A Phase III, Multicenter, Open-Label, Uncontrolled Study To Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, And Pharmacodynamics Of Satralizumab In Pediatric Patients With AQP4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 8
- 试验地点
- 21
- 主要终点
- Apparent clearance [CL/F] of satralizumab
研究概览
简要总结
This study will primarily evaluate the pharmacokinetics of satralizumab in pediatric patients aged 2-11 years with anti-aquaporin-4 (AQP4) antibody seropositive neuromyelitis optica spectrum disorder (NMOSD). Efficacy, safety, tolerability, and pharmacodynamics will be evaluated in a descriptive manner, given the small number of patients who will be enrolled in this study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age at screening 2-11 years, inclusive
- •Body weight at screening >=10 kg
- •For female patients of childbearing potential (postmenarchal): agreement to either remain completely abstinent (refrain from heterosexual intercourse) or to use a reliable means of contraception
- •Diagnosed as having NMOSD with AQP4 antibody seropositive status as defined by the Wingerchuk 2015 criteria Clinical evidence of at least one documented attack (including first attack) in the last year prior to screening
- •Neurological stability for >=30 days prior to both screening and baseline
- •Expanded Disability Status Scale (EDSS) 0 to 6.5
- •For patients receiving a baseline immunosuppressant treatment and planning to continue on these therapies, treatment must be at stable dose for 4 weeks prior to baseline
排除标准
- •Pregnancy or lactation
- •Evidence of other demyelinating disease mimicking NMOSD
- •Active or presence of recurrent bacterial, viral, fungal, mycobacterial infection, or other infection at baseline
- •Evidence of chronic active hepatitis B or C
- •Evidence of untreated latent or active tuberculosis (TB)
- •Receipt of a live or live-attenuated vaccine within 6 weeks prior to baseline
- •History of severe allergic reaction to a biologic agent
研究组 & 干预措施
Cohort 2 Participants with body weight ≥20kg to <40kg
Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.
干预措施: Satralizumab (Drug)
Cohort 1: Participants with body weight ≥10kg to <20kg
Satralizumab will be administered SC Q6W in a cohort of at least 2 evaluable patients
干预措施: Satralizumab (Drug)
Cohort 3 Participants with body weight ≥40kg
Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.
干预措施: Satralizumab (Drug)
结局指标
主要结局
Apparent clearance [CL/F] of satralizumab
时间窗: Week 48
Area under the concentration-time curve [AUC] of satralizumab
时间窗: Week 48
Apparent volume of distribution [V/F] of satralizumab
时间窗: Week 48
Summary of observed serum concentration [Cthrough] of satralizumab
时间窗: Week 48
次要结局
- Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study(Week 48)
- Change from baseline in visual acuity at Weeks 24 and 48(Baseline, Week 24, Week 48)
- Proportion of relapse-free patients by Week 48(Week 48)
- Change from baseline in EuroQol 5-Dimension, Youth (EQ-5D-Y) score and its proxy at Weeks 24 and 48(Baseline, Week 24, Week 48)
- Time to relapse requiring rescue therapy(Week 48)
- Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48(Baseline, Week 24, Week 48)
- Incidence and severity of adverse events(Week 48)
- Time to first relapse (TFR) after randomization, defined as the time from randomization until the first occurrence of relapse, as determined by the investigator(Week 48)
- Change from baseline in FACES Pain Rating Scale at Weeks 24 and 48(Baseline, Week 24, Week 48)