Participants of the three previous cohorts and a new cohort will be subjected to 6 months bumetanide treatment using multiple baseline Single Case Experimental Designs. The primary outcome is the change, relative to baseline, in a set of patient reported outcome measures focused on direct and indirect effects of sensory processing difficulties. Secondary outcome measures include the conventional questionnaires \'social responsiveness scale\', \'repetitive behavior scale\', \'sensory profile\' and \'aberrant behavior scale\'. Resting-state EEG measurements will be performed at several time-points including at Tmax after the first administration. Assessment of cognitive endpoints will be conducted using the novel Emma Tool box, an in-house designed battery of computerized tests to measure neurocognitive functions in children.
This study aims to replicate previously shown effects of bumetanide in NDD subpopulations, validate a recently proposed treatment prediction effect methodology and refine endpoint measurements.
EudraCT: 2020-002196-35, registered 16 November 2020, https://www.clinicaltrialsregister.eu/ctr-search/trial/2020-002196-35/NL.
前三个队列和新队列的参与者将使用多个基线单例实验设计进行6个月的布美他尼治疗。主要结果是变化,相对于基线,在一组患者报告的结果测量集中在感觉处理困难的直接和间接影响。次要结果测量包括常规问卷“社会反应量表”,“重复行为量表”,“感官配置文件”和“异常行为量表”。静息状态EEG测量将在几个时间点进行,包括在第一次给药后的Tmax。认知终点的评估将使用新的艾玛工具箱进行,内部设计的一系列计算机化测试,用于测量儿童的神经认知功能。
本研究旨在复制先前显示的布美他尼在NDD亚群中的作用,验证最近提出的治疗预测效果方法,并完善终点测量。
EudraCT:2020-002196-35,注册于2020年11月16日,https://www。临床试验登记。欧盟/ctr-search/trial/2020-002196-35/NL。