Usability, efficacy, and safety of candidate tocilizumab biosimilar CT-P47 self-administration via auto-injector and pre-filled syringe in patients with rheumatoid arthritis: a single-arm, open-label, phase 3 study

CT-P47 is a candidate tocilizumab biosimilar that is currently in clinical development. We assessed the usability of CT-P47 self-administration via auto-injector (AI) in patients with rheumatoid arthritis (RA). This was a 12-week, single-arm, open-label, multiple-dose, Phase 3 study. Patients self-injected CT-P47 (162 mg/0.9 mL) via AI at Weeks 0 and 2, and then every other week via pre-filled syringe (PFS) from Week 4 through Week 10. The primary endpoint was POST-Self-Injection Assessment Questionnaire (SIAQ) at Week 2. Efficacy, safety, and immunogenicity were also assessed. Thirty-three patients were enrolled. Mean scores for all POST-SIAQ domains at Week 2 exceeded 8, except for ‘self-confidence’ (7.11) and ‘satisfaction with self-injection’ (7.98), indicating positive patient experiences with CT-P47 AI. Furthermore, an observer-completed checklist found that all patients successfully followed the required steps for self-injection. Efficacy, assessed by Disease Activity Score in 28 joints and its components, showed improvements from baseline to Week 12. No new safety signals were observed; the most common adverse events were leukopenia, neutropenia, and injection-site reaction, each occurring in 3 (9.1%) patients. CT-P47 self-administered using an AI showed successful usability in patients with moderate-to-severe RA. ClinicalTrials.gov identifier: NCT05725434.

CT-P47是一款在研托珠单抗（tocilizumab）生物类似药候选品种。本研究评估了CT-P47通过自动注射器（auto-injector, AI）实施自我给药在类风湿关节炎（rheumatoid arthritis, RA）患者中的使用可行性。本研究为一项为期12周的单臂、开放标签、多剂量3期临床试验。受试者于第0周和第2周通过自动注射器自我注射CT-P47（162mg/0.9mL），自第4周至第10周则改为每两周通过预充式注射器（pre-filled syringe, PFS）给药。本研究的主要终点为第2周的注射后自我给药评估问卷（POST-Self-Injection Assessment Questionnaire, SIAQ）评分。同时还对疗效、安全性及免疫原性进行了评估。本研究共纳入33例患者。第2周时，除“自信心”维度评分为7.11、“自我注射满意度”维度评分为7.98外，POST-SIAQ所有维度的平均评分均超过8分，提示患者对CT-P47自动注射器给药的体验良好。此外，观察者填写的核查清单显示，所有受试者均成功完成了自我注射的规定操作步骤。以28关节疾病活动度评分（Disease Activity Score in 28 joints）及其分项指标评估的疗效结果显示，从基线至第12周患者病情有所改善。未观察到新的安全性信号；最常见的不良事件为白细胞减少症、中性粒细胞减少症及注射部位反应，各有3例（9.1%）患者发生。综上，采用自动注射器自我给药的CT-P47在中重度类风湿关节炎患者中展现出良好的使用可行性。临床试验注册号：ClinicalTrials.gov标识符：NCT05725434。