Transcranial Stimulation in Spino-Cerebellar Ataxia

脊髓小脑共济失调的经颅刺激

基本信息

批准号：
8621719
负责人：
Alvaro Pascual-Leone
金额：
$ 25.4万
依托单位：
BETH ISRAEL DEACONESS MEDICAL CENTER
依托单位国家：
美国
项目类别：
财政年份：
2013
资助国家：
美国
起止时间：
2013-09-01 至 2015-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8621719
关键词：
Biological Neural Networks Biomechanics Brain Brain Part Brain region Cerebellar Ataxia Cerebellar degeneration Cerebellum Clinical Clinical Trials Complex Double-Blind Method Electromagnetics Equilibrium Functional disorder Future Gait Genes Genetic Guidelines Hereditary Disease Human Individual Intervention Investigation Knowledge Lead Limb Ataxia Magnetic Resonance Imaging Magnetism Mental Depression Mental disorders Metric Morbidity - disease rate Movement Multi-Institutional Clinical Trial Muscle Musculoskeletal Equilibrium Neurodegenerative Disorders Neuronavigation Outcome Paresis Parkinson Disease Parkinson&apos s Dementia Patients Peripheral Nerves Pharmaceutical Preparations Physiology Pilot Projects Plant Roots Posture Quality of life Quantitative Evaluations Randomized Randomized Controlled Trials Recruitment Activity Resistance Series Spinocerebellar Ataxias Stroke Structure Symptoms System Testing Transcranial magnetic stimulation Traumatic Brain Injury Treatment Efficacy Walking balance testing base disability evidence base experience falls family genetics follow-up functional decline functional disability functional gain functional improvement functional outcomes improved improved mobility motor control nervous system disorder neurophysiology novel strategies novel therapeutic intervention public health relevance repetitive transcranial magnetic stimulation spinal tract

项目摘要

DESCRIPTION (provided by applicant): Spinocerebellar ataxia (SCA) refers to a family of genetic diseases that cause progressive problems with gait and balance, as well as other debilitating symptoms. Even though the genes responsible for many of the SCA subtypes are known, and even though we know that they all are associated with damage of the cerebellum and other specific parts of the brain, there is no cure for SCA and we still lack an effective symptomatic treatment. We propose a novel approach using noninvasive transcranial magnetic stimulation (TMS) to improve balance, gait, and posture in patients with SCA. We will recruit 20 patients with genetically-confirmed SCA. Half will be randomly assigned to a real intervention, and half to a sham (i.e., control) intervention. The TMS intervention will consist of 20 stimulatio sessions over a four week period. At baseline and at follow-up, all patients will undergo comprehensive assessments including several SCA rating scales, along with sophisticated tests of balance (i.e., walking, standing and muscle coordination). Patients will also complete a series of neurophysiologic tests to evaluate the function of the cerebellum and its connections before and after the intervention. This will help us test the clinical utility of the intervention and als gain new knowledge about the basis for the disability in SCA. In pilot studies we have shown already that TMS is safe when applied to the cerebellum and that it can improve balance in patients. We now will conduct a more systematic, larger, carefully controlled proof- of-principle clinical trial We anticipate that patients receiving real rTMS will show better balance, fewer falls, and improved mobility while those undergoing sham stimulation will show no benefits. If our prediction is correct, this study will provide evidence-based support for a new treatment to improve the lives of patients with SCA, and the proposed quantitative evaluations will examine possible objective end-points for a future, larger multi-site clinical trial.

描述（由申请人提供）：脊髓小脑性共济失调（SCA）是指导致步态和平衡进行性问题以及其他衰弱症状的一系列遗传疾病。尽管导致许多 SCA 亚型的基因是已知的，并且尽管我们知道它们都与小脑和大脑其他特定部位的损伤有关，但 SCA 无法治愈，而且我们仍然缺乏有效的症状治疗方法。治疗。我们提出了一种使用无创经颅磁刺激 (TMS) 来改善 SCA 患者的平衡、步态和姿势的新方法。我们将招募 20 名基因确诊的 SCA 患者。一半将被随机分配到真实干预组，一半被分配到假干预（即对照）干预组。 TMS 干预将包括为期 4 周的 20 次刺激课程。在基线和随访时，所有患者都将接受全面评估，包括几种 SCA 评级量表，以及复杂的平衡测试（即行走、站立和肌肉协调）。患者还将完成一系列神经生理学测试，以评估干预前后小脑的功能及其连接。这将帮助我们测试干预措施的临床效用，并获得有关 SCA 残疾基础的新知识。在试点研究中，我们已经证明，TMS 应用于小脑时是安全的，并且可以改善患者的平衡能力。我们现在将进行一项更系统、更大规模、仔细控制的原理验证临床试验。我们预计接受真正 rTMS 的患者将表现出更好的平衡性、更少的跌倒和改善的活动能力，而接受假刺激的患者将不会表现出任何益处。如果我们的预测正确，这项研究将为改善 SCA 患者生活的新疗法提供循证支持，并且拟议的定量评估将为未来更大规模的多中心临床试验检查可能的客观终点。