Journal article
How PPMI Enabled the First Interventional Platform Trial to Test Therapies in Participants With Early-Stage Neuronal Alpha-Synuclein Disease
Annals of neurology
05/07/2026
DOI: 10.1002/ana.78250
PMID: 42095573
Abstract
The Path to Prevention (P2P) platform trial is a multicenter, multi-regimen, proof of concept, phase 2A, randomized clinical trial evaluating the safety and early efficacy of investigational products for the treatment of early-stage Neuronal Alpha-Synuclein disease (NSD) populations. The P2P trial is nested within the Parkinson's Progression Markers Initiative (PPMI), which means that its conceptualization, design, and implementation are based on learnings from the PPMI study. P2P will recruit eligible participants from the PPMI cohort. P2P will also leverage PPMI infrastructure including PPMI study sites and PPMI study cores including the site management, data management, biorepository, imaging, statistical, and data science cores for study execution. This paper reviews the conceptual design of P2P focusing on its relationship with PPMI to demonstrate how the connectivity between PPMI and P2P is essential for the success of the P2P study. P2P is an important step forward in clarifying the clinical trial design and regulatory path for interventions in the early-stage NSD population. ANN NEUROL 2026.
Details
- Title: Subtitle
- How PPMI Enabled the First Interventional Platform Trial to Test Therapies in Participants With Early-Stage Neuronal Alpha-Synuclein Disease
- Creators
- Tanya Simuni - Northwestern UniversityChristopher S Coffey - University of IowaCatherine M Kopil - Michael J. Fox FoundationCora Allen-Savietta - Berry & Associates (United States)Barbara Wendelberger - Berry & Associates (United States)Amy Crawford - Berry & Associates (United States)Ruth B Schneider - University of RochesterAndrew Siderowf - University of PennsylvaniaKarl Kieburtz - University of RochesterCaroline M Tanner - University of California, San FranciscoThomas F Tropea - Institute for Neurodegenerative DisordersHeidi Whalen - Institute for Neurodegenerative DisordersLianne Ramia - Institute for Neurodegenerative DisordersCornelia Kamp - University of RochesterCecilia Reyes - Institute for Neurodegenerative DisordersKimberly Fabrizio - Institute for Neurodegenerative DisordersTawny Willson - Michael J. Fox FoundationErika Merriam - Institute for Neurodegenerative DisordersMaggie McGuire Kuhl - Michael J. Fox FoundationSohini Chowdhury - Michael J. Fox FoundationKenneth Marek - Institute for Neurodegenerative DisordersParkinson's Progression Markers Initiative
- Resource Type
- Journal article
- Publication Details
- Annals of neurology
- DOI
- 10.1002/ana.78250
- PMID
- 42095573
- NLM abbreviation
- Ann Neurol
- ISSN
- 1531-8249
- eISSN
- 1531-8249
- Publisher
- Wiley
- Grant note
- Michael J. Fox Foundation for Parkinson's Research AbbVie BIAL Critical Path Institute Edmond J. Safra Foundation Eli Lilly, Gain Therapeutics GE HealthCare Voyager Therapeutics Weston Family Foundation
PPMI - a public-private partnership - is funded by The Michael J. Fox Foundation for Parkinson's Research and funding partners, including AbbVie, Alamar Biosciences, Aligning Science Across Parkinson's, Arrowhead Pharma, AskBio, BIAL, BioArctic, Biohaven, BlueRock Therapeutics, Bristol-Myers Squibb, Calico Labs, Capsida Biotherapeutics, Critical Path Institute, DaCapo Brainscience, Denali, Edmond J. Safra Foundation, Eli Lilly, Gain Therapeutics, GE HealthCare, Genentech, GSK, Insitro, Johnson & Johnson Innovative Medicine, Lundbeck, Merck, Neumora, Neuron23, Novartis, Regeneron, Roche, Sanofi, Tenvie, UCB, VanquaBio, Voyager Therapeutics, and the Weston Family Foundation.
- Language
- English
- Electronic publication date
- 05/07/2026
- Academic Unit
- Biostatistics
- Record Identifier
- 9985161335602771
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