NCT02035501Unknown2 期
Treatment of TNNT1-Myopathy With L-Tyrosine. A Double-blind, Placebo-controlled Crossover Trial.
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- 1. goal attainment score
研究概览
简要总结
The purpose of the study is to investigate if treatment with L-Tyrosine improves selected outcome measures of TNNT1 myopathy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- — 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients
- •any age with TNNT1-related myopathy
排除标准
- •Patients who are non-cooperative or parents/ legal guardians who are unwilling to sign consent form
研究组 & 干预措施
L-Tyrosine
Active Comparator
干预措施: L-Tyrosine (Drug)
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
1. goal attainment score
时间窗: 3 month
次要结局
未报告次要终点
研究者
Shimon Edverson
MD
Hadassah Medical Organization
研究点 (1)
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