2022-502072-23-00招募中3 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
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 4
- 试验地点
- 3
- 主要终点
- • Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29) • Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29) • Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)
研究概览
简要总结
Assess the pharmacokinetics (PK) of zilucoplan (ZLP) administered by subcutaneous (sc) injection once daily for pediatric study participants from 2 to <18 years of age with generalized Myasthenia Gravis (gMG). Assess the pharmacodynamics (PD) of ZLP administered by sc injection once daily in pediatric study participants from 2 to <18 years of age with 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
- •Participant must be 2 to <18 years of age at the time of signing the Informed consent/assent according to local regulation.
- •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.
- •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
- •Participant has a prior history of meningococcal disease.
- •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
-
Auxiliary
Participants receiving -
干预措施: - (Drug)
结局指标
主要结局
• Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29) • Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29) • Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)
• Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29) • Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29) • Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)
次要结局
- • Occurrence of treatment-emergent adverse events (TEAEs) during the course of the study
- • Occurrence of treatment-emergent serious adverse events (TESAE)s
- • Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)
- • Occurrence of treatment-emergent infections
- • Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29).
- • Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29).
- • Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29).
- • Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA PIS) at Week 4 (Day 29).
- • Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29).
研究者
UCB Cares
Scientific
UCB Biopharma
研究点 (3)
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