Statistical analysis plan for the motor neuron disease systematic multi-arm adaptive randomised trial (MND-SMART).

ABSTRACT:

Background

MND-SMART is a platform, multi-arm, multi-stage, multi-centre, randomised controlled trial recruiting people with motor neuron disease. Initially, the treatments memantine and trazodone will each be compared against placebo, but other investigational treatments will be introduced into the trial later. The co-primary outcomes are the Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALS-FRS-R) functional outcome, which is assessed longitudinally, and overall survival.

Methods

Initially in MND-SMART, participants are randomised 1:1:1 via a minimisation algorithm to receive placebo or one of the two investigational treatments with up to 531 to be randomised in total. The comparisons between each research arm and placebo will be conducted in four stages,

SUBMITTER: Parker RA 

PROVIDER: S-EPMC9843918 | biostudies-literature | 2023 Jan

REPOSITORIES: biostudies-literature