NCT04963270终止3 期
A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study To Evaluate Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Patients With Generalized Myasthenia Gravis
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 188
- 试验地点
- 76
- 主要终点
- DB Period: Mean Change From Baseline in Total Myasthenia Gravis Activities of Daily Living (MG-ADL) Score in the AChR+ Population
研究概览
简要总结
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab compared with placebo in participants with generalized myasthenia gravis (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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed Informed Consent Form
- •For adolescent patients: Informed Consent Form for study participation signed by the parents or a legal guardian, and patient assent obtained, as per local requirements
- •Ability to comply with the study protocol procedures
- •Confirmed diagnosis of gMG (anti-AChR, anti-MuSK or anti-LRP4 present at screening)
- •A total MG-ADL score of ≥ 5 points at screening with more than 50% of this score attributed to non-ocular items
- •MGFA severity Class II-IV
- •Ongoing gMG treatment at a stable dose
- •For female patients of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use adequate contraception during the treatment period and for at least 3 months after the final dose of satralizumab.
排除标准
- •History of thymectomy within 12 months prior to screening
- •Ocular MG (MGFA Class I) and myasthenic crisis (MGFA Class V) within the last 3 months prior to screening
- •Known disease other than gMG that would interfere with the course and conduct of the study
- •Positive screening tests for hepatitis B virus (HBV) and hepatitis C virus (HCV)
- •Evidence of latent or active tuberculosis (excluding patients receiving chemoprophylaxis for latent tuberculosis infection)
- •Receipt of live or live attenuated vaccine within 6 weeks prior to baseline
- •Pregnant or breastfeeding, or intending to become pregnant during the study or within 3 months after the last dose
研究组 & 干预措施
Satralizumab
Experimental
Participants will receive Satralizumab at Weeks 0, 2, 4, and Q4W thereafter. Adolescent patients who first enter the study in the OLE period will receive satralizumab SC loading doses at Week 0, 2, and 4 in the OLE, followed by maintenance doses Q4W thereafter and will remain on stable background therapy until Week 24 of the OLE.
干预措施: Satralizumab (Drug)
Placebo
Placebo Comparator
Participants will receive placebo at Weeks 0, 2, 4, and Q4W thereafter
干预措施: Placebo (Other)
结局指标
主要结局
DB Period: Mean Change From Baseline in Total Myasthenia Gravis Activities of Daily Living (MG-ADL) Score in the AChR+ Population
时间窗: At Week 24
The MG-ADL scale was used to assess the degree of gMG symptoms (six items: diplopia, ptosis, difficulties with chewing, swallowing, talking, and respiratory problems) and functional limitations in carrying out activities of daily living (two items: ability to brush teeth or comb hair and impairment in the ability to arise from a chair) that are present and clinically relevant in gMG participants. Each of the eight items was ranked on a 0-3 scale, with 3 representing the most severe symptoms or impaired performance and 0 representing no symptoms or impaired performance. The total MG-ADL score was calculated as the sum of each item score, with a maximum score ranging from 0 (least severe symptoms/impairment) to 24 (most severe symptoms/impairment). Higher scores indicate greater disease severity.
次要结局
- DB Period: Mean Change From Baseline in QMG Score in OP at Week 24(At Week 24)
- DB Period: Mean Change From Baseline in Total MG-ADL Score in the Overall Population (OP) at Week 24(At Week 24)
- DB Period: Percentage of Participants With a ≥ 2-point Reduction From Baseline in Total MG-ADL Score in AChR+ Population at Week 24(At Week 24)
- DB Period: Percentage of Participants With a ≥ 2-point Reduction From Baseline in Total MG-ADL Score in OP at Week 24(At Week 24)
- DB Period: Mean Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score in AChR+ Population at Week 24(At Week 24)
- DB Period: Mean Change From Baseline in Myasthenia Gravis Quality of Life 15 Scale (Revised) (MG-QOL 15r) Total Score in AChR+ Population at Week 24(At Week 24)
- DB Period: Mean Change From Baseline in MG-QOL 15r Total Score in OP at Week 24(At Week 24)
- DB Period: Mean Change From Baseline in Quality of Life in Neurological Disorders (Neuro-QoL) Fatigue Subscale Total Score in AChR+ Population at Week 24(At Week 24)
- DB Period: Mean Change From Baseline in Neuro-QoL Fatigue Subscale Total Score in OP at Week 24(At Week 24)
- DB Period: Mean Change From Baseline in Total Myasthenia Gravis Composite (MGC) Score in AChR+ Population at Week 24(At Week 24)
- DB Period: Percentage of Participants With a ≥ 3-point Reduction From Baseline in QMG Score in AChR+ Population at Week 24(At Week 24)
- DB Period: Percentage of Participants With a ≥ 3-point Reduction From Baseline in QMG Score in OP at Week 24(At Week 24)
- DB Period: Percentage of Participants With a ≥ 3-point Reduction From Baseline in Total MGC Score in AChR+ Population at Week 24(At Week 24)
- DB Period: Mean Change From Baseline in Total MGC Score in OP at Week 24(At Week 24)
- DB Period: Percentage of Participants Who Achieved Minimal Symptom Expression (Total MG-ADL Score of 0 or 1) in OP at Week 24(At Week 24)
- DB Period: Percentage of Participants With at Least One gMG-Related Exacerbation Between Baseline and Week 24 in OP(Baseline up to Week 24)
- DB Period: Duration of Meaningful Improvement, Defined as ≥ 2-Point Reduction From Baseline in Total MG-ADL Score in OP(Baseline, Week 24)
- DB Period: Number of Participants With Adverse Events (AEs)(Day 1 up to approximately 24 weeks)
- DB Period: Percentage of Participants With a ≥ 3-point Reduction From Baseline in Total MGC Score in OP at Week 24(At Week 24)
- DB Period: Percentage of Participants in OP Receiving Rescue Therapy Between Baseline and Week 24(Baseline up to Week 24)
- DB Period: Percentage of Participants Who Achieved Minimal Symptom Expression (Total MG-ADL Score of 0 or 1) in AChR+ Population at Week 24(At Week 24)
- DB Period: Percentage of Participants With at Least One gMG-Related Exacerbation Between Baseline and Week 24 in AChR+ Population(Baseline up to Week 24)
- DB Period: Percentage of Participants in AChR+ Population Receiving Rescue Therapy Between Baseline and Week 24(Baseline up to Week 24)
- Number of Participants With Anti-drug Antibodies (ADAs) to Satralizumab(Baseline to Week 24)
- DB Period: Duration of Meaningful Improvement, Defined as ≥ 2-Point Reduction From Baseline in Total MG-ADL Score in AChR+ Population(Baseline, Week 24)
- DB Period: Serum Levels of Soluble Interleukin-6 Receptors (sIL-6R)(Baseline, Weeks 2, 4, 8, 12, 16, 20 and 24)
- DB Period: Serum Levels of Interleukin-6 (IL-6)(Baseline, Weeks 2, 4, 8, 12, 16, 20 and 24)
- Serum Concentrations of Satralizumab(Weeks 0, 2, 4, 8, 12, 16, 20 and 24)
研究者
研究点 (76)
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