NCT06764160已完成3 期
An Open-label, Single-arm, Multi-center Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Participants With Refractory Generalized Myasthenia Gravis (gMG)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 6
- 主要终点
- To assess the efficacy of eculizumab in the treatment of refractory gMG based on the improvement in the MG-ADL
研究概览
简要总结
This is an open-label, single-arm, multi-center study to evaluate the efficacy, safety, PK, PD, and immunogenicity of eculizumab in Chinese participants with refractory gMG. Approximately 15 participants will be enrolled in the study.
详细描述
This post-approval study is an open-label, single-arm, multi-center study to evaluate the efficacy, safety, PK, PD, and immunogenicity of eculizumab in Chinese participants with refractory gMG. There will be 3 periods in this study: Screening Period (up to 4 weeks), Treatment Period (26 weeks, including an Induction Phase and a Maintenance Phase), and Safety Follow-up Period (8 weeks). The overall study duration for an individual participant is estimated to be up to 38 weeks. Approximately 15 participants will be enrolled in the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Diagnosis of MG must be made by the following tests:
- •Positive serologic test for anti-AChR Abs as confirmed at Screening, and
- •One of the following:
- •Abnormal neuromuscular transmission demonstrated by repetitive nerve stimulation, or
- •History of positive anticholinesterase test, eg, neostigmine test, or
- •Participant has demonstrated improvement in MG signs on oral cholinesterase inhibitors as assessed by the treating physician
- •MGFA Clinical Classification Class II to IV at Screening
- •MG-ADL total score must be ≥ 6 at Screening and Day 1
- •Participants who have:
- •Failed treatment with 2 or more ISTs over one year (either in combination or as monotherapy), ie, continue to have impairment ADLs (persistent weakness, experience crisis, or unable to tolerate IST) despite ISTs or,
- •Failed at least one IST and require chronic PE or IVIg to control symptoms, ie, participants who require PE or IVIg on a regular basis for the management of muscle weakness at least 2 cycles over last 12 months
排除标准
- •Any untreated thymic malignancy, carcinoma, or thymoma
- •History of thymectomy or any other thymic surgery within 6 months prior to Screening. Participants with a history of treated thymic malignancy or carcinoma are eligible if they meet all of the following conditions:
- •Treatment completed > 5 years prior to the Screening Visit
- •No recurrence within the 5 years prior to the Screening Visit
- •No radiological indication of recurrence in a computed tomography (CT) or magnetic resonance imaging (MRI) scan, including administration of intravenous (IV) contrast, performed within 6 months of first dose on Day 1
- •Weakness only affecting ocular or peri-ocular muscles (MGFA Class I)
- •MG crisis at Screening (MGFA Class V). However, such participants may be rescreened with Alexion approval once they are treated and medically stable, in the opinion of the Investigator
- •History of N meningitidis infection or unresolved meningococcal disease
研究组 & 干预措施
Eculizumab
Experimental
Eculizumab
干预措施: Eculizumab (Drug)
结局指标
主要结局
To assess the efficacy of eculizumab in the treatment of refractory gMG based on the improvement in the MG-ADL
时间窗: from baseline at Week 26
The mean change of Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) total score from baseline at Week 26 along with 2-sided 95% CI
次要结局
- To assess the efficacy of eculizumab in the treatment of refractory gMG by QMG(from baseline at Week 26)
- To assess the efficacy of eculizumab in the treatment of refractory gMG by MGC(from baseline at Week 26)
- To assess the efficacy of eculizumab in the treatment of refractory gMG by MG-QoL 15r(from baseline at Week 26)
- To assess the efficacy of eculizumab in the treatment of refractory gMG by MG-ADL responder status(from baseline at Week 26)
- To assess the efficacy of eculizumab in the treatment of refractory gMG by QMG responder analysis(from baseline at Week 26)
- To assess the safety and tolerability of eculizumab in the treatment of refractory gMG(from baseline at Week 26)
- To characterize the PK of eculizumab in participants with refractory gMG(from baseline at Week 26)
- To characterize the PD of eculizumab in participants with refractory gMG(from baseline at Week 26)
- To characterize the immunogenicity of eculizumab in participants with refractory gMG(from baseline at Week 26)
研究者
研究点 (6)
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