ArticleJournal of Parkinson's disease2023
Outcome Measures for Disease-Modifying Trials in Parkinson's Disease: Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative.
Article in Journal of Parkinson's disease, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.
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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.
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Who cites it
15 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Digital Outcomes as Biomarkers of Disease Progression in Early Parkinson's Disease: A Systematic Review.Movement disorders : official journal of the Movement Disorder Society · 2025Pooled it
- Validating new symptom emergence as a patient-centric outcome measure for PD clinical trials.Parkinsonism & related disorders · 2024Trial
- Clinical trials in late-stage Parkinson's disease.Journal of Parkinson's disease · 2026Review
- Disease-Duration-Specific Percentiles for Prospective Subtyping of Parkinson's Disease: A PPMI-Based Study.Movement disorders clinical practice · 2026Article
- How to advance the pharmacological management of cognitive impairment in Parkinson's disease.Journal of Parkinson's disease · 2026Review
- Evolution of disease modifying therapy clinical trial design and therapeutic endpoints for Parkinson's disease.Journal of Parkinson's disease · 2026Review
- The future of clinical trials? Monitoring mobility as an outcome measure in Parkinson's disease: The mobilise-D study.Journal of Parkinson's disease · 2026Article
- The patient-centric development of an inclusive multi-arm multi-stage trial for Parkinson's disease.Brain communications · 2026Article
- Neuroprotection in Parkinson Disease.Neurology and therapy · 2025Review
- Wrist accelerometry and machine learning sensitively capture disease progression in prodromal Parkinson's disease.NPJ Parkinson's disease · 2025Article
- Evidence for the Link Between Non-Motor Symptoms, Kinematic Gait Parameters, and Physical Function in People with Parkinson's Disease.Bioengineering (Basel, Switzerland) · 2025Article
- Outcome Selection for Research Studies in Movement Disorders.Movement disorders clinical practice · 2024Article
- Digital biomarkers for non-motor symptoms in Parkinson's disease: the state of the art.NPJ digital medicine · 2024Review
- Long-Term Medication Profiles in Parkinson's Disease under Subthalamic Deep Brain Stimulation: A Controlled Study.Movement disorders clinical practice · 2024Article
- Impacts on study design when implementing digital measures in Parkinson's disease-modifying therapy trials.Frontiers in digital health · 2024Article
Corrections and comments
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Authors and funding
24 authors.
Funding
Abstract
backgroundMulti-arm, multi-stage (MAMS) platform trials can accelerate the identification of disease-modifying treatments for Parkinson's disease (PD) but there is no current consensus on the optimal outcome measures (OM) for this approach.
objectiveTo provide an up-to-date inventory of OM for disease-modifying PD trials, and a framework for future selection of OM for such trials.
methodsAs part of the Edmond J Safra Accelerating Clinical Trials in Parkinson Disease (EJS ACT-PD) initiative, an expert group with Patient and Public Involvement and Engagement (PPIE) representatives' input reviewed and evaluated available evidence on OM for potential use in trials to delay progression of PD. Each OM was ranked based on aspects such as validity, sensitivity to change, participant burden and practicality for a multi-site trial. Review of evidence and expert opinion led to the present inventory.
resultsAn extensive inventory of OM was created, divided into: general, motor and non-motor scales, diaries and fluctuation questionnaires, cognitive, disability and health-related quality of life, capability, quantitative motor, wearable and digital, combined, resource use, imaging and wet biomarkers, and milestone-based. A framework for evaluation of OM is presented to update the inventory in the future. PPIE input highlighted the need for OM which reflect their experience of disease progression and are applicable to diverse populations and disease stages.
conclusionWe present a range of OM, classified according to a transparent framework, to aid selection of OM for disease-modifying PD trials, whilst allowing for inclusion or re-classification of relevant OM as new evidence emerges.
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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.