Department of Paediatrics, Monash University, Clayton, Victoria, Australia
Department of Paediatrics, Monash University, Clayton, Victoria, Australia.
BMJ Open. 2024 Apr 17;14(4):e071266. doi: 10.1136/bmjopen-2022-071266.
Fetal alcohol spectrum disorder (FASD) is a neurodevelopmental disorder caused by alcohol exposure during pregnancy. FASD is associated with neurodevelopmental deviations, and 50%-94% of children with FASD meet the Diagnostic and Statistical Manual of Mental Disorders-fifth edition diagnostic criteria for attention deficit hyperactivity disorder (ADHD). There is a paucity of evidence around medication efficacy for ADHD symptoms in children with FASD. This series of N-of-1 trials aims to provide pilot data on the feasibility of conducting N-of-1 trials in children with FASD and ADHD.
A pilot N-of-1 randomised trial design with 20 cycles of stimulant and placebo (four cycles of 2-week duration) for each child will be conducted (n=20) in Melbourne, Australia.Feasibility and tolerability will be assessed using recruitment and retention rates, protocol adherence, adverse events and parent ratings of side effects. Each child's treatment effect will be determined by analysing teacher ADHD ratings across stimulant and placebo conditions (Wilcoxon rank). N-of-1 data will be aggregated to provide an estimate of the cohort treatment effect as well as individual-level treatment effects. We will assess the sample size and number of cycles required for a future trial. Potential mediating factors will be explored to identify variables that might be associated with treatment response variability.
The study was approved by the Hospital and Health Service Human Research Ethics Committee (HREC/74678/MonH-2021-269029), Monash (protocol V6, 25 June 2023).Individual outcome data will be summarised and provided to participating carers and practitioners to enhance care. Group-level findings will be presented at a local workshop to engage stakeholders. Findings will be presented at national and international conferences and published in peer-reviewed journals. All results will be reported so that they can be used to inform prior information for future trials.
NCT04968522.
胎儿酒精谱系障碍（FASD）是一种由怀孕期间暴露于酒精引起的神经发育障碍。FASD 与神经发育偏差有关，50%-94%的 FASD 儿童符合《精神障碍诊断与统计手册》第五版（DSM-5）注意力缺陷多动障碍（ADHD）的诊断标准。关于 FASD 儿童 ADHD 症状的药物疗效，证据很少。本系列 N-of-1 试验旨在为在 FASD 和 ADHD 儿童中进行 N-of-1 试验的可行性提供初步数据。
将在澳大利亚墨尔本进行一项 N-of-1 随机试验设计的试点研究（n=20），每个儿童有 20 个周期的兴奋剂和安慰剂（每个周期持续 2 周，共 4 个周期）。将通过招募和保留率、方案依从性、不良事件和家长对副作用的评分来评估可行性和耐受性。每个孩子的治疗效果将通过分析教师在兴奋剂和安慰剂条件下的 ADHD 评分来确定（Wilcoxon 等级）。N-of-1 数据将汇总，以提供队列治疗效果的估计以及个体水平的治疗效果。我们将评估未来试验所需的样本量和周期数。将探索潜在的中介因素，以确定与治疗反应变异性相关的变量。
该研究已获得医院和卫生服务人体研究伦理委员会（HREC/74678/MonH-2021-269029）和莫纳什大学（协议 V6，2023 年 6 月 25 日）的批准。个体结果数据将进行总结并提供给参与的照顾者和从业者，以加强护理。组级研究结果将在当地研讨会上展示，以吸引利益相关者。研究结果将在国家和国际会议上展示，并发表在同行评议的期刊上。所有结果都将报告，以便为未来的试验提供参考信息。
NCT04968522。
