Outcome Measures for Disease-Modifying Trials in Parkinson's Disease: Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative.

ABSTRACT:

Background

Multi-arm, multi-stage (MAMS) platform trials can accelerate the identification of disease-modifying treatments for Parkinson's disease (PD) but there is no current consensus on the optimal outcome measures (OM) for this approach.

Objective

To provide an up-to-date inventory of OM for disease-modifying PD trials, and a framework for future selection of OM for such trials.

Methods

As part of the Edmond J Safra Accelerating Clinical Trials in Parkinson Disease (EJS ACT-PD) initiative, an expert group with Patient and Public Involvement and Engagement (PPIE) representatives' input reviewed and evaluated available evidence on OM for potential use in trials to delay progression of PD. Each OM was ranked based on aspects such as validity, sensitivity to change, p

SUBMITTER: Gonzalez-Robles C 

PROVIDER: S-EPMC10578294 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature