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Mexiletine versus lamotrigine in non-dystrophic myotonias: a randomised, double-blind, head-to-head, crossover, non-inferiority, phase 3 trial
The Lancet Neurology ( IF 46.5 ) Pub Date : 2024-09-18 , DOI: 10.1016/s1474-4422(24)00320-x
Vinojini Vivekanandam PhD, Iwona Skorupinska MSc, Dipa L Jayaseelan PhD, Emma Matthews PhD, Prof Richard J Barohn MD, Prof Michael P McDermott PhD, Prof Michael G Hanna MD

Non-dystrophic myotonias are skeletal muscle channelopathies caused by ion channel dysfunction. Symptom onset is frequently in the first decade of life, causing disability in a young cohort. Although there is no cure, symptomatic treatments exist. Previous trials provide evidence of the efficacy of mexiletine. More recently, lamotrigine has been shown to be effective. Both treatments have different profiles, including pharmacokinetics and adverse events. This trial aimed to investigate whether lamotrigine is non-inferior to mexiletine to directly inform clinical practice.

中文翻译:


美西律与拉莫三嗪治疗非肌营养不良性肌强直:一项随机、双盲、头对头、交叉、非劣效性、3 期试验



非营养不良性肌强直是由离子通道功能障碍引起的骨骼肌通道病。症状通常在生命的头十年出现,导致年轻群体残疾。虽然无法治愈,但存在对症治疗。以前的试验提供了美西律疗效的证据。最近,拉莫三嗪已被证明有效。两种治疗方法具有不同的特点,包括药代动力学和不良事件。该试验旨在调查拉莫三嗪是否不劣于美西律,以直接为临床实践提供信息。
更新日期:2024-09-18
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