Considerations for Amyotrophic Lateral Sclerosis (ALS) Clinical Trial Design.

ABSTRACT: Thoughtful clinical trial design is critical for efficient therapeutic development, particularly in the field of amyotrophic lateral sclerosis (ALS), where trials often aim to detect modest treatment effects among a population with heterogeneous disease progression. Appropriate outcome measure selection is necessary for trials to provide decisive and informative results. Investigators must consider the outcome measure's reliability, responsiveness to detect change when change has actually occurred, clinical relevance, and psychometric performance. ALS clinical trials can also be performed more efficiently by utilizing statistical enrichment techniques. Innovations in ALS prediction models allow for selection of participants with less heterogeneity in disease progression rates without requi

SUBMITTER: Fournier CN 

PROVIDER: S-EPMC9275386 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature