BOTULINUM TOXIN FOR SPASTICITY IN CEREBRAL PALSY

描述（改编自申请人的描述）：本申请的目的 研究是为了评估肉毒杆菌毒素注射（BTX）在 减少痉挛，改善功能和活动能力， 痉挛性双瘫，一种常见的脑瘫（CP）。 本研究提出 在五个科学领域评估治疗效果 与残疾相关：病理生理学、损伤、功能限制， 残疾和社会限制。 研究设计是前瞻性的， 随机、双盲临床试验，评价期为6个月。 将招募40名4 - 12岁的儿童，并随机分为两组 组 A组将获得BTX。 B组将接受安慰剂 注射 将在基线时进行设盲评价，3 周、8周、12周和24周。 B组受试者将接受 24周后给予BTX治疗。 结果变量的建议，以提供信息，在五个不同的 域. 病理生理学的变化将通过定量 肌电运动学测量。 减值变动将 由跨关节扭矩的机电测量表示， 踝关节使用痉挛测量系统。 功能变化 限制将由粗大运动功能的变化表示为 通过粗大运动功能测量、体格检查参数， 使用能量成本指数的能量消耗和运动步态分析。 残疾的变化将通过年龄的系列评估来表示 使用加拿大职业绩效的适当任务绩效 measure. 社会限制的任何变化都将通过使用 在社区和学校活动领域实现目标。 样本量基于证明所需的把握度计算 粗大运动功能测量和痉挛测量的变化 系统 每名患者需要进行大量评估 必须将受试者入组分布在前四名， 半年的研究。

DESCRIPTION (Adapted from the applicant's description): The purpose of this study is to evaluate the effectiveness of botulinum toxin injection (BTX) in reducing spasticity and improving function and mobility in children with spastic diplegia, a common form of cerebral palsy (CP). This study proposes to evaluate the effects of the treatment across the five domains of science relative to disability: pathophysiology, impairment, functional limitation, disability, and societal limitation. The study design is a prospective, randomized, double-masked clinical trial with a 6 month evaluation period. Forty children, ages 4-12 years, will be recruited and randomized into two groups. Group A will receive BTX. Group B will receive a placebo injection. Outcomes will be assessed in a masked evaluation at baseline, 3 weeks, 8 weeks, 12 weeks, and 24 weeks. Group B subjects will have the opportunity for BTX treatment after 24 weeks. Outcome variables are proposed to provide information in the five different domains. Change in pathophysiology will be represented by quantitative electromyographic kinesiology measurements. Change in impairment will be represented by electromechanical measurement of joint torque across the ankle joint using the Spasticity Measurement System. Change in functional limitation will be represented by changes in gross motor function as measured by the Gross Motor Function Measure, physical exam parameters, energy expenditure using the Energy Cost Index, and kinematic gait analysis. Changes in disability will be represented by serial assessments of age appropriate task performance using the Canadian Occupational Performance Measure. Any change in societal limitation will be measured by the use of Goal Attainment Scaling in the areas of community and school activities. Sample size has been based on the power calculation necessary to demonstrate a change in the Gross Motor Function Measure and the Spasticity Measurement System. The large number of assessments required for each patient necessitate that subject enrollment be distributed over the first four and one half years of the study.