/ Active, not recruitingPhase 1/2 ACT001胶囊在复发性视神经脊髓炎谱系疾病相关性视神经炎受试者中安全性和药代动力学的开放、剂量递增I/IIa期临床研究
[Translation] An open-label, dose-escalation Phase I/IIa clinical study of the safety and pharmacokinetics of ACT001 capsules in subjects with recurrent neuromyelitis optica spectrum disorder-associated optic neuritis
主要目的: 1) 评估NMOSD-ON受试者口服ACT001的安全性和耐受性。 2) 评估NMOSD-ON受试者单次及多次给药后ACT001及其主要代谢产物的药代动力学特征。 次要目的: 1) 初步评估ACT001的中枢神经系统抗炎、减少复发作用。 2) 为II期试验探索推荐剂量。 探索性目的: 评估给药前后视觉功能、水通道蛋白4抗体(AQP4 IgG)的滴度(或浓度)以及其他生物标志物的变化。
[Translation] Primary objectives: 1) To evaluate the safety and tolerability of oral administration of ACT001 in NMOSD-ON subjects. 2) To evaluate the pharmacokinetic characteristics of ACT001 and its major metabolites after single and multiple administration in NMOSD-ON subjects. Secondary objectives: 1) To preliminarily evaluate the anti-inflammatory and relapse-reducing effects of ACT001 in the central nervous system. 2) To explore the recommended dose for Phase II trials. Exploratory objectives: To evaluate the changes in visual function, titer (or concentration) of aquaporin 4 antibody (AQP4 IgG) and other biomarkers before and after administration.
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