Edmond J. Safra Accelerating Clinical Trials in Parkinson’s disease (EJS ACT-PD) - a Multi-arm Multi-stage Platform Trial for potential disease modifying approaches

Research summary

Hypothesis: Selected treatments that target key neurodegenerative / neuroprotective pathways in PD will slow disease progression compared to placebo. Aims: • To assess the clinical and cost-effectiveness of at least 3 potential disease modifying therapies (DMTs) compared with a shared placebo on the rate of progression of Parkinson's Disease (PD). • To establish sustainable infrastructure for the long-term evaluation of clinical and cost effectiveness of further potential DMTs in PD. • To recruit a representative study population. Methods: • We will recruit 1200 participants from 40 UK sites over 3-4 years. Participants will be randomised 1:1:1 to one of 2 active treatment arms or placebo. • The primary outcome is rate of change in MDS-UPDRS parts I and II combined. Early stage analyses will be undertaken at approximately 2 and 3 years following trial opening,with potential for discontinuation of therapy arms which do not fulfil continuation criteria. Formal efficacy analysis will be undertaken when 266 participants from each trial arm have completed 36 months of treatment. • Core-funded research staff will support delivery at key sites in different UK regions to maximise inclusivity and participant retention. Study visits will be at 3 months,6 months and then 6-monthly for a total treatment duration of 36 months and be deliverable remotely or in-person. The trial delivery infrastructure that we create will support the addition of future therapy trial arms.

Principal Investigator

Dr Ashwini Oswal

Contact us

Email: dendron@ouh.nhs.uk

IRAS number

1009921