Koch Marcus W, Camara-Lemarroy Carlos, Strijbis Eva, Mostert Jop, Leavitt Victoria M, Repovic Pavle, Bowen James D, Comtois Jacynthe, Uitdehaag Bernard, Cutter Gary
Department of Clinical Neurosciences, University of Calgary, Calgary, AB, Canada.
Department of Neurology, MS Center Amsterdam, Amsterdam University Medical Center, Amsterdam, The Netherlands.
Mult Scler. 2024 Dec;30(5_suppl):41-47. doi: 10.1177/13524585241274620. Epub 2024 Sep 8.
While relapsing-remitting multiple sclerosis (MS) has many therapeutic options, progressive forms of MS remain largely untreatable. Phase 2 clinical trials are our main tool to advance new treatments for progressive MS. Given the complexities of progressive MS, it will likely require many phase 2 trials to improve its treatment. To conduct informative and efficient phase 2 trials, it is important that such trials are designed in a way that they can identify a successful treatment as quickly and with as few participants as possible. In this topical review, we discuss cohort selection, outcome selection, cohort enrichment, and dosing selection as strategies to optimize the efficiency of phase 2 clinical trials in progressive MS.
虽然复发缓解型多发性硬化症（MS）有多种治疗选择，但进展型MS在很大程度上仍无法治疗。2期临床试验是推进进展型MS新疗法的主要工具。鉴于进展型MS的复杂性，可能需要进行许多2期试验来改善其治疗。为了开展信息丰富且高效的2期试验，重要的是这些试验的设计方式要能够以尽可能少的参与者尽快确定成功的治疗方法。在本专题综述中，我们讨论了队列选择、结局选择、队列富集和剂量选择，将其作为优化进展型MS 2期临床试验效率的策略。
