NCT06298552进行中（未招募）3 期
A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod IV in Adult Participants With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- argenx
- 入组人数
- 119
- 试验地点
- 180
- 主要终点
- MG-ADL Total Score Change From Baseline
研究概览
简要总结
The primary purpose of this study is to measure the efficacy and safety of efgartigimod IV compared to placebo in participants with Acetylcholine Receptor Binding Antibody (AChR-Ab) seronegative Generalized Myasthenia Gravis (gMG). The study consists of a Part A where participants will be randomized to receive either efgartigimod IV or placebo IV and a Part B where participants completing part A will receive open-label efgartigimod IV. Participants will be in the study for up to (approximately) 3.5 years.
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
- Double (Participant, Investigator)
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
- •The participant is at least 18 years of age and the local legal age of consent for clinical studies when signing the ICF.
- •The participant is capable of providing signed informed consent and following with protocol requirements.
- •The participant agrees to use contraceptive measures consistent with local regulations and the women of child-bearing potential (WOCBP) must have a negative serum pregnancy test result at screening and a negative urine pregnancy test result at baseline before receiving the study drug.
- •The participant has no known weakness in infancy and later develop fatigable weakness after aged 16 years and diagnosed with acquired gMG of both of the following:
- •History of abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation (RNS) or is for anti-muscle-specific kinase antibodies (MuSK-Ab) seropositive
- •Either a history of positive edrophonium chloride test OR a demonstrated improvement in MG signs with treatments such as oral acetylcholinesterase (AChE) inhibitors, plasma exchange (PLEX), immunoabsorption, or intravenous immunoglobulin (IVIg)/ subcutaneous immunoglobulin (SCIg) treatment
- •The participant is receiving a stable dose of MG therapy before screening, including acetylcholinesterase (AChE) inhibitors, steroids, or nonsteroidal immunosuppressive therapies (NSISTs) in combination or alone
排除标准
- •Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of gMG or puts the participant at undue risk
- •History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer
- •Clinically significant active infection that is not sufficiently resolved in the investigator's opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV
- •History of or current alcohol, drug, or medication abuse as assessed by the investigator
- •Pregnant or lactating state or intention to become pregnant during the study
- •Live or live-attenuated vaccine received <4 weeks before screening
- •Worsening muscle weakness secondary to concurrent infections or medications
- •Received a thymectomy less than 3 months before screening or thymectomy is planned during the study
- •The complete list of exclusion criteria can be found in the protocol.
研究组 & 干预措施
Efgartigimod IV
Experimental
Patients receiving efgartigimod IV in both part A and part B
干预措施: Efgartigimod IV (Biological)
Placebo
Placebo Comparator
Patients receiving placebo during part A and receiving efgartigimod IV during part B
干预措施: Efgartigimod IV (Biological)
Placebo
Placebo Comparator
Patients receiving placebo during part A and receiving efgartigimod IV during part B
干预措施: Placebo IV (Other)
结局指标
主要结局
MG-ADL Total Score Change From Baseline
时间窗: Up to 29 days in part A
The Myasthenia Gravis Activities of Daily Living (MG-ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (no symptoms) to 24 (most severe symptoms).
MG-ADL total score change from baseline
时间窗: Up to 29 days (part A)
The Myasthenia Gravis Activities of Daily Living (MG- ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (normal symptoms) to 24 (most severe symptoms)
次要结局
- QMG Total Score Change From Baseline(Up to 29 days in part A)
- Proportion of Participants Who Are Both MG-ADL and QMG Responders in Part A(Up to 8 weeks (part A))
- Proportion of Participants With MSE(Up to 8 weeks (part A) + 3 years (part B))
- Proportion of Participants Who Are MG-ADL Responders in Part A(Up to 8 weeks (part A))
- Proportion of Participants Who Are QMG Responders in Part A(Up to 8 weeks (part A))
- Proportion of Participants Who Are Early MG-ADL Responders in Part A(Up to 8 weeks (part A))
- MG-ADL Total Score Change From Baseline Over Time in Part A and Part A+B(Up to 8 weeks (part A) + 3 years (part B))
- MG-QoL15r Change From Baseline Over Time in Part A and Part A+B(Up to 8 weeks (part A) + 3 years (part B))
- EQ-5D-5L VAS Change From Baseline Over Time in Part A and Part A+B(Up to 8 weeks (part A) + 3 years (part B))
- Total IgG Concentrations Percent Changes From Baseline Over Time in Part A and Part A+B(Up to 8 weeks (part A) + 3 years (part B))
- QMG total score change from baseline(Up to 8 weeks (part A) + 2 years (part B))
- Proportion of participants who are both MG-ADL and QMG responders in part A(Up to 8 weeks (part A))
- Proportion of participants with MSE(Up to 8 weeks (part A) + 2 years (part B))
- Proportion of participants who are MG-ADL responders in part A(Up to 8 weeks (part A))
- Proportion of participants who are QMG responders in part A(Up to 8 weeks (part A))
- Proportion of participants who are early MG-ADL responders in part A(Up to 8 weeks (part A))
- MG-ADL total score change from baseline over time in part A and part A+B(Up to 8 weeks (part A) + 2 years (part B))
- MG-QoL15r change from baseline over time in part A and part A+B(Up to 8 weeks (part A) + 2 years (part B))
- EQ-5D-5L VAS change from baseline over time in part A and part A+B(Up to 8 weeks (part A) + 2 years (part B))
- Incidence of AEs and SAEs in part A and part A+B(Up to 8 weeks (part A) + 2 years (part B))
- Total IgG concentrations percent changes from baseline over time in part A and part A+B(Up to 8 weeks (part A) + 2 years (part B))
研究者
研究点 (180)
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