An Open-label Extension Study to Evaluate Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 入组人数
- 12
- 试验地点
- 17
- 主要终点
- Occurrence of TEAEs leading to permanent withdrawal of IMP up to the EOS Visit
研究概览
简要总结
The purpose of the study is to assess the long term safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in pediatric participants with generalized Myasthenia Gravis (gMG) aged ≥2 at the time of the Screening Visit of MG0006.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Study participant must meet one of the following:
- •Study participant completed MG0006 according to the protocol
- •Study participant completed the MG0006 Treatment Period and has a worsening of generalized myasthenia gravis (gMG) symptoms in the Observation Period of MG0006
排除标准
- •Study participant met any mandatory withdrawal or mandatory permanent investigational medicinal product (IMP) discontinuation criteria in MG0006 or permanently discontinued IMP
- •Study participant has a known hypersensitivity to any components of the IMP or other neonatal Fc receptor (FcRn) drugs
- •Study participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at Baseline, and could jeopardize or compromise the study participant's ability to participate in this study
研究组 & 干预措施
rozanolixizumab
Study participants will receive pre-defined doses of rozanolixizumab. Each treatment cycle in each Treatment Period (TP) consists of 6 subcutaneous (sc) administrations of rozanolixizumab at 1-week intervals. Each Treatment Period will be initiated upon the discretion of the Investigator based on the medical needs of the study-participant.
干预措施: rozanolixizumab (Drug)
结局指标
主要结局
Occurrence of TEAEs leading to permanent withdrawal of IMP up to the EOS Visit
时间窗: From Baseline up to the EOS Visit (up to 52 weeks)
An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit
时间窗: From Baseline up to the EOS Visit (up to 52 weeks)
AESMs are: Severe and/or serious headache, suspected aseptic meningitis, severe Gastrointestinal (GI) disorders, and opportunistic infection.
Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit
时间窗: From Baseline up to the EOS Visit (up to 52 weeks)
Serious TEAEs are any untoward medical incidence in a subject during administered study treatment, whether or not these events are related to study treatment and additionally are emergent untoward medical occurrence that at any dose: * Results in death * Is life-threatening * Requires in patient hospitalisation or prolongation of existing hospitalisation * Results in persistent disability/incapacity * Is a congenital anomaly or birth defect * Important medical events
次要结局
- Percent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP)(From Baseline to the end of Week 6 of each TP (up to 52 weeks))
- Absolute change in total IgG from Baseline to the end of Week 6 of each TP(From Baseline to the end of Week 6 of each TP (up to 52 weeks))
- Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG ADL) total score at the end of Week 6 of each TP(From Baseline to the end of Week 6 of each TP (up to 52 weeks))
- Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 of each TP(From Baseline to the end of Week 6 of each TP (up to 52 weeks))