NCT05644561进行中（未招募）3 期
Phase 3, Open-label, Single-arm, Multicenter Study Evaluating Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants (6 to <18 Years) With Generalized Myasthenia Gravis (gMG)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
- 试验地点
- 12
- 主要终点
- Serum Concentration of Ravulizumab
研究概览
简要总结
The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with 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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of gMG confirmed by a positive serologic test for anti-AChR antibodies (Abs) obtained at Screening and/or during Screening Period
- •Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to Class IV at Screening
- •Participants receiving treatment must be on a stable dosing regimen of adequate duration prior to Screening and during the Screening Period.
- •Eculizumab-experienced participants must have been enrolled and treated with eculizumab in Study ECU-MG-303 for at least 6 months (180 days) and must have been on a stable dose for ≥ 2 months (60 days) prior to Screening.
- •All participants must be vaccinated against meningococcal infection
排除标准
- •Medical Conditions
- •Any untreated thymic malignancy, carcinoma, or thymoma.
- •Participants with a history of treated benign thymoma
- •History of thymectomy, thymomectomy, or any thymic surgery within the 12 months prior to Screening
- •History of N meningitidis infection
- •Known to be human immunodeficiency virus (HIV) positive
- •History of unexplained infections
- •Known or suspected history of drug or alcohol abuse or dependence within 1 year prior to the start of the Screening Period
研究组 & 干预措施
Ravulizumab Intravenous (IV) Infusion
Experimental
All participants will receive a weight-based loading dose of ravulizumab IV on Day 1, followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for participants weighing ≥ 20 kg, or once every 4 weeks (q4w) for participants weighing < 20 kg, for a total of 122 weeks of treatment.
干预措施: Ravulizumab (Drug)
结局指标
主要结局
Serum Concentration of Ravulizumab
时间窗: Day 1 predose through Week 18 predose
Serum Free C5 Concentration
时间窗: Day 1 predose through Week 18 predose
次要结局
- Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18(Baseline, Up to Week 18)
- Number of Participants With ≥3 point Reduction Compared to Baseline in the MG-ADL Total Score Over Time Through Week 18(Baseline through Week 18)
- Number of Participants With Anti-Drug Antibody (ADA) at Week 18(Baseline through Week 18)
- Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events(Baseline up to Week 126 (8 weeks after last dose of study drug))
- Change From Baseline in Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score at Up to Week 18(Baseline, Up to Week 18)
- Change in Status from Week 10 in Myasthenia Gravis Foundation of America Postintervention Status (MGFA-PIS) as Assessed by the Investigator or Neurologist at Up to Week 18(Week 10, Up to Week 18)
- Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline(Baseline through Week 18)
- Number of Participants With ≥5-point Reduction Compared to Baseline in the QMG Total Score Over Time Through Week 18(Baseline through Week 18)
- Number of Participants That Improve or Remain Stable in QMG Total Score at Week 18 Compared to Baseline(Baseline through Week 18)
- Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at Up to Week 18(Baseline, Up to Week 18)
- Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18(Baseline, Up to Week 18)
- Change From Baseline in Myasthenia Gravis Composite (MGC) Score at Up to Week 18(Baseline, Up to Week 18)
研究者
研究点 (12)
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