BOTULINUM TOXIN FOR SPASTICITY IN CEREBRAL PALSY

描述(改编自申请人的描述)：本申请的目的 本研究旨在评价肉毒杆菌毒素注射(BTX)治疗慢性阻塞性肺疾病的疗效。 减少痉挛，改善功能和活动能力的儿童 痉挛双瘫，脑性瘫痪的一种常见形式。这项研究提出 评估五个科学领域的治疗效果 与残疾相关：病理生理学、损伤、功能受限、 残疾和社会限制。研究设计是前瞻性的， 随机双掩蔽临床试验，评估期为6个月。 40名4-12岁的儿童将被招募并随机分成两组 组。A组将接受BTX。B组将接受安慰剂 注射。结果将在基线的掩蔽评估中进行评估，3 周、8周、12周和24周。B组受试者将拥有 24周后有机会接受BTX治疗。 提出了结果变量以提供以下五个不同方面的信息 域名。病理生理学的变化将由定量的 肌电运动学测量。减值的变化将是 表示为整个关节扭矩的机电测量 踝关节使用痉挛测量系统。功能上的变化 限制将表现为粗大运动功能的变化，如 通过粗大运动功能测量，体检参数， 使用能量成本指数的能量消耗，以及运动学步态分析。 残疾方面的变化将通过对年龄的连续评估来体现 使用加拿大职业绩效的适当任务绩效 测量。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的扩展。 样本量是根据演示所需的功率计算得出的 粗大运动功能测量和痉挛测量的变化 系统。每个患者需要进行大量的评估 有必要将科目注册分布在前四个班级和 一年半的研究。

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.