[Translation] A two-cohort, open-label, single-arm, multicenter study to evaluate the efficacy, safety and tolerability, pharmacokinetics, and pharmacodynamics of emapalumab in children and adults 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
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 objective of this study is to demonstrate the efficacy of Emapalumab in the treatment of patients with symptomatic juvenile idiopathic arthritis and adult-onset Still's disease with secondary macrophage activation syndrome and children and adults with systemic lupus erythematosus with secondary macrophage activation syndrome.
The secondary objectives of this study are to demonstrate the efficacy of Emapalumab in the tapering of glucocorticoids; to evaluate the duration of the onset of Emapalumab treatment; to evaluate the efficacy of Emapalumab in overall remission; to evaluate the sustained efficacy of Emapalumab treatment; to evaluate the survival of patients after Emapalumab treatment; to evaluate the safety and tolerability of Emapalumab; to evaluate the patient-reported outcomes of macrophage activation syndrome in patients treated with Emapalumab; to determine the pharmacokinetic characteristics of Emapalumab; to determine the pharmacodynamic characteristics of Emapalumab; and to determine the immunogenicity of Emapalumab.