Spinal Cord Innovation in Pediatrics to Treat Cerebral Palsy (SCiP Clinical Study)

儿科脊髓创新治疗脑瘫（SCiP 临床研究）

• 批准号: 10760810
• 负责人: Parag Gad
• 金额: $ 92.46万
• 依托单位: SPINEX, INC.
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-09-22 至 2025-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10760810
• 关键词: 21 year old; Address; Adult; Affect; Age; Agreement; Botulinum Toxin Type A; Brain; Breakthrough device; Cardiovascular system; Caring; Cerebral Palsy; Child; Childhood; Classification; Clinical; Clinical Research; Communication; Development; Devices; Diagnosis; Double-Blind Method; Epilepsy; Equilibrium; Esthesia; Etiology; Feedback; Functional disorder; Future; Impaired cognition; Impairment; Individual; International; Intervention; Intramuscular Injections; Life; Maintenance; Measures; Motor; Motor Activity; Movement; Muscle; Muscle function; Musculoskeletal; Nervous System Physiology; Neurogenic Bladder; Neurologic; Operative Surgical Procedures; Outcome; Pain; Pain management; Parents; Pathway interactions; Pediatrics; Perception; Persons; Pharmaceutical Preparations; Physical therapy; Physiological; Pilot Projects; Population Study; Posture; Prevalence; Procedures; Proprioception; Quality of life; Randomized, Controlled Trials; Reporting; Rhizotomy procedure; Risk; Safety; Sample Size; Sensorimotor functions; Severities; Spastic Cerebral Palsy; Spinal; Spinal Cord; Spinal cord injury; Statistical Data Interpretation; Symptoms; Testing; Therapeutic; Time; Update; Vertebral column; aged; clinically significant; design; disability; follow-up; functional electrical stimulation; functional improvement; improved; information processing; innovation; meetings; neural; neural circuit; neural network; neurological rehabilitation; neuromuscular plasticity; neuromuscular system; neuroregulation; novel therapeutics; pilot trial; primary outcome; prospective; recruit; rehabilitation strategy; response; secondary outcome; sensory input; skills; spasticity; spinal nerve posterior root; standard of care; symptom management; therapy design; transcutaneous stimulation; usability

Summary Cerebral Palsy (CP) is the most common motor disability in childhood. Population-based studies from around the world report prevalence estimates ranging from 1.5 to 4 per 1000 individuals. Globally, approximately 17 million people have CP. It is a diagnosis that includes a wide range of symptoms with heterogeneous etiologies of neural and cardiovascular origins. A wide range of functional disorders typically become obvious in CP during early development. For example, the majority of children classified as Gross Motor Functional Classification Scale (GMFCS) Level I are expected to reach their motor potential between 7-9 years and remain stable after that until age 21 where they may decline due to pain, weakness and stiffness. While movements such as stepping and posture are amongst the most recognizable impairments, absence or abnormal sensations, perception, impaired cognition, communication skills, epilepsy, spasticity, poor control of fine movements and marked secondary musculoskeletal problems can emerge with time. Presently, all available interventions are designed to minimize the severity of symptoms rather than correcting the neurological etiology. Two commonly preferred treatments, selective dorsal root rhizotomy and repetitive administration of botulinum toxin A (Botox) directly target the neuromuscular system to counter spasticity. While these two strategies minimize some of the disruptive movements associated with spasticity; the long-term benefits of these interventions are less clear and both cause irreversible changes to the neuromuscular system that may be detrimental to future function. In the present proposal we have merged ideas and concepts derived from both recent clinical observations of CP and decades of studies of spinal cord injury to arrive at a subject-specific rehabilitation strategy driven by activity- based mechanisms during transcutaneous spinal neuromodulation. The objectives of our neuromodulation procedures are to amplify the plasticity of the neuromuscular system and help children gain the ability to perform coordinated movements through activity-dependent plasticity. We hypothesized that transcutaneous spinal neuromodulation during activity-based neurorehabilitation therapy (ABNT) can transform the neural networks in children diagnosed with spastic CP to improve voluntary postural and locomotor activity as captured on the GMFM88 scores. This strategy could provide a specific physiological target of fundamental significance in the control of movement that can be used to improve function and provide new care pathways for children with CP.

概括 脑瘫（CP）是儿童期最常见的运动障碍。来自各地的基于人群的研究 世界报告的患病率估计为每 1000 人 1.5 至 4 人。全球约有 17 百万人有CP。这是一种包括具有异质病因的广泛症状的诊断 神经和心血管起源。多种功能障碍通常在 CP 期间变得明显 早期发展。例如，大多数儿童被归类为粗大运动功能分类 量表 (GMFCS) I 级预计在 7-9 年内达到其运动潜力，并在之后保持稳定 直到 21 岁，他们的能力可能会因疼痛、虚弱和僵硬而衰退。当进行诸如踏步之类的动作时 和姿势是最容易识别的损伤、缺乏或异常的感觉、知觉、 认知、沟通能力受损、癫痫、痉挛、精细动作控制不良和明显 随着时间的推移，可能会出现继发性肌肉骨骼问题。目前，所有可用的干预措施均已设计 尽量减轻症状的严重程度，而不是纠正神经病因。两个普遍首选 治疗、选择性背根根切断术和直接重复注射肉毒杆菌毒素 A (Botox) 针对神经肌肉系统来对抗痉挛。虽然这两种策略最大限度地减少了一些 与痉挛相关的破坏性运动；这些干预措施的长期效益尚不明确，而且 两者都会导致神经肌肉系统发生不可逆的变化，可能不利于未来的功能。在 在目前的提案中，我们融合了来自最近 CP 和 CP 临床观察的想法和概念。 对脊髓损伤进行了数十年的研究，以得出由活动驱动的针对特定主题的康复策略 经皮脊髓神经调节过程中的基础机制。我们神经调节的目标 程序旨在增强神经肌肉系统的可塑性并帮助儿童获得执行能力 通过活动依赖性可塑性协调运动。我们假设经皮脊柱 基于活动的神经康复治疗 (ABNT) 期间的神经调节可以改变神经网络 诊断患有痉挛性脑瘫的儿童，以改善自主姿势和运动活动（如记录在图上） GMFM88 分数。该策略可以提供具有根本意义的特定生理目标 运动控制可用于改善脑瘫儿童的功能并提供新的护理途径。