Statistical design of Path to Prevention platform trial in early neuronal alpha-synuclein disease

The Path to Prevention (P2P) platform trial in early neuronal alpha-synuclein disease is designed with 125 participants per active regimen and 96–144 weeks follow-up.

• Why it matters: Early-stage neuronal alpha-synuclein disease lacks efficient, well-modeled interventional designs, and the gap is accelerated evaluation of therapies before Parkinson’s disease becomes clinically manifest.
• What they did: P2P uses a two-stage randomization scheme, longitudinal Bayesian mixed-effects models comparing active regimens to a shared placebo, and supplements placebo estimates with non-randomized PPMI data.
• The result: Simulations using 275 P2P-eligible PPMI participants (Age 60–81; 35% Female; 65% Male) show each regimen is well-powered to detect 40% slowing versus randomized control, with up to 4% power gains per endpoint using registry controls.

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