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

doi:10.1002/ana.78311

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

Kennedy Krieger Institute

Research output: Contribution to journal › Article › peer-review

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.

Original language	English (US)
Pages (from-to)	477-486
Number of pages	10
Journal	Annals of neurology
Volume	100
Issue number	3
DOIs	https://doi.org/10.1002/ana.78311
State	Published - Sep 2026

ASJC Scopus subject areas

Neurology
Clinical Neurology

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10.1002/ana.78311

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AU - Vermilion, Jennifer

AU - Paganoni, Sabrina

AU - Plotkin, Scott R.

AU - Quintana, Melanie

AU - Calvert, Sara B.

AU - Whiteman, Ineka T.

AU - Wang, Raymond Y.

AU - Moore, Nadia

AU - Augustine, Erika F.

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AB - 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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The Case for Master Protocols for Rare Neurological Diseases

Abstract

ASJC Scopus subject areas

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