An orally administered, fixed‐dose coformulation of sodium phenylbutyrate‐taurursodiol (PB‐TURSO) significantly slowed functional decline in a randomized, placebo‐controlled, phase 2 trial in ALS (CENTAUR). Herein we report results of a long‐term survival analysis of participants in CENTAUR. In CENTAUR, adults with ALS were randomized 2:1 to PB‐TURSO or placebo. Participants completing the 6‐month (24‐week) randomized phase were eligible to receive PB‐TURSO in the open‐label extension. An all‐cause mortality analysis (35‐month maximum follow‐up post‐randomization) incorporated all randomized participants. Participants and site investigators were blinded to treatment assignments through the duration of follow‐up of this analysis. Vital status was obtained for 135 of 137 participants originally randomized in CENTAUR. Median overall survival was 25.0 months among participants originally randomized to PB‐TURSO and 18.5 months among those originally randomized to placebo (hazard ratio, 0.56; 95% confidence interval, 0.34‐0.92; P = .023). Initiation of PB‐TURSO treatment at baseline resulted in a 6.5‐month longer median survival as compared with placebo. Combined with results from CENTAUR, these results suggest that PB‐TURSO has both functional and survival benefits in ALS.

中文翻译：

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苯丁酸钠-牛磺二醇治疗肌萎缩侧索硬化的 CENTAUR 试验参与者的长期生存率

在 ALS (CENTAUR) 的一项随机、安慰剂对照、2 期试验中，口服固定剂量的苯丁酸钠-牛磺二醇 (PB-TURSO) 复合制剂显着减缓了功能衰退。在此，我们报告了 CENTAUR 参与者的长期生存分析结果。在 CENTAUR 中，患有 ALS 的成年人以 2:1 的比例随机分配到 PB-TURSO 或安慰剂组。完成 6 个月（24 周）随机阶段的参与者有资格在开放标签扩展中接受 PB-TURSO。一项全因死亡率分析（随机化后最长 35 个月的随访）纳入了所有随机参与者。在此分析的随访期间，参与者和现场调查人员对治疗分配不知情。最初在 CENTAUR 随机分配的 137 名参与者中有 135 名获得了生命状态。中位总生存期为 25。最初随机分配至 PB-TURSO 的参与者中为 0 个月，而最初随机分配至安慰剂的参与者中为 18.5 个月（风险比，0.56；95% 置信区间，0.34-0.92；P = .023)。与安慰剂相比，在基线开始 PB-TURSO 治疗导致中位生存期延长 6.5 个月。结合 CENTAUR 的结果，这些结果表明 PB-TURSO 在 ALS 中具有功能和生存益处。