Results
SE, ALS
AMYOTROPHIC LATERAL SCLEROSIS, AMYOTROPHIC LATERAL SCLEROSIS
A total of 134 individuals in PRO‐ACT met inclusion criteria for this analysis and had known mortality information (full analysis set; Fig. Baseline characteristics.Data are presented as mean (SD), range unless otherwise noted.ALS, amyotrophic lateral sclerosis; ALSFRS‐R, Amyotrophic Lateral Sclerosis Functional Rating Scale‐Revised; PB and TURSO, sodium phenylbutyrate and taurursodiol; PRO‐ACT, Pooled Resource Open‐Access ALS Clinical Trials; SD, standard deviation; VC, vital capacity (forced or slow).Denotes covariate used for propensity score matching.At study baseline.This represents the minimum value at baseline. All participants met VC criteria for trial inclusion at screening.Change in ALSFRS‐R total score progression rate from baseline through 24 weeks: CENTAUR trial population versus PRO‐ACT external control group.ALSFRS‐R, Amyotrophic Lateral Sclerosis Functional Rating Scale‐Revised; ITT, intent‐to‐treat; PB and TURSO, sodium phenylbutyrate and taurursodiol; PRO‐ACT, Pooled Resource Open‐Access ALS Clinical Trials; SE, standard error.Modified ITT population, which excluded two participants who died soon after randomization.Compared with the observed median (IQR) overall survival of 23.54 (14.56–39.32) months in the CENTAUR PB and TURSO group,Kaplan–Meier analyses: CENTAUR PB and TURSO group and PRO‐ACT external control group. Kaplan–Meier plot for the CENTAUR PB and TURSO group is from the ITT analysis previously conducted at a cutoff date coinciding with the final participant visit in CENTAUR.