An ISA to Master Protocol ARGX-999-2-MG-2000 for an Exploratory, Phase 2a, Proof-of-Concept Study to Evaluate the Safety, Tolerability, and Efficacy of Empasiprubart IV as Add-On Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- argenx
- 入组人数
- 70
- 试验地点
- 32
- 主要终点
- Incidence of adverse events and serious adverse events in parts A and B
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 ISA1 is to evaluate the safety and therapeutic relevance of empasiprubart as add-on therapy to efgartigimod in participants with AChR-Ab seropositive generalized myasthenia gravis.
The ADAPT Forward master protocol is registered on https://clinicaltrials.gov/study/NCT07294170
More information can be found here: https://clinicaltrials.argenx.com/adaptforward1
详细描述
Once the master protocol and ISA1 screening periods are completed, eligible participants can enroll in the run-in period (part A) where they will receive efgartigimod IV. Eligible participants can then continue to the add-on period (part B) where they will receive both efgartigimod IV and empasiprubart IV.
Participants who are not eligible for part B will continue directly to the safety follow-up period (part C) where they will receive efgartigimod IV only.
The study duration for each participant is approximately up to 54 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 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
- •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 of ISA screening or is willing to receive immunization at least 14 days before the first study drug administration
排除标准
- •Clinical diagnosis of systemic lupus erythematosus (SLE)
- •Any known complement deficiency
- •Current administration of a complement inhibitor or received zilucoplan or eculizumab <2 months or ravulizumab <6 months before the first study drug administration
- •Patients proven to be refractory to efgartigimod (ie, not achieving a clinically meaningful improvement in total Myasthenia Gravis Activities of Daily Living (MG-ADL) score defined as an improvement of ≥2 points)
研究组 & 干预措施
Efgartigimod IV + Empasiprubart IV
Participants receive efgartigimod IV in part A, B and C and empasiprubart IV in part B
干预措施: Efgartigimod IV (Biological)
Efgartigimod IV + Empasiprubart IV
Participants receive efgartigimod IV in part A, B and C and empasiprubart IV in part B
干预措施: Empasiprubart IV (Biological)
Efgartigimod IV (part A + C)
Participants not eligible for part B, receiving efgartigimod IV in part A and C
干预措施: Efgartigimod IV (Biological)
结局指标
主要结局
Incidence of adverse events and serious adverse events in parts A and B
时间窗: Up to 21 weeks
次要结局
- MG-ADL total score change from baseline at week 18 in part B (cycle 2 day 29) compared with MG ADL total score change from baseline at week 4 in part A(Up to 18 weeks)
- Proportion of participants reaching MSE at any point in part B cycles 1 and 2, and part B cycles 1 or 2(Up to 21 weeks)
- MG-ADL total score changes from baseline over time in part B compared with part A(Up to 21 weeks)
- QMG total score change from baseline at week 18 in part B (cycle 2 day 29) compared with QMG total score change from baseline at week 4 in part A(Up to 18 weeks)
- QMG total score changes from baseline over time in part B compared with part A(Up to 21 weeks)
- Proportion of participants who have positive PASS in part B cycles 1 and 2, and part B cycles 1 or 2(Up to 21 weeks)
- Proportion of participants who have a 50% MG ADL total score improvement in part B cycles 1 and 2(Up to 21 weeks)