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The Case for Master Protocols for Rare Neurological Diseases.

Jennifer Vermilion·Sabrina Paganoni·Scott R Plotkin·Melanie Quintana·Sara B Calvert·Ineka T Whiteman·Raymond Y Wang·Nadia Moore·Erika F Augustine
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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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HumansRare DiseasesNervous System DiseasesClinical Trials as TopicNeuronal Ceroid-LipofuscinosesResearch DesignJournal ArticleReview

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Jennifer Vermilion, Sabrina Paganoni, Scott R Plotkin, Melanie Quintana, Sara B Calvert, Ineka T Whiteman, Raymond Y Wang, Nadia Moore, Erika F Augustine. 2026-07-21. The Case for Master Protocols for Rare Neurological Diseases.. https://doi.org/10.1002/ana.78311

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