Department of Neurology, Leiden University Medical Center, Leiden, the Netherlands; Duchenne Center Netherlands, the Netherlands.
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, London, UK.
Neuromuscul Disord. 2023 Nov;33(11):897-910. doi: 10.1016/j.nmd.2023.10.003. Epub 2023 Oct 11.
There are multiple avenues for therapeutic development in Duchenne muscular dystrophy (DMD), which are highlighted in the first section of this report for the "10 years of Clinical trials in DMD - What have we learned?" workshop. This report then provides an overview of the presentations made at the workshop grouped into the following core themes: trial outcomes, disease heterogeneity, meaningfulness of outcomes and the utility of real-world data in trials. Finally, we present the consensus that was achieved at the workshop on the learning points from 10 years of clinical trials in DMD, and possible action points from these. This includes further work in expanding the scope and range of trial outcomes and assessing the efficacy of new trial structures for DMD. We also highlight several points which should be addressed during future interactions with regulators, such as clinical meaningfulness and the use of real-world data.
杜氏肌营养不良症（DMD）有多种治疗开发途径，在本报告的第一部分中，针对“DMD 临床试验 10 年——我们学到了什么？”研讨会突出了这些途径。本报告概述了研讨会的演讲，这些演讲分为以下核心主题：试验结果、疾病异质性、结果的意义以及真实世界数据在试验中的实用性。最后，我们展示了研讨会就 DMD 临床试验 10 年的学习要点以及从这些要点中可能采取的行动达成的共识。这包括进一步扩大试验结果的范围和范围，并评估新的 DMD 试验结构的疗效。我们还强调了在与监管机构未来互动中应解决的几个问题，例如临床意义和真实世界数据的使用。
