The Case for Master Protocols for Rare Neurological Diseases

IF 7.5 1区医学 Q1 CLINICAL NEUROLOGY

Annals of Neurology Pub Date : 2026-08-21 Epub Date: 2026-07-21 DOI:10.1002/ana.78311

Jennifer Vermilion MD, Sabrina Paganoni MD, PhD, Scott R. Plotkin MD, PhD, Melanie Quintana PhD, Sara B. Calvert PharmD, Ineka T. Whiteman PhD, Raymond Y. Wang MD, Nadia Moore BS, Erika F. Augustine MD, MS

引用次数: 0

Abstract

Master protocol trials allow for simultaneous multiple hypothesis testing within a common framework and might be applicable for rare diseases. In May 2025, the Network for Excellence in Neuroscience Clinical Trials convened a multistakeholder conference to discuss master protocol trials in rare neurological disorders. In this paper, we explore how master protocol trial designs may apply to rare neurological disorders, using the neuronal ceroid lipofuscinoses as an example. Through shared protocol elements and trial infrastructure, master protocols may decrease cost and improve efficiency in testing potential therapeutics in rare disease, accelerating the delivery of urgently needed therapies to patients. ANN NEUROL 2026;100:477–486

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罕见神经系统疾病总方案的案例。

主方案试验允许在一个共同框架内同时进行多个假设检验，可能适用于罕见疾病。2025年5月，神经科学临床试验卓越网络召开了一次多方利益相关者会议，讨论罕见神经疾病的主方案试验。在本文中，我们探讨了主方案试验设计如何应用于罕见的神经系统疾病，以神经元类脂褐膜病为例。通过共享协议要素和试验基础设施，主协议可以降低成本，提高罕见病潜在治疗方法测试的效率，加快向患者提供急需的治疗方法。Ann neurol 2026。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Annals of Neurology 医学-临床神经学

CiteScore

18.00

自引率

1.80%

发文量

270

审稿时长

3-8 weeks

期刊介绍： Annals of Neurology publishes original articles with potential for high impact in understanding the pathogenesis, clinical and laboratory features, diagnosis, treatment, outcomes and science underlying diseases of the human nervous system. Articles should ideally be of broad interest to the academic neurological community rather than solely to subspecialists in a particular field. Studies involving experimental model system, including those in cell and organ cultures and animals, of direct translational relevance to the understanding of neurological disease are also encouraged.