Nikles J, O'Sullivan J D, Mitchell G K, Smith S S, McGree J M, Senior H, Dissanyaka N, Ritchie A
UQ Centre for Clinical Research, The University of Queensland, Brisbane, Australia.
Royal Brisbane & Women's Hospital (RBWH), Neurology Department, School of Medicine, St Andrew's, Wesley and RBWH Hospitals, The University of Queensland, Brisbane, Australia.
Contemp Clin Trials Commun. 2019 Jul 8;15:100397. doi: 10.1016/j.conctc.2019.100397. eCollection 2019 Sep.
40% of Parkinson's Disease (PD) sufferers experience insomnia, impacting health and quality of life for patients and family members, especially carers. There is little evidence that current treatments are effective.
To determine the effectiveness of melatonin in reducing insomnia in 44 individuals with PD using N-of-1 trials. To aggregate group data to arrive at population estimates of effectiveness (measured by improvements in PDSS-2) and safety (measured by adverse events) of melatonin in improving insomnia in PD. To assess the feasibility of offering N-of-1 trials for insomnia in PD.
Participants will receive either immediate-release melatonin or placebo in random order in 3 paired two-week treatment periods (12 weeks total). Based on their response in a two-week run-in period on 3 mg daily, they will trial either 3 mg or 6 mg. Patients will keep daily sleep diaries and wear a MotionWatch throughout. After the trial patients will discuss their individual report with their doctor, which provides direct feedback about effectiveness and safety of melatonin for them.
We will analyse N-of-1 tests 1) individually: effects of melatonin on PDSS-2 and safety will be reported; and 2) aggregated across individual N-of-1 studies, combined using a Bayesian multilevel random effects model, which will account for repeated measures on individuals over time, and will return posterior estimates of overall treatment effect, and effect in each individual.
ACTRN12617001103358.
40%的帕金森病（PD）患者存在失眠问题，这对患者及其家庭成员，尤其是护理人员的健康和生活质量产生了影响。几乎没有证据表明目前的治疗方法有效。
通过单病例试验确定褪黑素对44名帕金森病患者失眠症状的疗效。汇总分组数据，得出褪黑素改善帕金森病患者失眠的有效性（以帕金森病睡眠量表第二版（PDSS-2）的改善情况衡量）和安全性（以不良事件衡量）的总体估计值。评估为帕金森病患者失眠提供单病例试验的可行性。
参与者将在3个为期两周的配对治疗期（共12周）内，以随机顺序接受速释褪黑素或安慰剂治疗。根据他们在为期两周的导入期内每天服用3毫克的反应，他们将试验3毫克或6毫克的剂量。患者将每天记录睡眠日记，并全程佩戴运动监测仪。试验结束后，患者将与医生讨论他们的个人报告，该报告提供了褪黑素对他们有效性和安全性的直接反馈。
我们将对单病例试验进行分析：1） 单独分析：报告褪黑素对PDSS-2的影响和安全性；2） 汇总各个单病例研究的数据，使用贝叶斯多层随机效应模型进行合并，该模型将考虑个体随时间的重复测量，并返回总体治疗效果和每个个体效果的后验估计值。
ACTRN12617001103358。
