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.
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
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
Parallel
Satralizumab
DrugParticipants will receive satralizumab treatment for a minimum of 48 weeks and then will have the opportunity to enter an optional satralizumab extension (OSE) period.
Summary of observed serum concentration [Cthrough] of satralizumab
Apparent clearance [CL/F] of satralizumab
Apparent volume of distribution [V/F] of satralizumab
Area under the concentration-time curve [AUC] of satralizumab
Proportion of relapse-free patients by Week 48
Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study
Time to first relapse (TFR) after randomization, defined as the time from randomization until the first occurrence of relapse, as determined by the investigator
Time to relapse requiring rescue therapy
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