文摘
客观的报告的研究设计阶段2、多中心、随机、安慰剂对照,与这些相应平行的组织研究(阿尔达,NCT05225675)将评估安全、功效、PK, PD和MMN argx - 117的成人。
背景多病灶的运动神经病变(MMN)是一种慢性的免疫介导的神经病变特点是进步的不对称的弱点主要影响远侧上肢。MMN常伴随着anti-GM1 IgM抗体针对轴膜郎飞氏结,导致激活的补体经典驱动后续损伤轴突。丙种球蛋白是唯一被证明有效的治疗。argx - 117是一种新型的单克隆抗体,抑制补体因子2 (C2)。使用MMN的体外模型,argx - 117显示阻止IgM-mediated经典途径补体激活运动神经元和雪旺细胞。argx - 117与MMN正在研究在成人阶段2、多中心、随机、安慰剂对照,与这些相应平行的组织学习。
设计/方法48参与者将招募并分配给两个剂量组各参与者(24)和随机2:1在每个队列接收argx - 117或安慰剂。关键的入选标准包括:诊断可能的或明确的MMN每2010外汇/ pn的指导方针,稳定的丙种球蛋白治疗方案,丙种球蛋白治疗依赖。基于不良事件的主要目标是安全监测和安全评估。二级目标包括评估有效性测量的强度和功能残疾,药物动力学、药效学(C2和CH50)和免疫原性argx - 117。完成16周治疗期后,参与者可能进入一个开放的扩展研究,或输入随访期间安全。
结果这个正在进行的第二阶段研究将评估的安全性和有效性argx - 117与MMN参与者。
结论这项研究的结果将通知和直接argx - 117年未来的研究在这个病人的人口。
脚注
披露:艾伦博士已经收到个人薪酬在500 - 4999美元的范围为Argenyx担任顾问。艾伦博士已经收到个人薪酬在500 - 4999美元的范围为csl贝林担任顾问。艾伦博士已经收到个人薪酬在500 - 4999美元的范围为武田担任顾问。艾伦博士已经收到个人薪酬在500 - 4999美元的范围为Grifols担任顾问。艾伦博士已经收到个人薪酬在500 - 4999美元的范围为Argenx担任顾问。艾伦博士已经收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会grifols。艾伦博士已经收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会Alexion。艾伦博士已经收到个人薪酬在10000 - 49999美元的范围在CSL贝林的演讲人。Nobile-Orazio博士已经收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会ArgenX,比利时。Nobile-Orazio博士已经收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会局部反馈,法国。 Dr. Nobile-Orazio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche, Switzerland. Dr. Nobile-Orazio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL Behring. Dr. Nobile-Orazio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen, USA. The institution of Dr. Nobile-Orazio has received research support from Takeda/Shire. Dr. Peric has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Peric has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mylan. An immediate family member of Dr. Katzberg has received personal compensation for serving as an employee of Sanofi Genzyme. Dr. Katzberg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Katzberg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Katzberg has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Octapharma. Dr. Katzberg has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for UCB. Mrs. Cadour has nothing to disclose. Miss Van de Walle has received personal compensation for serving as an employee of argenx bv. Mrs. Van Hoomissen has received personal compensation for serving as an employee of argenx. Mr. Jacquemyn has received personal compensation for serving as an employee of argenx. Dr. Van de Steen has received personal compensation for serving as an employee of argenx. Dr. Van de Steen has stock in argenx. Dr. van der Pol has nothing to disclose.
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