2025-522492-28-00招募中2 期
Master Protocol Full Title: A Master Protocol for an Exploratory, Phase 2a, Proof-of-Concept Platform Study to Evaluate the Safety, Tolerability, and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis. ISA 1 Full Title: 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
- 3
- 试验地点
- 2
- 主要终点
- MP - 1. Incidence, severity, and relatedness of AEs and SAEs
研究概览
简要总结
MP - To determine the safety and tolerability of multiple regimens (monotherapy or add-on therapy to backbone therapy) in MG subtypes.
ISA 1 - To determine the safety and tolerability of empasiprubart as add-on therapy to efgartigimod.
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Part C
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •MP Main inclusion criteria: o Is at least 18 years of age and the local legal age of consent for clinical studies o Has been diagnosed with MG consistent with MG's clinical features and with symptom severity appropriate for currently enrolling ISAs, per the investigator’s clinical judgment o If receiving MG therapy, including nonsteroidal immunosuppressive drugs (NSIDs), corticosteroids, or acetylcholinesterase (AChE) inhibitors either in combination or alone, the participant should receive a stable dosage before master protocol screening
- •ISA 1 Main inclusion criteria:- 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
- •MP Main exclusion criteria: - Known autoimmune disease or any medical condition other than the indication under study that would interfere with an accurate assessment of clinical symptoms of MG or puts the participant at undue risk. - Is MGFA (Myasthenia Gravis Foundation of America) Class V
- •ISA 1 Main exclusion criteria: - 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).
结局指标
主要结局
MP - 1. Incidence, severity, and relatedness of AEs and SAEs
MP - 1. Incidence, severity, and relatedness of AEs and SAEs
MP - 2. Clinically meaningful changes in vital signs, ECGs, and laboratory parameters
MP - 2. Clinically meaningful changes in vital signs, ECGs, and laboratory parameters
ISA 1 - 1. Incidence, severity, and relatedness of adverse events and serious adverse events in parts A and B
ISA 1 - 1. Incidence, severity, and relatedness of adverse events and serious adverse events in parts A and B
ISA 1 - 2. Clinically meaningful changes in vital signs, electrocardio grams, and laboratory parameters in parts A and B
ISA 1 - 2. Clinically meaningful changes in vital signs, electrocardio grams, and laboratory parameters in parts A and B
次要结局
- MP - 1. Change in MG disease severity as measured by change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score or Myasthenia Gravis Impairment Index (MGII) total score
- MP - 2. Proportion of participants reaching minimal symptom expression (MSE) and other percentage improvements at variable time points.
- ISA1 - 1. Myasthenia Gravis Activities of Daily Living (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
- ISA 1 - 2. Proportion of participants reaching minimal symptom expression (MSE) at any point in part B cycles 1 and 2, and part B cycles 1 or 2
- ISA 1 - 3. MG-ADL total score actual values and changes from baseline over time in part B compared with part A
- ISA 1 - 4. Quantitative Myasthenia Gravis (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
- ISA 1 - 5. QMG total score actual values and changes from baseline over time in part B compared with part A
- ISA 1 - 6. Proportion of participants who have a 50% MG-ADL total score improvement in part B cycles 1 and 2
- ISA 1 - 7. Proportion of participants who have positive patient acceptable symptom state (PASS) in part B cycles 1 and 2, and part B cycles 1 or 2
研究者
Chief Scientific Officer
Scientific
Argenx
研究点 (2)
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