"Treatment With L-citrulline in Patients With Post-polio Syndrome - a Randomized Double Blind Placebo Controlled Study"
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Mean change of 6 Minute Walking Distance(6MWD)
研究概览
简要总结
The purpose of the study is to show that the intake of L-citrulline improves muscle function in patients with Post-Polio Syndrome (PPS).
详细描述
This is a single center, randomized, double-blind, placebo controlled study. The study medication consists of L-citrulline, respectively placebo given orally; 5 g L-citrulline or placebo will be given three times daily. The duration of the study is 48 weeks and comprehends one screening and four study visits.
Additionally, in order to investigate whether PPS is associated with an atrophy of spinal cord gray and potentially also white matter as well as motor cortex atrophy, it is planned to offer all trial participants an additional optional spinal cord/brain MRI investigation and a MRI at week 48 and a follow-up one year later together with a clinical and physiotherapy assessment during extension phase. We don't expect Citrulline to affect the spinal cord gray or white and gray matter.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Prior paralytic poliomyelitis with evidence of motor neuron loss
- •A period of partial or complete functional recovery after acute paralytic Poliomyelitis
- •Slowly progressive and persistent new muscle weakness or decreased endurance, with or without generalized fatigue, muscle atrophy, or muscle and joint pain. Symptoms that persist for at least a year
- •Exclusion of other neuromuscular, medical and skeletal abnormalities as causes of symptoms
- •Patients older than 18 years at time of Screening
- •ability to walk 150m in the 6 min. Walking distance (6MWD)
- •females of childbearing potential willing to use contraceptive during the study
排除标准
- •Previous (3 months or less) or concomitant participation in any other therapeutic trial
- •Use of L-citrulline or L-arginine within the last 3 months
- •Known individual hypersensitivity to L-citrulline
- •Known or suspected malignancy
- •Other chronic disease or clinical relevant limitation of renal, liver, heart function according to discretion of investigator
- •Pregnant or breast-feeding women
- •Severe renal failure (calculated glomerular filtration rate (GFR) < 30 ml/min)
研究组 & 干预措施
L-citrulline
15 g L-citrulline p.o. per day (3x 5g) for 24 weeks
干预措施: 15g L-citrulline daily p.o. (Drug)
Placebo
L-citrulline Placebo 3 times daily p.o. for 24 weeks
干预措施: Placebo (Drug)
结局指标
主要结局
Mean change of 6 Minute Walking Distance(6MWD)
时间窗: Baseline to week 48
次要结局
- Change of quantitative muscle MRI (IDEAL-CPMG with T₂ and lipid quantitation and dynamic 31P-MR spectroscopy)(Baseline to week 48)
- Change of serum concentrations for markers of muscle necrosis(Baseline to week 48)
- Mean change of motor function measure (MFM) total score(Baseline to week 48)
- Change of serum concentrations for markers of oxidative stress(Baseline to week 48)
- Change of serum concentrations for markers of nitrosative stress(Baseline to week 48)
- Change of serum concentrations for markers of mitochondrial related genes(Baseline to week 48)
研究者
Dirk Fischer
Prof. Dr. med.
University Hospital, Basel, Switzerland