Addressing heterogeneity in amyotrophic lateral sclerosis CLINICAL TRIALS.

ABSTRACT: Amyotrophic lateral sclerosis (ALS) is a debilitating neurodegenerative disorder with complex biology and significant clinical heterogeneity. Many preclinical and early phase ALS clinical trials have yielded promising results that could not be replicated in larger phase 3 confirmatory trials. One reason for the lack of reproducibility may be ALS biological and clinical heterogeneity. Therefore, in this review, we explore sources of ALS heterogeneity that may reduce statistical power to evaluate efficacy in ALS trials. We also review efforts to manage clinical heterogeneity, including use of validated disease outcome measures, predictive biomarkers of disease progression, and individual clinical risk stratification. We propose that personalized prognostic models with use of predictive bioma

SUBMITTER: Goyal NA 

PROVIDER: S-EPMC7496557 | biostudies-literature | 2020 Aug

REPOSITORIES: biostudies-literature