A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 3 Trial to Evaluate the Efficacy, Safety and Tolerability of ARGX-113 in Patients With Myasthenia Gravis Having Generalized Muscle Weakness
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- argenx
- 入组人数
- 167
- 试验地点
- 132
- 主要终点
- Percentage of MG-ADL Responders During Cycle 1 (C1); Analyzed in the AChR-Ab Seropositive Population
研究概览
简要总结
A randomized, double-blind, placebo controlled, multicenter Phase 3 trial to evaluate the efficacy, safety, tolerability, quality of life and impact on normal daily activities of ARGX-113 in patients with gMG.
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
- •Patients with the ability to understand the requirements of the trial, provide written informed consent, and comply with the trial protocol procedures.
- •Male or female patients aged ≥ 18 years.
- •Diagnosis of MG with generalized muscle weakness meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, IVa and IVb.
- •Other, more specific inclusion criteria are defined in the protocol
排除标准
- •Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.
- •Male patients who are sexually active and do not intend to use effective methods of contraception during the trial or within 90 days after the last dosing or male patients who plan to donate sperm during the trial or within 90 days after the last dosing.
- •MGFA Class I and V patients.
- •Patients with worsening muscle weakness secondary to concurrent infections or medications.
- •Patients with known seropositivity or who test positive for an active viral infection at Screening with:
- •Hepatitis B Virus (HBV) (except patients who are seropositive because of HBV vaccination)
- •Hepatitis C Virus (HCV)
- •Human Immunodeficiency Virus (HIV)
- •Other, more specific exclusion criteria are further defined in the protocol.
结局指标
主要结局
Percentage of MG-ADL Responders During Cycle 1 (C1); Analyzed in the AChR-Ab Seropositive Population
时间窗: Baseline up to Day 63 (end of TC1)
The MG-ADL is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The scale comprises 2 items on daily life activities and 6 items on symptoms. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity. A patient was considered an MG-ADL responder during C1 if there was a reduction of ≥2 points on the MG-ADL total score (compared to baseline of C1 \[C1B\]) for ≥4 consecutive weeks with the first reduction occurring no later than 1 week after the last infusion of IMP in C1.
次要结局
- Percentage of Quantitative Myasthenia Gravis (QMG) Responders During C1; Analyzed in the AChR-Ab Seropositive Population(Baseline up to Day 63 (end of TC1))
- Percentage of MG-ADL Responders During C1; Analyzed in the Overall Population(Baseline up to Day 63 (end of TC1))
- Percentage of Time That Patients Had a Clinically Meaningful Improvement (CMI) in MG-ADL Total Score up to and Including Day 126; Analyzed in the AChR-Ab Seropositive Population(Baseline up to Day 126)
- Percentage of Early MG-ADL Responders During C1; Analyzed in the AChR-Ab Seropositive Population(Baseline up to Day 63 (end of TC1))
- Time From Week 4 to Qualify for Retreatment; Analyzed in the AChR-Ab Seropositive Population(Week 4 up to Day 182 (end of study [EoS]))