NCT06607627招募中3 期
An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+)
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 12
- 试验地点
- 13
- 主要终点
- Maximum Observed Serum Concentration (Cmax) of Gefurulimab
研究概览
简要总结
The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study.
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
- 6 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.
- •All participants must be vaccinated against meningococcal infection from serogroups A, B, C, W, and Y within 3 years and at least 2 weeks prior to the first dose of study intervention administration.
- •Rest of World (ROW) specific inclusion criteria:
- •Participant must be 6 to < 18 years of age at the time of signing the informed consent/assent.
- •All participants must be vaccinated against meningococcal infection from serogroups A, C, W, Y (and B where available) within 3 years prior to study intervention on Day
- •If vaccination occurs < 2 weeks prior to Day 1, the participants will receive prophylactic antibiotics for at least 2 weeks after initial meningococcal vaccination for serogroups A,C,W,Y (and B, where available)
- •Global inclusion criteria:
- •Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV
- •Positive serological test for autoantibodies against AChR
排除标准
- •History of thymectomy, or any other thymic surgery within 12 months prior to Screening
- •Untreated thymic malignancy, carcinoma, or thymoma
- •History of Neisseria meningitidis infection
- •Pregnancy, breastfeeding, or intention to conceive during the course of the study
研究组 & 干预措施
Gefurulimab
Experimental
On Day 1, participants will receive a weight based loading dose followed by a weekly maintenance dose for up to 122 weeks.
干预措施: Gefurulimab (Combination Product)
结局指标
主要结局
Maximum Observed Serum Concentration (Cmax) of Gefurulimab
时间窗: Day 1 predose through Week 18 predose
Serum Free Complement Component 5 (C5) Concentration
时间窗: Day 1 predose through Week 18 predose
次要结局
- Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)(Day 1 through Week 134)
- Change in Quantitative Myasthenia Gravis Score for Disease Severity (QMG) Total Score From Baseline Through Week 18(Baseline through Week 18)
- Number of Participants With ≥ 5-point Reduction From Baseline in the QMG Total Score Through Week 18(Baseline through Week 18)
- Change in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score From Baseline Through Week 18(Baseline through Week 18)
- Number of Participants With ≥ 3-point Reduction From Baseline in the MG-ADL Total Score Through Week 18(Baseline through Week 18)
- Number of Participants With Anti-drug Antibodies (ADAs)(Baseline through Week 134)
- Number of ADA Positive Participants With Pre-existing Immunoreactivity,Treatment-emergent or Treatment-boosted ADA Responses(Baseline through Week 134)
- Number of ADA Positive Participants With Neutralizing Antibody (NAb) Positive or Negative Status(Baseline through Week 134)
研究者
研究点 (13)
Loading locations...
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