Complement is likely to have a role in refractory generalised myasthenia gravis, but no approved therapies specifically target this system. Results from a phase 2 study suggested that eculizumab, a terminal complement inhibitor, produced clinically meaningful improvements in patients with anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis. We further assessed the efficacy and safety of eculizumab in this patient population in a phase 3 trial.

中文翻译：

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Eculizumab在抗乙酰胆碱受体抗体阳性的难治性重症肌无力（REGAIN）中的安全性和有效性：3期，随机，双盲，安慰剂对照，多中心研究。

补体可能在难治性广义重症肌无力中起作用，但是没有批准的疗法专门针对该系统。来自2期研究的结果表明，依库丽单抗是一种终末补体抑制剂，在抗乙酰胆碱受体抗体阳性的难治性重症肌无力患者中产生了临床上有意义的改善。在一项3期试验中，我们进一步评估了依库丽单抗在该患者人群中的疗效和安全性。