Objective: To design an optimal multi-arm, multi-stage (MAMS) platform trial for disease-modifying therapies (DMTs) in Parkinson’s disease (PD).

Background: The current clinical trials process cannot match the urgent pace required to assess DMTs for PD. Long delays between clinical trial phases, and running multiple sequential trials greatly increases the time, cost, and number of control participants needed to find answers. Site infrastructure is repeatedly set-up and dismantled due to funding interruptions, removing opportunities to build on delivery frameworks and expertise. Recruitment and retention strategies need improvement to target diverse populations and produce generalisable results. The Edmond J Safra Accelerating Clinical Trials in PD (EJS ACT-PD) initiative has been set-up to address such inefficiencies. This type of MAMS trial offers a cost-effective and innovative approach to clinical trial methodology: simultaneous assessment of multiple therapies against a shared placebo cohort all the way to a Phase 3 efficacy assessment; early identification of futile treatments; and seamless replacement of ineffective arms with alternative candidate interventions whilst retaining core trial infrastructure.

Method: Working groups including professional, patient, and carer representation, were tasked with selecting the most efficient trial design, promising treatments and outcome measures, and assessing UK infrastructure. A wide patient and carer network and a Community Advisory Panel ensured perspectives from research-naïve and under-served groups. A national Site Scoping Survey identified current gaps in PD research capacity across the UK.

Results: The trial will be an inclusive, phase 3 study of three therapies vs a shared placebo; the primary outcome will be a patient-reported measure of PD impact. To encourage inclusivity the trial has broad inclusion criteria and allows for either in-person or remote visits to support participation of both participants and sites across the UK. Core trial staff and a tiered delivery model based on site capabilities will support the involvement and development of less experienced sites.

Conclusion: MAMS trials offer an exciting opportunity to accelerate the assessment of potential therapies for Parkinson’s whilst building on national infrastructure and encouraging diverse participant recruitment.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/innovating-clinical-trial-design-to-accelerate-assessment-of-disease-modifying-treatments-for-parkinsons-the-ejs-act-pd-initiative/