Objective: To scope and develop infrastructure for the first multi-arm multi-stage (MAMS) platform trial for Parkinson’s disease, the Edmond J Safra ACT-PD initiative (EJS ACT-PD).

Background: This novel approach investigates multiple treatments in parallel allowing for early stopping and replacement of treatments that are ineffective at interim stages. The trial will run for many years and is set to become a main avenue to test disease modifying therapies within the UK. With recruitment anticipated to commence in 2024, developing infrastructure to enable its delivery is critical.

Method: One of 6 working groups (WG), the EJS ACT-PD infrastructure WG brings together national experts in Parkinson’s trials, representatives from the NIHR Clinical Research Network, trial delivery staff including those involved in running MAMS trials as well as two representatives from the EJS ACT-PD Patient and Public Involvement and Engagement WG. It meets monthly to identify and resolve important infrastructure challenges.

Results: The WG has identified 4 key infrastructure challenges:
1. Site capability: a survey has been constructed to scope availability of equipment, experience in administering relevant clinical scales, experience and barriers to remote trial delivery and digital outcomes as well as bio-sample handling and storage at sites.
2. Workforce requirements: the WG is working closely with the NIHR to expand a national rater training programme to include Parkinson’s relevant clinical scales, as well as improving workforce digital literacy, to increase the geographical reach of the trial.
3. Data linkage: with the advent of digitrials and the possibility of capturing outcomes via the use of routine healthcare datasets, the WG is exploring with NHS Digital the possibility of linkage to routine healthcare records.
4. Recruitment tools: to improve trial access, the WG is exploring linking with existing patient registries and recruitment tools, as well as developing resources for reaching under-served communities.

Conclusion: Greater availability of research is a key priority for people with Parkinson’s and their care partners. The trial delivery landscape is rapidly evolving with technology affording us the opportunity to deliver trials that are more patient-centred and inclusive. Realising these benefits will require the identified infrastructure challenges to be addressed, which is the next phase of this project.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/developing-infrastructure-for-a-multi-arm-multi-stage-trial-in-parkinsons/