Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 5
- 主要终点
- Mean change of muscle metabolism
研究概览
简要总结
The purpose of the study is to show that the intake of L-arginine and metformin improves muscle function and delays disease progression in patients with Duchenne's muscular dystrophy.
详细描述
This is an investigator-initiated, open-label, single-center, proof-of-concept-study. The study medication consists of L-arginine and metformin. The duration of the study is 16 weeks and comprehends one screening and four study visits.
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
- 7 Years 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Molecular diagnosis of DMD
- •Patients 7 - 10 years of age at time of screening
排除标准
- •Previous (3 months or less) or concomitant participation in another therapeutic trial
- •Use of L-arginine, L-citrulline or metformin within the last 3 months
- •Known individual hypersensitivity to L-citrulline or metformin
- •Other chronic disease or clinical relevant limitation of renal, liver, heart function according to discretion of the investigator
研究组 & 干预措施
L-arginine and metformin
7.5 g L-arginine p.o. and 500 mg metformin p.o. per day (3x 2.5 g, respectively 3x 250 mg) for 16 weeks
干预措施: Metformin (Drug)
L-arginine and metformin
7.5 g L-arginine p.o. and 500 mg metformin p.o. per day (3x 2.5 g, respectively 3x 250 mg) for 16 weeks
干预措施: L-Arginine (Drug)
结局指标
主要结局
Mean change of muscle metabolism
时间窗: baseline to week 16
mitochondrial protein expression analysis in muscular biopsies
次要结局
- In vivo change of muscle metabolism(baseline to week 16)
研究者
Dirk Fischer
MD
University Hospital, Basel, Switzerland