A randomized, double-blind, placebo-controlled, 52-week study (no. NCT03068468) evaluated gosuranemab, an anti-tau monoclonal antibody, in the treatment of progressive supranuclear palsy (PSP). In total, 486 participants dosed were assigned to either gosuranemab (n = 321) or placebo (n = 165). Efficacy was not demonstrated on adjusted mean change of PSP Rating Scale score at week 52 between gosuranemab and placebo (10.4 versus 10.6, P = 0.85, primary endpoint), or at secondary endpoints, resulting in discontinuation of the open-label, long-term extension. Unbound N-terminal tau in cerebrospinal fluid decreased by 98% with gosuranemab and increased by 11% with placebo (P < 0.0001). Incidences of adverse events and deaths were similar between groups. This well-powered study suggests that N-terminal tau neutralization does not translate into clinical efficacy.

一项为期 52 周的随机、双盲、安慰剂对照研究（编号 NCT03068468）评估了 gosuranemab（一种抗 tau 单克隆抗体）在治疗进行性核上性麻痹 (PSP) 中的作用。总共有 486 名参与者被分配到 gosuranemab ( n  = 321) 或安慰剂 ( n  = 165)。Gosuranemab 和安慰剂在第 52 周时 PSP 评定量表评分的调整平均变化（10.4 对 10.6，P  = 0.85，主要终点）或次要终点未证实疗效，导致停用开放标签、长期扩大。脑脊液中未结合的 N 末端 tau 使用 gosuranemab 减少了 98%，安慰剂增加了 11%（P < 0.0001)。各组之间不良事件和死亡的发生率相似。这项强有力的研究表明，N 末端 tau 中和不会转化为临床疗效。