Objective: To describe the study design and proposed timeline of the first interventional study in  biomarker defined prodromal Parkinson’s Disease (PD).

Background: P2P is a platform, Phase 2 randomized double blind multi-center, multi-regimen clinical trial evaluating the safety and early efficacy of investigational products for the treatment of biomarker-defined prodromal PD. The study is “nested” within the Parkinson’s Progression Marker Initiative (PPMI) and sponsored by the MJFF.

Method: P2P is a perpetual platform trial with a single Master Protocol dictating the conduct of the trial and regimen specific subprotocols outlining intervention specific aspects for each arm.  Qualified participants will be recruited from the PPMI prodromal cohort, based on the presence of aSN neuronal pathology (SAA in spinal fluid), dopaminergic dysfunction (DaTscan imaging) and clinical prodromal features including RBD or hyposmia.

The study’s Multiple Primary Endpoints include 1) DAT imaging as measured by the rate of progression in the mean striatum Specific Binding Ratio (SBR) and 2) rate of progression in the MDS-UPDRS part III score. Secondary endpoints include safety, tolerability and feasibility. The study will have an array of exploratory clinical (including digital) and biomarker measures. Participants will first be randomized in an equal manner among all of the regimen-specific sub-protocols for which they are eligible. After randomization to a specific subprotocol, participants will be randomized to an active arm or placebo (N=125 per arm) in a K:1 ratio with K denoting the number of active interventions. Intervention duration will be at least 24 months (until the last participant in that regimen completes 24 months). The study is 82% powered to detect a slowing in either primary endpoint for each regimen , assuming a 20% slowing in DAT and a 35% slowing in MDS-UPDRS Part III.

Results: Interventions are being selected by a Therapeutic Evaluation Committee from > 15 industry submitted applications. The study targets to start enrolment in the first 2 regimens in the second half of 2024.

Conclusion: We report the design of the first platform interventional study targeting the biomarker-defined prodromal PD population. Platform design allows efficiency of operational infrastructure, ability to share placebo arm and perpetual testing of the promising interventional candidates.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/path-to-prevention-p2p-therapeutics-platform-trial-in-biomarker-defined-prodromal-parkinsons-disease-study-design/