NCT06055959招募中2 期
A Multicenter Open-Label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, Tolerability, and Activity of Zilucoplan in Pediatric Study Participants From 2 to Less Than 18 Years of Age With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 8
- 试验地点
- 17
- 主要终点
- Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
研究概览
简要总结
The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).
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
- 12 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •United States of America (USA) specific inclusion criterion:
- •- Participant must be 12 to <18 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 to <18 years of age at the time of signing the Informed consent/assent according to local regulation
- •Global inclusion criteria:
- •Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by a prior positive serologic test result to acetylcholine receptor (AChR) prior to Screening
- •Participant meets the criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening
- •Participants with gMG, including:
- •An MG-activities of daily living (MG-ADL) total score of 6 or more in adolescents from 12 years to <18 years of age at Screening
- •Documented weakness in at least 1 limb, neck, or bulbar muscle in children from 2 years to <12 years of age at Screening (does not apply to US)
- •Documented vaccination against meningococcal infections within 3 years prior to study start. If not fully vaccinated, participants must receive appropriate prophylactic antibiotic treatment until at least 2 weeks after the initial dose of vaccine(s)
排除标准
- •Participant has known positive serology for muscle-specific kinase
- •Participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the participant's ability to participate in this study
- •Participant has had a thymectomy within 6 months prior to Baseline
- •Participant has minimal Manifestation Status of MG based on the clinical judgement of the Investigator
- •Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous antibiotics within 4 weeks prior to Baseline
研究组 & 干预措施
Zilucoplan Arm
Experimental
Study participants will receive zilucoplan in pre-defined dose based on their weight.
干预措施: Zilucoplan (Drug)
结局指标
主要结局
Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
时间窗: Week 4 (Day 29)
Blood samples will be collected for measurement of plasma concentrations of ZLP on Day 29 predose.
Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)
时间窗: Week 4 (Day 29)
Samples for measurement of sRBC lysis will be collected on Day 29 predose.
Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)
时间窗: Week 4 (Day 29)
Samples for measurement of C5 will be collected on Day 29 predose.
次要结局
- Occurence of treatment-emergent adverse events (TEAEs) during the course of the study(From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15))
- Occurrence of treatment-emergent serious adverse events (TESAEs)(From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15))
- Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)(From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15))
- Occurrence of treatment-emergent infections(From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15))
- Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29)(Week 4 (Day 29))
- Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29).(Week 4 (Day 29))
- Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29)(Week 4 (Day 29))
- Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA-PIS) at Week 4 (Day 29)(Week 4 (Day 29))
- Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29)(Week 4 (Day 29))
研究者
研究点 (17)
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