An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Activity of Zilucoplan in Pediatric Study Participants With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis
概览
- 阶段
- 3 期
- 干预措施
- Zilucoplan
- 疾病 / 适应症
- Generalized Myasthenia Gravis
- 发起方
- UCB Biopharma SRL
- 入组人数
- 8
- 试验地点
- 18
- 主要终点
- Occurence of treatment-emergent infections
- 状态
- Enrolling By Invitation
- 最后更新
- 8天前
概览
简要总结
The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants
研究者
入排标准
入选标准
- •United States of America (USA) specific inclusion criterion:
- •\- Participant must be ≥ 12 years of age at the time of signing the Informed Consent/Assent according to local regulation.
- •Rest of World (ROW) specific inclusion criterion:
- •\- Participant must be ≥ 2 years of age at the time of signing the Informed Consent/Assent according to local regulation.
- •Global specific inclusion criteria:
- •Participant has completed the MG0014 according to the protocol, and further treatment with zilucoplan is in the interest of the participant in the investigator´s opinion
- •Participant agrees to receive booster vaccinations against meningococcal infections during the study, if clinically indicated according to the local standard of care
排除标准
- •Study participant met any mandatory investigational medicinal product (IMP) withdrawal or mandatory permanent discontinuation criteria in MG0014 or permanently discontinued IMP
- •Participant has known positive serology for muscle-specific kinase
- •Participant has known hypersensitivity to any components of the IMP
- •Participant has a prior history of meningococcal disease
研究组 & 干预措施
Zilucoplan Arm
Study participants will receive Zilucoplan at a pre-defined dose based on their weight.
干预措施: Zilucoplan
结局指标
主要结局
Occurence of treatment-emergent infections
时间窗: Baseline (Day 1) to Safety Follow-up (up to Week 60)
Percentage of participants who experienced treatment-emergent infections as adverse events. 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.
Occurence of treatment emergent adverse events during the course of the study
时间窗: Baseline (Day 1) to Safety Follow-up (up to Week 60)
An adverse event (AE) is any untoward medical occurence in a patient or clinical investigation where the study participant 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.
Occurence of treatment-emergent serious adverse events (TESAEs)
时间窗: Baseline (Day 1) to Safety Follow-up (up to Week 60)
A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: Results in death Is life-threatening Requires inpatient hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical event.
Occurence of treatment-emergent advserse events leading to permanent withdrawal of investigational medicinal product
时间窗: Baseline (Day 1) to Safety Follow-up (up to Week 60)
An adverse event (AE) is any untoward medical occurence in a participant or clinical investigation administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.
次要结局
- Sheep red blood cell (sRBC) lysis activity at Week 52(Week 52)
- Plasma concentration of Zilucoplan at Week 52(Week 52)
- Blood complement component 5 (C5) levels at Week 52(Week 52)
- Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52(Week 52)
- Quantitative Myasthenia Gravis (QMG) score at Week 52(Week 52)