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Drug Highest PhasePhase 3 |
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Pozelimab与Cemdisiran联合治疗在症状性全身型重症肌无力患者中的有效性和安全性
[Translation] Efficacy and safety of pozelimab combined with cemdisiran in patients with symptomatic generalized myasthenia gravis
主要目的为: 在症状性全身型重症肌无力(gMG)患者中评价pozelimab+cemdisiran对受体征和症状影响的日常功能的影响。 本研究的次要目的为: 1. 评价pozelimab+cemdisiran(即联合治疗)和cemdisiran单药治疗对以下方面的影响: 临床医生评估的重症肌无力(MG)体征和肌力; 症状性gMG患者受体征和症状影响的日常功能(仅cemdisiran单药治疗); 受MG体征和症状影响的日常功能改善的患者比例;临床医生评估的MG体征和肌力改善的患者比例;健康相关的生活质量;出现极轻度MG症状的患者比例;患者和临床医生报告的MG体征和症状。 2. 评价pozelimab+cemdisiran联合治疗和cemdisiran单药治疗的安全性和耐受性。 3. 评估血清中总pozelimab的浓度 4. 评估血浆中总C5的浓度 5. 评估血浆中cemdisiran及其代谢物的浓度 6. 评估pozelimab的免疫原性 7. 评估cemdisiran的免疫原性 8.研究pozelimab+cemdisiran联合治疗和cemdisiran单药治疗对补体激活的影响
[Translation] The primary objective is to evaluate the effect of pozelimab + cemdisiran on daily functioning affected by signs and symptoms in patients with symptomatic generalized myasthenia gravis (gMG). The secondary objectives of this study are to evaluate the effects of pozelimab + cemdisiran (i.e., combination therapy) and cemdisiran monotherapy on the following aspects: Clinician-assessed signs and strength of myasthenia gravis (MG); Daily functioning affected by signs and symptoms in patients with symptomatic gMG (cemdisiran monotherapy alone); The proportion of patients with improvement in daily functioning affected by MG signs and symptoms; The proportion of patients with improvement in MG signs and strength as assessed by clinicians; Health-related quality of life; The proportion of patients with minimal MG symptoms; Patient- and clinician-reported signs and symptoms of MG. 2. Evaluate the safety and tolerability of pozelimab + cemdisiran combination therapy and cemdisiran monotherapy. 3. Evaluate the concentration of total pozelimab in serum 4. Evaluate the concentration of total C5 in plasma 5. Evaluate the concentration of cemdisiran and its metabolites in plasma 6. Evaluate the immunogenicity of pozelimab 7. Evaluate the immunogenicity of cemdisiran 8. Study the effects of pozelimab + cemdisiran combination therapy and cemdisiran monotherapy on complement activation
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