Evidence map›Paper›PMID 42802821›Full record

ArticleBrain communications2026

The patient-centric development of an inclusive multi-arm multi-stage trial for Parkinson's disease.

Marie-Louise Zeissler, Matthew Burnell, Thomas R Barber, Yoav Ben-Shlomo, Caroline S Clarke, Sally Collins, Carl Counsell, Joy Duffen, Mark J Edwards, Romy Ellis-Doyle and 16 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Brain communications, 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

26 authors.

Marie-Louise ZeisslerTranslational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne NE1 7RU, UK.ORCID https://orcid.org/0000-0002-6232-4284
Matthew BurnellMedical Research Council Clinical Trials Unit, University College London, London WC1V 6LJ, UK.
Thomas R BarberNational Hospital for Neurology and Neurosurgery, London WC1N 3BG, UK.
Yoav Ben-ShlomoPopulation Health Sciences, University of Bristol, Bristol BS8 2PS, UK.ORCID https://orcid.org/0000-0001-6648-3007
Caroline S ClarkeResearch Department of Primary Care and Population Health, University College London, London NW3 2PF, UK.
Sally CollinsTranslational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne NE1 7RU, UK.
Carl CounsellInstitute of Applied Health Sciences, University of Aberdeen, Aberdeen AB25 2ZD, UK.
Joy DuffenCure Parkinson's, London W1W 6XX, UK.
Mark J EdwardsInstitute of Psychiatry, Psychology & Neuroscience (IoPPN), King's College London, University of London, London SE5 8AB, UK.
Romy Ellis-DoyleDepartment of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.
Cristina Gonzalez-RoblesDepartment of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.
Kate HockeyExpert by Experience, London, UK.
Anna JewellExpert by Experience, London, UK.
Michael LawtonPopulation Health Sciences, University of Bristol, Bristol BS8 2PS, UK.ORCID https://orcid.org/0000-0002-3419-0354
Georgia MillsDepartment of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.
Rebecca PettyFaculty of Health, University of Plymouth, Plymouth, PL6 8BT, UK.
Dorothy SalathielExpert by Experience, London, UK.
Sue WhippsExpert by Experience, London, UK.
Alan WhoneSchool of Physiology, Pharmacology & Neuroscience, University of Bristol, Bristol BS8 1TD, UK.
Sonia GandhiDepartment of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.
Camille B CarrollTranslational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne NE1 7RU, UK.
Thomas FoltynieDepartment of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.ORCID https://orcid.org/0000-0003-0752-1813
James R CarpenterMedical Research Council Clinical Trials Unit, University College London, London WC1V 6LJ, UK.
Roger A BarkerDepartment of Clinical Neurosciences, University of Cambridge, Cambridge CB2 0QQ, UK.
EJS ACT-PD Consortium
EJS ACT-PD Consortium

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

There is an urgent need to speed up clinical trials for potential disease-modifying therapies that slow progression of Parkinson's disease. The Edmond J Safra Accelerating Clinical Trials in Parkinson's Disease (EJS ACT-PD) initiative set out to develop a patient-centric and inclusive platform trial using an innovative multi-arm, multi-stage (MAMS) design. We describe the work of the EJS ACT-PD consortium, which included over 90 experts including people with Parkinson's disease and care partners across six working groups. There were five key challenges: developing an efficient MAMS trial that meets regulatory requirements and is acceptable to people with Parkinson's; mitigating participant heterogeneity; reducing the risk of simultaneous arm closures; introducing different treatment regimens into a MAMS trial; and maximizing deliverability and accessibility of a MAMS trial. The final design was achieved through an iterative process and critical appraisal of data modelling exploring the impact of participant numbers per arm, trial durations, and endpoints. Every aspect of the design was informed by people with Parkinson's disease and care partners whose advice ensured acceptability, deliverability, and accessibility. The EJS ACT-PD trial will start with one placebo and three treatment arms, each with 400 participants, with a primary endpoint at 3 years powered to detect a 30% reduction in rate of progression of MDS-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part I and II summed scores. Interim analyses will test for activity using the summed inverse variance weighted MDS-UPDRS Part I, II, and remote Part III scores. Ineffective arms will be closed, allowing participants to be re-randomized. Cost-effectiveness analysis is embedded. Further promising treatments will be added in future arms, subject to additional funding. To maximize accessibility and inclusivity, the trial will allow for study visits to be conducted remotely based on participant preference. National sites were selected to maximize accessibility and inclusive recruitment with core funded staff placed within experienced trial sites to support training and delivery at less experienced centres. By involving people with Parkinson's and care partners as well as stakeholders with broad scientific expertise, we have developed an inclusive MAMS trial which captures all stages of disease with partial remote delivery and clear design rationales that will significantly speed up the testing of disease-modifying therapies in Parkinson's disease.

Indexed as

multi-arm multi-stageParkinson’s diseasepatient and public involvement and engagementtrial design

Identifiers

PMID42802821
PMCPMC13616524

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.