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：
8723915
负责人：
Alvaro Pascual-Leone
金额：
$ 21.53万
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/8723915
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）：脊椎发子共济失调（SCA）是指导致步态和平衡渐进问题的遗传疾病家族，以及其他令人衰弱的症状。即使已知负责许多SCA亚型的基因，尽管我们知道它们都与小脑和大脑其他特定部位的损害有关，但SCA无法治愈，我们仍然缺乏有效的症状治疗。我们提出了一种使用非侵入性经颅磁刺激（TMS）来改善SCA患者平衡，步态和姿势的新方法。我们将招募20例遗传确认的SCA患者。一半将随机分配给实际干预措施，将一半分为假（即控制）干预措施。 TMS干预措施将包括在四个星期内的20个刺激课程。在基线和随访时，所有患者将接受全面的评估，包括几个SCA评级量表，以及精致的平衡测试（即步行，站立和肌肉协调）。患者还将完成一系列神经生理测试，以评估小脑及其在干预之前和之后的连接功能。这将有助于我们测试干预措施的临床实用性，并获得有关SCA残疾基础的新知识。在试点研究中，我们已经表明，将TMS应用于小脑时是安全的，并且可以改善患者的平衡。现在，我们将进行更系统的，更大，精心控制的临床试验证明，我们预计接受真正的RTMS的患者将显示出更好的平衡，更少的跌倒和改善的移动性，而那些接受假刺激的患者不会显示出任何好处。如果我们的预测是正确的，这项研究将为新的治疗方法提供基于证据的支持，以改善SCA患者的生活，拟议的定量评估将检查可能的客观终点，以实现未来，更大的多站点临床试验。