ArticleMovement disorders : official journal of the Movement Disorder Society2026
Feasibility of Simon Two-Stage Futility Trials in People with Early, Symptomatically Treated Parkinson's Disease.
Article in Movement disorders : official journal of the Movement Disorder Society, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
backgroundDisease-modifying treatments are a critical unmet need in Parkinson's disease (PD). Phase 2 futility trials using the Simon two-stage design offer an efficient strategy to evaluate candidate treatments in an early PD population.
objectiveThe aim was to assess the feasibility of Simon two-stage futility trials in early, levodopa-treated PD subjects using historical patient-level clinical trial datasets.
methodsWe analyzed patient-level data from two completed trials, that is, STEADY-PD 3 (n = 336, untreated at baseline) and NET-PD LS1 (n = 1741, treated at baseline). We defined disability progression as a ≥5-point worsening on the motor (Part III) subscore of the Unified Parkinson's Disease Rating Scale at 12 and 24 months. We tested multiple scenarios, including the reanalysis of STEADY-PD 3 participant data after starting dopaminergic treatment. We assessed predictors of progression using logistic regression analysis and calculated sample size estimates.
resultsBoth trials showed similar progression rates at 12 months (~26%) and 24 months (~35%). In NET-PD LS1, older age and lower baseline motor scores were associated with worsening; no predictors were significant in STEADY-PD 3. We estimate that in futility trials that use OFF-state scores to assess motor performance, 39 early PD participants are required to detect significant disability worsening over an observation period of 12 months.
conclusionsPhase 2 futility trials using the Simon two-stage methodology are feasible in early PD, including in treated and untreated patients. OFF-state scores are preferable to ON-state scores as the primary outcome measure. Futility trials offer a smaller-scale, faster, and cost-effective approach to assessing new candidate treatments in PD. © 2025 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
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