Centre for Clinical Brain Sciences, University of Edinburgh Division of Medical and Radiological Sciences, Edinburgh, UK.
Euan MacDonald Centre for Motor Neuron Disease Research, University of Edinburgh Division of Medical and Radiological Sciences, Edinburgh, UK.
BMJ Open. 2021 Mar 23;11(3):e044996. doi: 10.1136/bmjopen-2020-044996.
Motor neuron disease (MND) is a rapidly progressive and fatal neurodegenerative disorder with limited treatment options. The Motor Neuron Disease Systematic Multi-Arm Randomised Adaptive Trial (MND-SMART) is a multisite UK trial seeking to address the paucity in effective disease-modifying drugs for people with MND (pwMND). Historically, neurological trials have been plagued by suboptimal recruitment and high rates of attrition. Failure to recruit and/or retain participants can cause insufficiently representative samples, terminated trials or invalid conclusions. This study investigates patient-specific factors affecting recruitment and retention of pwMND to MND-SMART. Improved understanding of these factors may improve trial protocol design, optimise recruitment and retention.
PwMND on the Scottish MND Register, Clinical Audit Research and Evaluation of MND (CARE-MND), will be invited to participate in a prospective observational cohort study that investigates factors affecting trial participation and attrition. We hypothesise that patient-specific factors will significantly affect trial recruitment and retention. Participants will complete the Hospital Anxiety and Depression Scale, 9-Item Patient Health Questionnaire and State-Trait Anxiety Inventory-Form Y to evaluate neuropsychiatric symptoms, the ALS-Specific Quality of Life Questionnaire-Brief Form and Centre for Disease Control and Prevention-Health-Related Quality of Life for quality of life and a novel study-specific questionnaire on Attitudes towards Clinical Trial Participation (ACT-Q). Clinical data on phenotype, cognition (Edinburgh Cognitive and Behavioural ALS Screen) and physical functioning (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) will also be collated. Caregivers will complete the Brief Dimensional Apathy Scale. After 12 months, a data request to MND-SMART will evaluate recruitment and retention. Descriptive statistics will summarise and compare assessments and participants reaching impairment thresholds. Variable groupings: attitudes, quality of life, cognition, behaviour, physical functioning, neuropsychiatric and phenotype. Univariate and multivariable logistic regression will explore association with participation/withdrawal in MND-SMART; presented as ORs and 95% CIs.
Ethical approval was provided by the West of Scotland Research Ethics Committee 3 (20/WS/0067) on 12 May 2020. The results of this study will be published in a peer-reviewed journal, presented at academic conferences and disseminated to participants and the public.
运动神经元病（MND）是一种快速进展且致命的神经退行性疾病，治疗选择有限。运动神经元疾病系统多臂随机适应性试验（MND-SMART）是一项多地点英国试验，旨在解决运动神经元病患者（pwMND）中有效疾病修饰药物的缺乏。从历史上看，神经学试验一直受到招募效果不佳和高退出率的困扰。未能招募和/或保留参与者可能导致代表性不足的样本、终止试验或无效结论。本研究调查了影响 MND-SMART 招募和保留 pwMND 的患者特定因素。更好地了解这些因素可能会改进试验方案设计，优化招募和保留。
苏格兰 MND 登记处、临床审计研究和 MND 评估（CARE-MND）中的 pwMND 将被邀请参加一项前瞻性观察队列研究，该研究调查影响试验参与和退出的因素。我们假设患者特定因素将显著影响试验招募和保留。参与者将完成医院焦虑和抑郁量表、9 项患者健康问卷和状态特质焦虑量表-Y 以评估神经精神症状、肌萎缩侧索硬化症特定生活质量问卷-简短形式和疾病控制和预防中心-健康相关生活质量，以及一项关于对临床试验参与的态度的新的研究特定问卷（ACT-Q）。还将收集表型、认知（爱丁堡认知和行为性肌萎缩侧索硬化症筛查）和身体功能（肌萎缩侧索硬化症功能评定量表修订版）的临床数据。护理人员将完成简短维度淡漠量表。12 个月后，将向 MND-SMART 提出数据请求，以评估招募和保留情况。描述性统计将总结和比较评估和达到损伤阈值的参与者。变量分组：态度、生活质量、认知、行为、身体功能、神经精神和表型。单变量和多变量逻辑回归将探索与 MND-SMART 参与/退出的关联；以 OR 和 95%CI 呈现。
伦理批准由西苏格兰研究伦理委员会 3 号（20/WS/0067）于 2020 年 5 月 12 日提供。本研究结果将发表在同行评议期刊上，在学术会议上展示，并传播给参与者和公众。
