ISA2 to Master Protocol ARGX-999-2-MG-2000 - an Exploratory, Phase 2a, Randomized, Double-Blinded, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Empasiprubart IV Monotherapy in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- argenx
- 入组人数
- 40
- 试验地点
- 4
- 主要终点
- Incidence of adverse events and serious adverse events in the DBTP
研究概览
简要总结
This study is part of the ADAPT Forward platform study (NCT07294170). ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients' side effects and improve their quality of life.
The aim of this ISA2 is to investigate the effects of empasiprubart in participants with AChR-Ab seropositive generalized myasthenia gravis (gMG).
The ADAPT Forward master protocol is registered on https://clinicaltrials.gov/study/NCT07294170
More information can be found here: https://clinicaltrials.argenx.com/adaptforward2
详细描述
Once the master protocol and ISA2 screening periods are completed, eligible participants will be randomized to receive empasiprubart IV or placebo in the double-blinded treatment period (DBTP). All participants will then receive open-label efgartigimod PH20 SC PFS in the safety follow-up period.
The study duration for each participant is approximately up to 45 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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Is seropositive for anti-acetylcholine receptor antibodies (AChR-Ab).
- •Has confirmed diagnosis of gMG and is Myasthenia Gravis Foundation of America (MGFA) Class II, III, IVa, or IVb.
- •Has documented immunization against encapsulated bacterial pathogens (Neisseria meningitidis and Streptococcus pneumoniae) within 5 years before ISA screening or will complete immunization at least 14 days before the first IMP administration.
排除标准
- •Clinical diagnosis of systemic lupus erythematosus (SLE).
- •Is receiving concurrent complement inhibitors (eg, eculizumab, zilucoplan, ravulizumab, or others). Participants who received zilucoplan or eculizumab >2 months or ravulizumab >6 months before baseline are allowed to participate.
- •Has received an FcRn antagonist, including efgartigimod, within 4 weeks before baseline.
- •Had prior empasiprubart exposure.
研究组 & 干预措施
Efgartigimod PH20 SC PFS
Participants receive open-label efgartigimod PH20 SC PFS in the safety follow-up period
干预措施: Efgartigimod PH20 SC PFS (Combination Product)
Empasiprubart IV
Participants receive empasiprubart IV in the DBTP
干预措施: Empasiprubart IV (Biological)
Placebo IV
Participants receive placebo IV in the DBTP
干预措施: Placebo IV (Other)
结局指标
主要结局
Incidence of adverse events and serious adverse events in the DBTP
时间窗: Up to 12 weeks
次要结局
- Proportion of participants who have a positive PASS at week 12(Up to 12 weeks)
- MG-ADL total score change from baseline at week 12(Up to 12 weeks)
- QMG total score change from baseline at week 12(Up to 12 weeks)
- MG-ADL total score change from baseline over time up to week 12(Up to 12 weeks)
- QMG total score change from baseline over time up to week 12(Up to 12 weeks)
- Proportion of participants reaching MSE at any point by week 12(Up to 12 weeks)
- Proportion of participants who have ≥3-point reduction in MG-ADL at week 12(Up to 12 weeks)
- Proportion of participants who have ≥5-point reduction in QMG at week 12(Up to 12 weeks)
- Proportion of participants who have a 50% MG-ADL total score improvement at week 12(Up to 12 weeks)