"A Double Blind Randomised Placebo Controlled Efficacy and Safety Study of L-citrulline and Metformin in Ambulant Children Aged Between 7 and 10 Years With Duchenne's Muscular Dystrophy"
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 47
- 试验地点
- 1
- 主要终点
- Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers)
研究概览
简要总结
The purpose of the study is to show that the intake of L-citrulline and metformin improves muscle function and delay of progression in patients with Duchenne's muscular dystrophy.
详细描述
This is a single center, randomized, double-blind, placebo controlled study. The study medication consists of L-citrulline and metformin, respectively placebo given orally; 2.5 g L-citrulline or placebo will be given 3 times daily, metformin containing 250 mg or placebo will be administered 3 times daily. The duration of the study is 26 weeks and comprehends one screening and three study visits.
Amendment 1: Amended eligibility criteria
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
- 78 Months 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Molecular diagnosis of DMD
- •Patients 6.5 - 10 years of age at time of screening
- •Ability to walk 150 m in the 6 min walking distance (6MWT)
- •D1 subdomain of the MFM scale >40%
- •stable treatment with steroids for >6 months or steroid naïve patients
排除标准
- •Previous (3 months or less) or concomitant participation in another therapeutic trial
- •Use of L-citrulline, L-arginine or metformin within the last 3 months
- •Known individual hypersensitivity to L-citrulline or metformin
- •known or suspected malignancy
- •Other chronic disease or clinical relevant limitation of renal, liver, heart function according to discretion of investigator
- •start of cortisone treatment or change in dosage <6 months prior to screening
研究组 & 干预措施
750 mg metformin and 7.5 g L-citrulline daily p.o.
7.5 g L-citrulline p.o. and 750 mg metformin daily p.o. (3x 2.5 g, respectively 3x 250 mg) for 26 weeks
干预措施: 750 mg metformin and 7.5 g L-citrulline daily p.o. (Drug)
Placebo
metformin placebo and L-citrulline placebo 3 times daily p.o. for 26 weeks
干预措施: Placebo (Drug)
结局指标
主要结局
Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers)
时间窗: baseline to week 26
次要结局
- Mean change of MFM total score, the D2, and D3 MFM subscores(baseline to week 26)
- Mean change of six minute walking distance (6MWD)(baseline to week 26)
- Change of quantitative muscle MRI (Magnetic Resonance Imaging) including muscle fat content (MFC) and T2 times of thigh muscles(baseline to week 26)
- Change in the plasma/urine concentration for markers of muscle necrosis, oxidative stress, nitrosative stress, and change of microRNA (miRNA)(baseline to week 26)
- Mean change of quantitative muscle force (QMT) of knee extension and elbow flexion using hand held dynamometry (HHD)(baseline to week 26)