CHANGES IN NEUROMUSCULAR JUNCTIONS IN ALS

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 在意大利对ALS患者进行了一项小型试点研究。16名参与者接受了300？450 mg/天锂（目标血清水平0.4-0.8 mEq/L）和利鲁唑; 28例受试者仅接受利鲁唑。通过ALSFRS-R测量，锂治疗的参与者表现出较慢的功能下降速率。 这项研究规模很小，参与者对治疗不设盲。 这是一项双盲、随机、安慰剂对照临床试验，评价锂和利鲁唑与安慰剂和利鲁唑相比的安全性和疗效。 大约250名志愿者将从美国和加拿大的多个中心招募，这些中心是东北ALS联盟（NEALS）和加拿大ALS临床试验和研究网络（CALS）的一部分。 主要结局指标是疾病进展，定义为至ALSFRS-R量表下降6分或死亡的时间。将在基线访视时进行ALSFRS-R评分;这将被视为每例受试者的基线评分。ALSFRS-R下降6分或以上将被定义为疾病进展和治疗失败。然后，受试者将在研究期间接受锂治疗（总计长达52周）。研究中心工作人员将对初始分组保持盲态，并对ALSFRS-R总分保持盲态，不知道受试者从安慰剂转换为锂剂的日期。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. A small pilot study was performed in Italy in participants with ALS. Sixteen participants were treated with 300 ¿?? 450mg/day of lithium (target serum level 0.4-0.8 mEq/L) and riluzole; 28 participants received only riluzole. Participants treated with lithium demonstrated a slower rate of functional decline as measured by the ALSFRS-R. The study was small and participants were not blinded to treatment. This is a double blind, randomized, placebo-controlled clinical trial evaluating the safety and efficacy of lithium and riluzole compared to placebo and riluzole. Approximately 250 volunteers will be recruited from multiple centers in the US and Canada that are part of the Northeast ALS Consortium (NEALS) and the Canadian ALS Clinical Trials and Research Network (CALS). The primary outcome measure is disease progression defined as the time to decline of 6 points in the ALSFRS-R scale or death. The ALSFRS-R will be administered at the Baseline Visit; this will be considered the baseline score for each participant. A drop of 6 points or more in the ALSFRS-R will be defined as disease progression and treatment failure. Participants will then be treated with lithium for the period of the study (up to 52 weeks total). Study site staff will remain blinded to initial group assignments and will remain blinded to the total ALSFRS-R scores and be unaware of the date when a participant is switched from placebo to lithium.