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Therapeutic Effects of Batoclimab in Chinese Patients with Generalized Myasthenia Gravis: A Double-Blinded, Randomized, Placebo-Controlled Phase II Study

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机构: [1]Fudan Univ, Huashan Hosp, Huashan Rare Dis Ctr, Dept Neurol, Shanghai, Peoples R China [2]Shandong First Med Univ, Dept Neurol, Affiliated Hosp 1, Jinan, Peoples R China [3]Shandong Prov Qianfoshan Hosp, Jinan, Peoples R China [4]Cent South Univ, Xiangya Hosp, Dept Neurol, Changsha, Peoples R China [5]Capital Med Univ, Xuanwu Hosp, Dept Neurol, Beijing, Peoples R China [6]Fujian Med Univ, Dept Neurol, Union Hosp, Fuzhou, Peoples R China [7]Beijing Hosp, Dept Neurol, Beijing, Peoples R China [8]Sichuan Univ, West China Hosp, Dept Neurol, Chengdu, Peoples R China [9]Harbour BioMed, Shanghai, Peoples R China [10]Natl Ctr Neurol Disorders, Shanghai, Peoples R China
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关键词: Batoclimab Neonatal Fc receptor Generalized myasthenia gravis Safety Efficacy

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Introduction We investigated the safety and explore potential efficacy of batoclimab administered subcutaneously in Chinese patients with generalized myasthenia gravis (gMG). Methods A randomized, double-blinded, placebo-controlled, parallel phase II study was conducted. First, in the double-blinded treatment period, eligible patients received batoclimab (680 mg), batoclimab (340 mg), or placebo on days 1, 8, 15, 22, 29, and 36. In the open-label treatment period, patients received batoclimab (340 mg) on days 50, 64, and 78. In the follow-up period, patients were examined on days 92, 106, and 120. The primary endpoint was Myasthenia Gravis Activities of Daily Living (MG-ADL) score change on day 43 from baseline. Results In total, 30 eligible patients were enrolled, with 11, 10, and 9 patients in the batoclimab 680 mg, batoclimab 340 mg, and placebo groups, respectively. MG-ADL score changes from baseline to day 43 were -2.2 +/- 0.9, -4.7 +/- 0.6, and -4.4 +/- 1.0 in the placebo, batoclimab 340 mg, and 680 mg groups, respectively. Similar changes were observed in Quantitative Myasthenia Gravis, Myasthenia Gravis Composite, and 15-item Myasthenia Gravis Quality of Life scores in the placebo, batoclimab 340 mg, and 680 mg groups, respectively. The proportion of patients with clinically significant improvement on day 43 was higher in the batoclimab groups. On day 120, all four scales in the placebo group had more significant improvement compared with the batoclimab groups, with total serum IgG levels reaching a plateau. No death or treatment-emergent adverse events (TEAEs) led to study discontinuation. Conclusion Batoclimab is effective and safe in Chinese patients with gMG.

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大类 | 3 区 医学
小类 | 4 区 临床神经病学
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大类 | 3 区 医学
小类 | 3 区 临床神经病学
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出版当年[2020]版:
Q1 CLINICAL NEUROLOGY
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Q1 CLINICAL NEUROLOGY

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第一作者机构: [1]Fudan Univ, Huashan Hosp, Huashan Rare Dis Ctr, Dept Neurol, Shanghai, Peoples R China [10]Natl Ctr Neurol Disorders, Shanghai, Peoples R China
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通讯机构: [1]Fudan Univ, Huashan Hosp, Huashan Rare Dis Ctr, Dept Neurol, Shanghai, Peoples R China [10]Natl Ctr Neurol Disorders, Shanghai, Peoples R China
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