Is It Possible to Conduct a Multi-Arm Multi-Stage Platform Trial in Parkinson's Disease: Lessons Learned from Other Neurodegenerative Disorders and Cancer.

ABSTRACT: Many potential disease modifying therapies have been identified as suitable for clinical evaluation in Parkinson's disease (PD). Currently, the evaluation of compounds in phase II and phase III clinical trials in PD are set up in isolation, a process that is lengthy, costly and lacks efficiency. This review will introduce the concept of a multi-arm, multi-stage (MAMS) trial platform which allows for the assessment of several potential therapies at once, transitioning seamlessly from a phase II safety and efficacy study to a phase III trial by means of an interim analysis. At the interim checkpoint, ineffective arms are dropped and replaced by new treatment arms, thereby allowing for the continuous evaluation of interventions. MAMS trial platforms already exist for prostate, renal and oroph

SUBMITTER: Zeissler ML 

PROVIDER: S-EPMC7242843 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature