A platform trial approach to proof-of-concept (POC) studies in autism spectrum disorder: Autism spectrum POC initiative (ASPI)

ABSTRACT:

Background

Over the past decade, autism spectrum disorder (ASD) research has blossomed, and multiple clinical trials have tested potential interventions, with varying results and no clear demonstration of efficacy. Lack of clarity concerning appropriate biological mechanisms to target and lack of sensitive, objective tools to identify subgroups and measure symptom changes have hampered the efforts to develop treatments. A platform trial for proof-of-concept studies in ASD could help address these issues. A major goal of a platform trial is to find the best treatment in the most expeditious manner, by simultaneously investigating multiple treatments, using specialized statistical tools for allocation and analysis. We describe the setup of a platform trial and perform simulations to

SUBMITTER:  

PROVIDER: S-EPMC10025278 | biostudies-literature | 2023 Jan

REPOSITORIES: biostudies-literature