NCT05346354进行中（未招募）2 期
A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
- 试验地点
- 17
- 主要终点
- Change From Baseline in the Annualized Relapse Rate at Week 50
研究概览
简要总结
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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
- •Participants must be anti-AQP4 Ab-positive and have a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
- •Complement inhibitor treatment-naïve participants must have had at least 1 attack or relapse in the last 12 months prior to the Screening Period.
- •Expanded Disability Status Scale (EDSS) score ≤
- •Eculizumab-experienced participants must be clinically stable per Investigator for 30 days and have been treated with eculizumab in Study ECU-NMO-303 for at least 90 days prior to screening with no missed doses within 2 months prior to Day
- •Participants who enter the study receiving supportive IST(s) (eg, corticosteroid, azathioprine [AZA], mycophenolate mofetil [MMF], methotrexate [MTX], tacrolimus [TAC], cyclosporin [CsA], or cyclophosphamide [CYC]) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration prior to Screening and remain on a stable dosing regimen during the Screening Period.
- •To reduce the risk of meningococcal infection (Neisseria meningitidis), all participants must be vaccinated against meningococcal infection.
- •Documented vaccination for Hib and S pneumoniae at least 14 days prior to Day 1 according to national/local guidelines for the applicable age group.
排除标准
- •Use of rituximab within 3 months prior to screening.
- •Currently treated with a biologic medications (other than eculizumab) that may affect immune system functioning, or has stopped treatment with a biologic medication that may affect immune system functioning, and 5 half lives of the medication have not elapsed by the time of the Screening Visit.
- •Use of intravenous immunoglobulin (IVIg) or plasma exchange (PE) within 3 weeks prior to Screening.
- •Participation in another investigational drug or investigational device study (other than Study ECU-NMO-303) within 5 half lives of that investigational product (if known) or 30 days before initiation of the first dose of study drug, whichever is longer.
- •Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening.
研究组 & 干预措施
Ravulizumab
Experimental
During the Primary Treatment Period, all participants will receive weight-based dosing of ravulizumab IV for a total of 50 weeks of treatment.
During the Extension Period, participants will continue to receive weight-based dosing of ravulizumab IV for up to 104 weeks.
干预措施: Ravulizumab (Drug)
结局指标
主要结局
Change From Baseline in the Annualized Relapse Rate at Week 50
时间窗: Baseline, Week 50
Time to First Adjudicated On-trial Relapse through Week 50
时间窗: Baseline through Week 50
次要结局
- Change From Baseline in Expanded Disability Status Scale Score At Week 50(Baseline, Week 50)
- Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50(Baseline through Week 50)
- Change From Baseline in Hauser Ambulation Index at Week 50(Baseline, Week 50)
- Change From Baseline in Confrontational Visual Fields at Week 50(Baseline, Week 50)
- Change From Baseline in Visual Acuity at Week 50(Baseline, Week 50)
- Change From Baseline in Color Vision at Week 50(Baseline, Week 50)
- Serum Ravulizumab Concentration(Predose and postdose (at end of infusion) on Day 1, Weeks 2, 10, 18, 26, and 42, and predose on Week 50)
研究者
研究点 (17)
Loading locations...
相似试验
已完成
3 期
An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSDNeuromyelitis Optica Spectrum DisorderNeuromyelitis OpticaNCT04201262Alexion Pharmaceuticals, Inc.58
已完成
3 期
Study in Pediatrics With HypEREosinophilic Syndrome (SPHERE)Hypereosinophilic SyndromeNCT04965636GlaxoSmithKline16
招募中
3 期
A Study To Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, And Pharmacodynamics Of Satralizumab In Pediatric Patients With Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD)Neuromyelitis Optica Spectrum DisorderNMOSDNCT05199688Hoffmann-La Roche8
已完成
3 期
Study of Ravulizumab in Pediatric Participants With HSCT-TMAThrombotic MicroangiopathyNCT04557735Alexion Pharmaceuticals, Inc.41
终止
2 期
A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum DisorderNeuromyelitis OpticaNeuromyelitis Optica Spectrum DisorderNCT04155424Alexion Pharmaceuticals, Inc.5