Schmidt Simone, Gocheva Vanya, Zumbrunn Thomas, Rubino-Nacht Daniela, Bonati Ulrike, Fischer Dirk, Hafner Patricia
Division of Neuropediatrics, University of Basel Children's Hospital, Spitalstrasse 33, Postfach 4031, Basel, Switzerland.
Division of Neurology, University Hospital Basel, Basel, Switzerland.
Trials. 2017 Mar 9;18(1):116. doi: 10.1186/s13063-017-1829-3.
Post-polio syndrome (PPS) is a condition that affects polio survivors years after recovery from an initial acute infection by the Poliomyelitis virus. Most often, patients who suffered from polio start to experience gradual new weakening in muscles, a gradual decrease in the size of muscles (muscle atrophy) and fatigue years after the acute illness. L-citrulline is known to change muscular metabolism synthesis by raising nitric oxide (NO) levels and increasing protein synthesis. This investigator-initiated, randomised, placebo-controlled, double-blind, trial aims to demonstrate that L-citrulline positively influences muscle function and increases muscular energy production in patients with PPS.
METHODS/DESIGN: Thirty ambulant PPS patients will be recruited in Switzerland. Patients will be randomly allocated to one of the two arms of the study (placebo:verum 1:1). After a 24-week run-in phase to observe natural disease history and progression, participants will be treated either with L-citrulline or placebo for 24 weeks. The primary endpoint is change in the 6-min Walking Distance Test. Secondary endpoints will include motor function measure, quantitative muscle force, quantitative muscle magnetic resonance imaging and magnetic resonance spectroscopy and serum biomarker laboratory analysis DISCUSSION: The aim of this phase IIa trial is to determine if treatment with L-citrulline shows a positive effect on clinical function and paraclinical biomarkers in PPS. If treatment with L-citrulline shows positive effects, this might represent a cost-efficient symptomatic therapy for PPS patients.
ClinicalTrial.gov, ID: NCT02801071 . Registered on 6 June 2016.
小儿麻痹后遗症（PPS）是一种在脊髓灰质炎病毒初次急性感染康复数年之后影响脊髓灰质炎幸存者的病症。大多数情况下，患过小儿麻痹症的患者在急性疾病数年之后开始逐渐出现肌肉新的无力、肌肉尺寸逐渐减小（肌肉萎缩）以及疲劳症状。已知L-瓜氨酸可通过提高一氧化氮（NO）水平和增加蛋白质合成来改变肌肉代谢合成。这项由研究者发起的随机、安慰剂对照、双盲试验旨在证明L-瓜氨酸对PPS患者的肌肉功能有积极影响，并增加肌肉能量产生。
方法/设计：将在瑞士招募30名能行走的PPS患者。患者将被随机分配到研究的两个组之一（安慰剂：真药 1:1）。在为期24周的导入期以观察自然疾病史和进展之后，参与者将接受L-瓜氨酸或安慰剂治疗24周。主要终点是6分钟步行距离测试的变化。次要终点将包括运动功能测量、定量肌力、定量肌肉磁共振成像和磁共振波谱以及血清生物标志物实验室分析。讨论：这项IIa期试验的目的是确定L-瓜氨酸治疗是否对PPS的临床功能和临床旁生物标志物有积极影响。如果L-瓜氨酸治疗显示出积极效果，这可能代表一种对PPS患者具有成本效益的对症治疗方法。
ClinicalTrial.gov，标识符：NCT02801071。于2016年6月6日注册。
