Undisclosed ALS therapy / Treeway, UMC Utrecht, Leiden University Medical Center - LARVOL DELTA

A joint model for assessing Mortality-Adjusted Progression (MAP) in Amyotrophic Lateral Sclerosis: Application to clinical trials of Sodium Phenylbutyrate and Taurursodiol (ALS-MND 2023) - " MAP offers an improved method to assess the treatment effect of ALS therapies on function while taking informative missing data due to death into account. Seven deaths occurred during the 24-week period (PB&TURSO, n = 5 [5.6%]; placebo, n = 2 [4.2%]). The primary analysis in the ITT population (N = 137, unadjusted for mortality) yielded a least-squares mean ALSFRS-R total score of 26.73 at Week 24 in the placebo arm and of 29.05 in the PB&TURSO arm; the respective MAP estimates were 26.72 and 29.04. Minimal differences after adjustment for death resulted in nearly identical treatment effect size: original ITT analysis, 2.32 points (95% CI 0.18–4.46; p = 0.034) and MAP analysis, 2.31 points (95% CI 0.21–4.42; p = 0.031), with slightly increased precision."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders

[VIRTUAL] SS 10.0 Advances in ALS therapy (ICNMD 2021) - No abstract available