[Translation] A study of pediatric and adult patients with macrophage activation syndrome (MAS) secondary to Still's disease (including symptomatic juvenile idiopathic arthritis and adult-onset Still's disease) or MAS secondary to systemic lupus erythematosus A two-cohort, open-label, single-arm, multicenter study to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of Emapalumab
本研究主要目的是证明 Emapalumab 治疗症状性青少年特发性关节炎和成人发病型斯蒂尔病继发巨噬细胞活化综合征患者及儿童和成人系统性红斑狼疮继发巨噬细胞活化综合征患者的疗效。
本研究次要目的是证明Emapalumab 在糖皮质激素逐渐减量方面的疗效;评估 Emapalumab 治疗起效时间;评估 Emapalumab 在总体缓解方面的疗效;评估Emapalumab 治疗的持续疗效;评估患者接受 Emapalumab 治疗后的生存情况;评估 Emapalumab 的安全性和耐受性;评估接受 Emapalumab 治疗的患者报告的巨噬细胞活化综合征结局;确定 Emapalumab 的药代动力学特征;确定 Emapalumab 的药效学特征;确定Emapalumab 的免疫原性。
[Translation] The primary purpose of this study is to demonstrate the efficacy of Emapalumab in the treatment of symptomatic juvenile idiopathic arthritis and macrophage activation syndrome secondary to adult-onset Still's disease and in pediatric and adult patients with macrophage activation syndrome secondary to systemic lupus erythematosus. Efficacy.
The secondary purposes of this study are to demonstrate the efficacy of Emapalumab in tapering glucocorticoids; to evaluate the time to onset of effect of Emapalumab; to evaluate the efficacy of Emapalumab in overall response; to evaluate the sustained efficacy of Emapalumab treatment; to evaluate the survival of patients after receiving Emapalumab treatment status; to assess the safety and tolerability of Emapalumab; to assess the outcomes of macrophage activation syndrome reported in patients treated with Emapalumab; to determine the pharmacokinetics of Emapalumab; to determine the pharmacodynamics of Emapalumab; to determine the immunogens of Emapalumab sex.