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
概览
- 阶段
- 2 期
- 干预措施
- rozanolixizumab
- 疾病 / 适应症
- Generalized Myasthenia Gravis
- 发起方
- UCB Biopharma SRL
- 入组人数
- 12
- 试验地点
- 26
- 主要终点
- Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit
- 状态
- 招募中
- 最后更新
- 8天前
概览
简要总结
The purpose of the study is to assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥2 to <18 years with generalized Myasthenia Gravis (gMG).
研究者
入排标准
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 (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-neonatal-Fc receptor (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
rozanolixizumab
Study participants will receive pre-defined doses of rozanolixizumab for 6 weeks.
干预措施: rozanolixizumab
结局指标
主要结局
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 18 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
Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) up to the EOS Visit
时间窗: From Baseline up to the EOS Visit (up to 18 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 (up to 18 weeks)
时间窗: From Baseline up to the EOS Visit (up to 18 weeks)
AESMs are: Severe and/or serious headache, suspected aseptic meningitis, severe Gastrointestinal (GI) disorders, and opportunistic infection.
次要结局
- Percent change in total Immunoglobulin G (IgG) from Baseline at the end of Week 6(From Baseline to the end of Week 6)
- Absolute change in total IgG from Baseline at the end of Week 6(From Baseline to the end of Week 6)
- Percent change from Baseline in myasthenia gravis (MG) autoantibody levels at the end of Week 6(From Baseline to the end of Week 6)
- Absolute change from Baseline in MG-specific autoantibody levels at the end of Week 6(From Baseline to 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(From Baseline to the end of Week 6)
- Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6(From Baseline to the end of Week 6)
- Evaluation of local tolerability at each scheduled assessment during TP1(At each scheduled assessment during TP1 (Baseline, week 2, 3, 4, 5, up to 6 weeks))
- Plasma concentration of rozanolixizumab at the 6-week treatment cycle(At the 6-week treatment cycle)
- Occurrence of other TEAEs (including headache, nausea, and infusion site reactions) during Treatment Period 1 (TP1) and Observation Period 1 (OP1)(During TP1 and OP1 (up to 14 weeks))
- Incidence of antidrug antibodies (ADAs) at the end of Week 6(At the end of Week 6)