A Phase 3, Multi-center, Randomized, Quadruple-blind, Placebo-controlled Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia Gravis (gMG)
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 233
- 试验地点
- 93
- 主要终点
- Change from Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score in acetylcholine receptor (AChR) Ab seropositive (AChRAb+) participants
研究概览
简要总结
The purpose of this 4-period study is to confirm the efficacy and safety of batoclimab in participants with gMG. In Period 1, participants will be randomized 1:1:1 to receive batoclimab 680 milligrams (mg) subcutaneously (SC) once a week (QW) or 340 mg SC QW or placebo. The primary efficacy endpoint will be assessed by change in the myasthenia gravis activities of daily living (MG- ADL) score in acetylcholine receptor antibody seropositive (AChRAb+) participants. In Period 2, participants previously treated with batoclimab will be re-randomized to stay on batoclimab (340 mg SC QW or 340 mg SC every two weeks) or receive placebo treatment. The secondary endpoint of maintenance of efficacy will be assessed by change in the MG- ADL score in AChRAb+ participants. Participants demonstrating a response to batoclimab during either Period 1 or 2 may enter the long-term extension (Period 3). Participants who complete Period 3 are eligible to participate in Period 4 (Optional Long-Term extension) according to their treatment assignment in Period 3.
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
- Triple (Participant, Investigator, Outcomes Assessor)
盲法说明
Sponsor Staff is also masked, so the study is Quadruple-blind.
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
- •Are ≥ 18 years of age at the Screening Visit.
- •Have mild to severe gMG by Myasthenia Gravis Foundation of America (MGFA) classification Class II, III, or IVa at the Screening Visit.
- •Have a QMG score ≥ 11 at the Screening and Baseline Visits.
- •Have a MG-ADL score of ≥ 5 at the Screening and Baseline Visits.
- •Additional inclusion criteria are defined in the protocol.
排除标准
- •Have experienced myasthenic crisis within 3 months of the Screening Visit.
- •Have had a thymectomy performed < 6 months prior to the Screening Visit or have a planned thymectomy during the study period.
- •Have any active or untreated malignant thymoma.
- •Have received any agent or therapy (exclusive of those identified within inclusion criteria) with immunosuppressive properties (e.g., stem cell therapy, chemotherapies) within the past year.
- •Have used anti-FcRn treatment within 3 months prior to the Screening Visit or have a documented history of non-response to prior anti-FcRn treatment.
- •Additional exclusion criteria are defined in the protocol.
研究组 & 干预措施
Batoclimab Maintenance Dose 2 (Period 2)
干预措施: Batoclimab 340 mg SC bi-weekly (Drug)
Batoclimab Induction Dose 2 (Period 1)
干预措施: Batoclimab 340 mg SC weekly (Drug)
Batoclimab Induction Dose 1 (Period 1)
干预措施: Batoclimab 680 mg SC weekly (Drug)
Placebo Induction Dose (Period 1)
干预措施: Matching Placebo SC (Drug)
Batoclimab Maintenance Dose 1 (Period 2)
干预措施: Batoclimab 340 mg SC weekly (Drug)
Placebo Maintenance Dose (Period 2)
干预措施: Matching Placebo SC (Drug)
结局指标
主要结局
Change from Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score in acetylcholine receptor (AChR) Ab seropositive (AChRAb+) participants
时间窗: Baseline (Day 1) to Week 12
MG-ADL is an 8-item, participant-reported questionnaire that assesses gMG symptoms and their effects on activities of daily living. Each item is assessed on a 4-point scale where a score of 0 represents normal function and a score of 3 represents loss of ability to perform that function. Total score ranges from 0 to 24, with higher scores indicating greater functional impairment and disability.
次要结局
- Percentage of AChRAb+ participants achieving MG-ADL score of 0 or 1 by Week 12(Up to Week 12)
- Percentage of participants with clinical laboratory-related TEAEs or treatment emergent laboratory abnormalities.(Up to 76 Weeks)
- Change from Baseline in Quantitative Myasthenia Gravis (QMG) score in AChRAb+ participants(Baseline (Day 1) to Week 12)
- Change from Baseline in MG-ADL score for AChRAb+ randomized withdrawal participants(Baseline (Week 12) to Week 24)
- Percentage of AChRAb+ participants with greater than equal to (>=) 3-point improvement in QMG score(Up to Week 12)
- Change from Baseline in MG-ADL score in AChRAB- (AChRAB negative) participants(Baseline (Day 1) to Week 12)
- Percentage of Participants With Clinically Significant Changes in Vital Sign Measurements(Up to 76 Weeks)
- Number of Participants with Clinically Significant Changes in Laboratory Results(Up to 76 Weeks)
- Percentage of Participants with Treatment Emergent Adverse Events (TEAEs)(Up to 76 Weeks)