(Administration period (ie. single-dose phase: from single administration day to 48 hours after the administration, multiple-dose phase: from the first administration day to 8 hours after the last administration))
- The Urinary Excretion of PD Marker(Single-dose: Day -1 before administration, 0-24 hr post-dose, and 24-48 hr post-dose, Multiple-doses: Day -1 before administration, 0 hr after administration on Day 1 and 4 to the following day (Day 2 and 5), and 0-24 hr after administration on Day 7.)