Objective: To provide continued consortium expertise to provide ongoing innovation and long-term sustainability in a multi-arm, multi-stage (MAMS) disease modification trial for Parkinson’s disease (PD).

Background: Since 2021, the Edmond J Safra Accelerating Clinical Trials in PD (EJS ACT-PD) Initiative convened >90 expert stakeholders, including people affected by PD, to increase research pace. Their input culminated in the EJS ACT-PD MAMS Platform trial protocol which will efficiently assess the clinical and cost-effectiveness of potential disease-modifying therapies (DMTs) for PD. The EJS Foundation has funded the Initiative to continue for an additional 5 years alongside the trial. Stage 2 of the initiative will prioritise future therapy arms; identify and incorporate innovations to improve trial performance and efficiency; and secure funding for relevant trial sub-studies and long-term trial delivery.

Method: A restructured consortium consists of 4 Working Groups: Trial Innovation, Treatment Selection, Funding and Commercialisation, Patient and Public Inclusion and Engagement (PPIE). A Community Advisory Panel and Wider PPIE Network continue to provide diverse input on decisions. Core team support ensures close collaboration and alignment between the Initiative groups and the Trial Committees.

Results: The trial will launch in 2025, recruiting 1,600 participants across 3 therapies vs placebo, from 40 UK sites. An iterative treatment selection process will be updated and implemented to identify promising treatments to include from arm 4 onwards, including engagement with commercial organisations. Innovations in outcome measures, statistical analysis, and recruitment and retention methodology will be considered for trial adaptations via protocol amendments. Funding applications will be developed for trial sub-studies and sustainment of the trial past the first 3 treatment arms.

Conclusion: The EJS ACT-PD Initiative has demonstrated how a dedicated expert consortium can successfully produce a cost-effective trial which is acceptable to patients. Maintaining this resource will provide a crucially important and supportive foundation to ensure the EJS ACT-PD MAMS platform trial can continue to meet the needs of the Parkinson’s community and remain at the forefront of innovation in PD research.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/ejs-act-pd-an-ongoing-initiative-to-support-innovation-and-sustainability-of-a-multi-arm-multi-stage-platform-trial-for-parkinsons-disease/