NCT02830763终止不适用
Clinical Study on the Safety and Efficacy of Medium-chain Fatty Acid Capsules (CNT-02) for Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Neutral Lipid Storage Disease With Myopathy (NLSD-M)
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan14 个研究点 分布在 2 个国家目标入组 2 人开始时间: 2016年9月5日最近更新:
适应症
干预措施
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- 入组人数
- 2
- 试验地点
- 14
- 主要终点
- The maximum walking distance in a 6-minute walk test at 3 months
研究概览
简要总结
This study is planning to evaluate the safety and clinical efficacy of medium-chain fatty acid capsules (food-grade CNT-02) in subjects with primary triglyceride deposit cardiomyovasculopathy (TGCV) and neutral lipid storage disease with myopathy (NLSD-M) associated with adipose triglyceride lipase (ATGL) genetic defects.
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
- 20 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients for whom ATGL gene deletion has been previously confirmed.
- •Patients who can take the investigational product orally.
- •Male and female who are at least 20 years old at the time of consent.
- •Patients who gave written informed consent.
排除标准
- •Patients with diabetic ketoacidosis.
- •Patients with the possibility of diabetic ketoacidosis (patients with poorly controlled diabetes mellitus [HbA1c>8.4%, NGSP])
- •Patients with terminal malignancy.
- •Pregnant or lactating women.
- •Patients who do not consent to using contraception while participating in this study.
- •Patients allergic to MCT oil.
- •Patients participating in other clinical trial.
- •Otherwise, patients determined to be ineligible for this study by the investigator or sub-investigator.
研究组 & 干预措施
Medium-chain Fatty Acid (CNT-02)
Experimental
干预措施: CNT-02 (Dietary Supplement)
结局指标
主要结局
The maximum walking distance in a 6-minute walk test at 3 months
时间窗: 3 months
Measuring maximum walking distance of 6-minute walk test. Calculating variation of those measured values from baseline to 3 months.
The maximum walking distance in a 6-minute walk test at 6 months
时间窗: 6 months
Measuring maximum walking distance of 6-minute walk test . Calculating variation of those measured values from baseline to 6 months.
次要结局
- MRC sum score in manual muscle testing (MMT) at 6 months(6 months)
- The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 6 months(6 months)
- Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography at 6 months(6 months)
- Fractions of lung volume such as % vital capacity, measured by spirometer at 6 months(6 months)
- MRC sum score in manual muscle testing (MMT) at 3 months(3 months)
- Fractions of lung volume such as % vital capacity, measured by spirometer at 3 months(3 months)
- The amount of fat measured by computed tomography (CT) of the skeletal muscle (fat deposition) at 3 months(3 months)
- Parameters of cardiac function, such as the left ventricular ejection fraction by echocardiography at 3 months(3 months)
- Serum free fatty acid levels at 3 months(3 months)
- Serum free fatty acid levels at 6 months(6 months)
研究者
研究点 (14)
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