Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.

STRENGTH ( NCT05386680 ) was a 52-week, phase 3b, single-arm, open-label, multicenter study evaluating OAV101 IT in participants with SMA aged 2 to <18 years who discontinued nusinersen or risdiplam.

STRENGTH (NCT05386680) 是一项为期52周的3b期、单臂、开放标签、多中心研究，评估了在2至<18岁的SMA患者中，已经停止使用nusinersen或risdiplam的参与者使用OAV101 IT的情况。

The primary objective was safety/tolerability.

主要目标是安全性/耐受性。

Twenty-seven participants were enrolled (mean (s.d.) age at OAV101 IT, 7.4 (3.35) years; range, 2.4-17.7 years) with prior nusinersen or risdiplam (mean (s.d.) duration of 4.3 (1.07) years; range, 1.86-6.18 years or 3.0 (2.02); range, 0.39-6.28 years).

共有27名参与者被纳入研究（在OAV101 IT时的平均（标准差）年龄为7.4（3.35）岁；范围为2.4-17.7岁），这些参与者之前接受过nusinersen或risdiplam治疗（平均（标准差）治疗持续时间为4.3（1.07）年；范围为1.86-6.18年或3.0（2.02）；范围为0.39-6.28年）。

All (n = 27) experienced at least one adverse event(AE), most frequently nasopharyngitis (n = 15, 55.6%), pyrexia (n = 14, 51.9%) and vomiting (n = 13, 48.1%).

所有患者(n = 27)至少经历了一次不良事件(AE)，其中最常见的是鼻咽炎(n = 15, 55.6%)、发热(n = 14, 51.9%)和呕吐(n = 13, 48.1%)。

Thirteen participants (48.1%) experienced treatment-related AEs, most frequently vomiting (n = 6, 22.2%), headache (n = 4, 14.8%) and pyrexia (n = 3, 11.1%).

有13名参与者(48.1%)经历了与治疗相关的不良事件(AEs)，其中最常见的是呕吐(n = 6, 22.2%)、头痛(n = 4, 14.8%)和发热(n = 3, 11.1%)。

Serious adverse events (SAEs) were reported for four participants (n = 4, 14.8%) (mostly respiratory infection related).

有四名参与者报告了严重不良事件（SAEs）（n = 4, 14.8%），大多数与呼吸系统感染相关。

No AEs leading to death or study discontinuation were reported.

没有报告导致死亡或研究中断的不良事件（AEs）。

Adverse events of special interest (AESI) reported were in categories of transient thrombocytopenia (n = 8, 29.6%), hepatotoxicity (n = 4, 14.8%) and signs and symptoms that may be suggestive of dorsal root ganglia toxicity (n = 2, 7.4%).

报告的特别关注的不良事件(AESI)包括短暂性血小板减少症(n = 8, 29.6%)、肝毒性(n = 4, 14.8%)以及可能提示背根神经节毒性的体征和症状(n = 2, 7.4%)。

The OAV101 IT safety profile was consistent with findings in treatment-naïve patients.

OAV101 IT的安全性特征与治疗前患者的发现一致。

Trial registration: ClinicalTrials.gov identifier: NCT05386680 .

试验注册：ClinicalTrials.gov注册号：NCT05386680 .