Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, London, UK.
National Hospital for Neurology and Neurosurgery, London, UK.
BMJ Open. 2021 May 28;11(5):e047993. doi: 10.1136/bmjopen-2020-047993.
Parkinson's disease (PD) is a common neurodegenerative disorder with substantial morbidity. No disease-modifying treatments currently exist. The glucagon like peptide-1 receptor agonist exenatide has been associated in single-centre studies with reduced motor deterioration over 1 year. The aim of this multicentre UK trial is to confirm whether these previous positive results are maintained in a larger number of participants over 2 years and if effects accumulate with prolonged drug exposure.
This is a phase 3, multicentre, double-blind, randomised, placebo-controlled trial of exenatide at a dose of 2 mg weekly in 200 participants with mild to moderate PD. Treatment duration is 96 weeks. Randomisation is 1:1, drug to placebo. Assessments are performed at baseline, week 12, 24, 36, 48, 60, 72, 84 and 96 weeks.The primary outcome is the comparison of Movement Disorders Society Unified Parkinson's Disease Rating Scale part 3 motor subscore in the practically defined OFF medication state at 96 weeks between participants according to treatment allocation. Secondary outcomes will compare the change between groups among other motor, non-motor and cognitive scores. The primary outcome will be reported using descriptive statistics and comparisons between treatment groups using a mixed model, adjusting for baseline scores. Secondary outcomes will be summarised between treatment groups using summary statistics and appropriate statistical tests to assess for significant differences.
This trial has been approved by the South Central-Berkshire Research Ethics Committee and the Health Research Authority. Results will be disseminated in peer-reviewed journals, presented at scientific meetings and to patients in lay-summary format.
NCT04232969, ISRCTN14552789.
帕金森病（PD）是一种常见的神经退行性疾病，发病率很高。目前尚无改变疾病进程的治疗方法。胰高血糖素样肽-1 受体激动剂 exenatide 在单中心研究中与 1 年内运动恶化减少相关。本项多中心英国试验的目的是确认在更多参与者中，经过 2 年的治疗，这些之前的阳性结果是否能够维持，以及药物暴露时间延长是否会产生累积效应。
这是一项在 200 名轻度至中度 PD 患者中进行的为期 96 周、2 毫克/周、为期 3 期、多中心、双盲、随机、安慰剂对照的 exenatide 试验。随机分组为 1:1，药物组与安慰剂组。评估在基线、第 12、24、36、48、60、72、84 和 96 周进行。主要结局是在 96 周时，根据治疗分配，比较实际定义的 OFF 药物状态下运动障碍协会统一帕金森病评定量表第 3 部分运动子评分。次要结局将比较各组之间其他运动、非运动和认知评分的变化。主要结局将使用描述性统计和混合模型进行报告，根据基线评分进行调整。次要结局将使用组间汇总统计和适当的统计检验进行总结，以评估是否存在显著差异。
该试验已获得南英格兰-伯克郡研究伦理委员会和英国健康研究局的批准。结果将在同行评议的期刊上发表，在科学会议上报告，并以通俗易懂的格式向患者报告。
NCT04232969，ISRCTN84744167。
