Pilot Study to Assess the Efficacy of L-Citrulline and Metformin in Adults With Becker's Muscular Dystrophy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks
研究概览
简要总结
The purpose of the study is to compare the effects of L-citrulline and metformin and their combination therapy on muscle function and force in patients with Becker muscular dystrophy (BMD).
详细描述
This is a single center, not-randomized open study. The study medication consists of L-citrulline and metformin administered orally; 5g citrulline and 500mg metformin tablets will be given 3 times daily.
Half of the patients will be treated with metformin during the first 6 weeks of the study. The other patients will receive initially L-citrulline for 6 weeks, before all patients will be treated with the combination therapy (metformin and L-citrulline) for another 6 week period.
The study lasts 12 weeks and includes the screening visit and 3 further visits at baseline, week 6 and week 12.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •18 years or older
- •Molecular or immunohistochemical diagnosis of BMD
- •ambulant at the time point of screening
排除标准
- •Participation in another therapeutic BMD study within the last 3 months
- •Use of L-Arginine, L-Citrulline or Metformin within the last 3 months
- •Other chronic disease or relevant limitation of renal, liver, heart function according to discretion of investigator
- •known hypersensitivity to L-citrulline or metformin
研究组 & 干预措施
Metformin and Metformin & L-Citrulline
1500 mg/d metformin for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks
干预措施: Metformin and Metformin & L-Citrulline (Drug)
L-Citrulline and Metformin & L-Citrulline
15 g/d L-citrulline for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks
干预措施: L-Citrulline and Metformin & L-Citrulline (Drug)
结局指标
主要结局
Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks
时间窗: week 6 and week 12
次要结局
- Change of laboratory parameters (oxidative and nitrosative stress)(week 6 and week 12)
- Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry)(week 6 and week 12)
- MFM total score and six minute walking distance (6MWD)(week 6 and week 12)
- Change of muscle fat content (MFC) (assessed by MRI)(week 6 and week 12)