Post-hoc analysis of radicava in amyotrophic lateral sclerosis published

Mitsubishi Tanabe Pharma America, Inc. announced results from a post-hoc analysis that showed how the Radicava (edaravone) Phase III study design minimized heterogeneity of the study population to successfully demonstrate a treatment effect in amyotrophic lateral sclerosis (ALS) in 24 weeks. Because ALS is a heterogeneous disease, and no two patients follow the exact same trajectory of decline over time, demonstrating a clinical response in trials can be challenging. The findings, published in Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, illustrate the importance of employing enrichment strategies when designing studies in ALS.

The patient population for the pivotal Phase III study (Study 19) was enriched with participants who would have tendency for disease progression, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), during the 24-week study time frame. Study authors concluded that reducing the number of slow progressing patients in Study 19 showed a treatment effect with edaravone at 24 weeks.

See: "Post-hoc analyses of the edaravone clinical trials Study 16 and Study 19: a step toward more efficient clinical trial designs in amyotrophic lateral sclerosis" Joseph M. Palumbo et al. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration.