Evidence map›Paper›PMID 42478739›Full record

ReviewAnnals of neurology2026

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

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Annals of neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Jennifer VermilionDepartment of Neurology, University of Rochester Medical Center, Rochester, New York, USA.ORCID https://orcid.org/0000-0003-3893-2922
Sabrina PaganoniSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Scott R PlotkinDepartment of Neurology and Cancer Institute; Mass General Brigham, Harvard Medical School, Boston, Massachusetts, USA.
Melanie QuintanaBerry Consultants, Austin, Texas, USA.
Sara B CalvertClinical Trials Transformation Initiative, Duke University, Durham, North Carolina, USA.ORCID https://orcid.org/0000-0003-4229-2729
Ineka T WhitemanBatten Disease Support, Research and Advocacy Foundation, Columbus, Ohio, USA.
Raymond Y WangDepartment of Metabolic Disorders, Rady Children's Health, Orange, California, USA.
Nadia MooreClinical Trials Unit, Kennedy Krieger Institute, Baltimore, Maryland, USA.
Erika F AugustineClinical Trials Unit, Kennedy Krieger Institute, Department of Neurology, Johns Hopkins Medicine, Baltimore, Maryland, USA.ORCID https://orcid.org/0000-0001-7257-8985

Funding

Clinical Coordinating Center Network of Excellence in Neuroscience Clinical TrialU01NS077179 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI Merit E CUDKOWICZ, Aleksandar Videnovic · 2011 to 2026
$43.4M
Network of Excellence in Neuroscience Clinical Trials (NEXT)-DCCU01NS077352 · NINDS · UNIVERSITY OF IOWA · PI CHRISTOPHER S. COFFEY · 2011 to 2026
$28.1M
University of Rochester Clinical Site within the NEXT Network of Clinical TrialsU24NS107165 · NINDS · UNIVERSITY OF ROCHESTER · PI ROBERT G. HOLLOWAY · 2018 to 2026
$3.2M
Kennedy Krieger Institute - Johns Hopkins University NeuroNEXT SiteU24NS134009 · NINDS · HUGO W. MOSER RES INST KENNEDY KRIEGER · PI Erika Augustine, Ellen M. Mowry · 2023 to 2026
$1.8M
FDA HHS U18 FD005292NINDS NIH HHS U01 NS077179NINDS NIH HHS U01 NS077352NINDS NIH HHS U24 NS107165NINDS NIH HHS U24 NS134009
6 · The paper itself

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.

Indexed as

Clinical Trials as TopicNervous System DiseasesRare DiseasesResearch DesignHumansNeuronal Ceroid-Lipofuscinoses

Identifiers

PMID42478739
PMCPMC13495805

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.