A Phase 3b, Randomized, Open-label, Parallel-Group Study to Evaluate Different Dosing Regimens of Intravenous Efgartigimod to Maximize and Maintain Clinical Benefit in Patients With Generalized Myasthenia Gravis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- argenx
- 入组人数
- 69
- 试验地点
- 78
- 主要终点
- Mean of the average MG-ADL total score change from baseline during the visit of week 1 through week 21 by regimen arm
研究概览
简要总结
The purpose of this open-label study is to investigate the efficacy, safety, and tolerability of a continuous regimen of efgartigimod compared with a cyclic regimen in participants with Generalized Myasthenia Gravis (gMG).
Participants will receive efgartigimod throughout the study. The participants will be randomized to the continuous regimen arm or to the cyclic regimen arm. The study consists of a part A (regimen comparison period) where participants will continue the treatment based on the treatment regimen arm they were assigned at randomization. Following part A, participants will enter part B (extension period) where all participants will receive efgartigimod in the continuous regimen. The study duration for participants is up to 138 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •At least 18 years of age, at the time of signing the informed consent.
- •Diagnosed with Generalized Myasthenia Gravis (gMG) with confirmed documentation and supported by a physical exam and confirmed seropositivity for anti-acetylcholine receptor antibodies (AChR-Abs).
- •Meets the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, or IV
排除标准
- •Any other condition that, in the opinion of the investigator, would interfere with an accurate assessment of the clinical symptoms of gMG and/or put the participant at undue risk
- •A thymectomy within 3 months of screening
- •Use of the following prior or concomitant therapies:
- •intravenous immunoglobulin (IVIg) or subcutaneous immunoglobulin (SCIg) within 14 days of day 1
- •Rituximab within 6 months of day 1
- •Eculizumab within 1 month of day 1
- •Other monoclonal antibodies (eg, adalimumab, tocilizumab, ixekizumab)
研究组 & 干预措施
Efgartigimod IV - continuous regimen
Participants receiving efgartigimod IV on a continuous regimen
干预措施: Efgartigimod IV (Biological)
Efgartigimod IV - cyclic regimen
Participants receiving efgartigimod IV on a cyclic regimen
干预措施: Efgartigimod IV (Biological)
结局指标
主要结局
Mean of the average MG-ADL total score change from baseline during the visit of week 1 through week 21 by regimen arm
时间窗: Up to 21 weeks
The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The total score varies between 0 and 24, with higher total scores indicating more impairment.
次要结局
- Percentage of time participants have a change in MG-ADL total score of at least 2 points from baseline during Week 4 through Week 21(Up to 21 weeks)
- Incidence of AEs, SAEs and AESIs(Up to 136 weeks)
- Change from baseline in the MG-ADL total score over time(Up to 126 weeks)
- Normalized area under the effect curve (AUEC) of MG-ADL total score improvement from baseline(Up to 21 weeks)
- Characterization of MG-ADL total score change from baseline(Up to 21 weeks)
- Percentage of participants who have a ≥ 2, 3, 4, or 5 points improvement in MG-ADL total score from baseline(Up to 21 weeks)
- Percentage of participants who achieve MSE, defined as a MG-ADL total score of 0 or 1(Up to 21 weeks)