Evidence map›Paper›PMID 36856727›Full record

ArticleBrain : a journal of neurology2023

Towards a multi-arm multi-stage platform trial of disease modifying approaches in Parkinson's disease.

Tom Foltynie, Sonia Gandhi, Cristina Gonzalez-Robles, Marie-Louise Zeissler, Georgia Mills, Roger Barker, James Carpenter, Anette Schrag, Anthony Schapira, Oliver Bandmann and 7 more

Abstract read
In one paragraph

Article in Brain : a journal of neurology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.

0numbers the graph read from it
0cells of the map it votes in
22citing 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

22 citing papers in PubMed.

  1. Review
  2. Article
  3. Review
  4. Repurposed drug prioritization pipeline for a multi-arm platform trial in clinical Alzheimer's disease.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2026
    Article
  5. Review
  6. Article
  7. Article
  8. Neuroprotection in Parkinson Disease.Neurology and therapy · 2025
    Review
  9. Article
  10. Treatment Selection and Prioritization for the EJS ACT-PD MAMS Trial Platform.Movement disorders : official journal of the Movement Disorder Society · 2025
    Article
  11. Article
  12. Article
  13. Article
  14. Validation of a 5-Year Prognostic Model for Parkinson's Disease.Movement disorders clinical practice · 2024
    Article
  15. Article
  16. Review
  17. Review
  18. If Art Were a Drug: Implications for Parkinson's Disease.Journal of Parkinson's disease · 2024
    Review
  19. Article
  20. Article
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

17 authors.

Tom FoltynieDepartment of Clinical & Movement Neurosciences, UCL Institute of Neurology, London WC1N 3BG, UK.ORCID 0000-0003-0752-1813
Sonia GandhiDepartment of Clinical & Movement Neurosciences, UCL Institute of Neurology, London WC1N 3BG, UK.
Cristina Gonzalez-RoblesDepartment of Clinical & Movement Neurosciences, UCL Institute of Neurology, London WC1N 3BG, UK.
Marie-Louise ZeisslerFaculty of Health, University of Plymouth, Plymouth PL4 9AA, UK.
Georgia MillsDepartment of Clinical & Movement Neurosciences, UCL Institute of Neurology, London WC1N 3BG, UK.
Roger BarkerJohn van Geest Centre for Brain Repair, Department of Clinical Neurosciences, University of Cambridge, Cambridge CB2 0PY, UK.
James CarpenterMRC Clinical Trials Unit at UCL, London WC1V 6LJ, UK.
Anette SchragDepartment of Clinical & Movement Neurosciences, UCL Institute of Neurology, London WC1N 3BG, UK.
Anthony SchapiraDepartment of Clinical & Movement Neurosciences, UCL Institute of Neurology, London WC1N 3BG, UK.ORCID 0000-0002-3018-3966
Oliver BandmannSheffield Institute for Translational Neuroscience, University of Sheffield, Sheffield S10 2HQ, UK.ORCID 0000-0001-6753-7847
Stephen MullinFaculty of Health, University of Plymouth, Plymouth PL4 9AA, UK.
Joy DuffenMRC Clinical Trials Unit at UCL, London WC1V 6LJ, UK.
Kevin McFarthingParkinson's Research Advocate, Oxford, UK.
Jeremy ChatawayDepartment of Clinical & Movement Neurosciences, UCL Institute of Neurology, London WC1N 3BG, UK.ORCID 0000-0001-7286-6901
Mahesh ParmarMRC Clinical Trials Unit at UCL, London WC1V 6LJ, UK.
Camille CarrollFaculty of Health, University of Plymouth, Plymouth PL4 9AA, UK.
EJS ACT-PD Consortium

Funding

Medical Research Council G106/1249Medical Research Council MR/T008199/1Medical Research Council MR/V007068/1Medical Research Council MR/W003724/1Medical Research Council MR/X023494/1Wellcome Trust 205167/Z/16/Z
6 · The paper itself

Abstract

An increase in the efficiency of clinical trial conduct has been successfully demonstrated in the oncology field, by the use of multi-arm, multi-stage trials allowing the evaluation of multiple therapeutic candidates simultaneously, and seamless recruitment to phase 3 for those candidates passing an interim signal of efficacy. Replicating this complex innovative trial design in diseases such as Parkinson's disease is appealing, but in addition to the challenges associated with any trial assessing a single potentially disease modifying intervention in Parkinson's disease, a multi-arm platform trial must also specifically consider the heterogeneous nature of the disease, alongside the desire to potentially test multiple treatments with different mechanisms of action. In a multi-arm trial, there is a need to appropriately stratify treatment arms to ensure each are comparable with a shared placebo/standard of care arm; however, in Parkinson's disease there may be a preference to enrich an arm with a subgroup of patients that may be most likely to respond to a specific treatment approach. The solution to this conundrum lies in having clearly defined criteria for inclusion in each treatment arm as well as an analysis plan that takes account of predefined subgroups of interest, alongside evaluating the impact of each treatment on the broader population of Parkinson's disease patients. Beyond this, there must be robust processes of treatment selection, and consensus derived measures to confirm target engagement and interim assessments of efficacy, as well as consideration of the infrastructure needed to support recruitment, and the long-term funding and sustainability of the platform. This has to incorporate the diverse priorities of clinicians, triallists, regulatory authorities and above all the views of people with Parkinson's disease.

Indexed as

COVID-19Parkinson DiseaseAdaptive Clinical Trials as TopicHumanscomplex innovative trial designmulti-armmulti-stageParkinson’s diseaseplatform trial

Identifiers

PMID36856727
PMCPMC10316775

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.