2022-502074-16-00招募中3 期
An open-label, single arm study evaluating the activity, safety, and pharmacokinetics of rozanolixizumab in pediatric study participants with moderate to severe generalized myasthenia gravis
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 7
- 试验地点
- 5
- 主要终点
- - Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit (up to 18 weeks) - Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) up to the EOS Visit (up to 18 weeks) - Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit (up to 18 weeks)
研究概览
简要总结
Assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥2 to <18 years with generalized Myasthenia Gravis (gMG)
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Study participant must be ≥2 to <18 years of age inclusive, at the time of signing the informed consent/assent according to local regulation - Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG) at Screening that includes a record confirming the presence of MG specific autoantibodies to acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) prior to Screening - Study participant has Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IVa at Screening - Study participant has received existing conventional treatment(s) for gMG (eg, pyridostigmine, corticosteroids, and/or immune suppressants) prior to Screening - Study participant has had an unsatisfactory clinical response or worsening of gMG symptoms and is in need of additional therapy (for example, plasma exchange (PEX) or treatment with intravenous immunoglobulin g (IVIg))
排除标准
- •Study participant with severe weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis at Screening or Baseline - Study participant has a known hypersensitivity to any components of the Investigational Medicinal Product (IMP) or other anti-FcRn medications - Study participant with any active or untreated thymoma - Study participant has a history of thymectomy within 6 months prior to Screening - Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of IMP - Study participant has received a live vaccination within 4 weeks prior to Baseline or intends to have a live vaccination during the course of the study
研究组 & 干预措施
-
Auxiliary
Participants receiving -
干预措施: - (Drug)
结局指标
主要结局
- Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit (up to 18 weeks) - Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) up to the EOS Visit (up to 18 weeks) - Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit (up to 18 weeks)
- Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit (up to 18 weeks) - Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) up to the EOS Visit (up to 18 weeks) - Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit (up to 18 weeks)
次要结局
- Percent change in total Immunoglobulin G (IgG) from Baseline at the end of Week 6
- Absolute change in total IgG from Baseline at the end of Week 6
- Percent change from Baseline in MG-specific autoantibodies (anti-AChR or anti-MuSK) levels at the end of Week 6
- Absolute change from Baseline in MG-specific autoantibodies (anti-AChR or anti-MuSK) levels at the end of Week 6
- Change from Baseline in myasthenia gravis-activities of daily living (MG-ADL) total score at the end of Week 6
- Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6
- Occurrence of other TEAEs (including headache, nausea, and infusion site reactions) during Treatment Period 1 (TP1) and Observation Period 1(OP1)
- Evaluation of local tolerability at each scheduled assessment during TP1
- Plasma concentration of rozanolixizumab at the 6-week treatment cycle
- Incidence of antidrug antibodies (ADAs) at the end of Week 6
研究者
UCB Cares
Scientific
UCB Biopharma
研究点 (5)
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