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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
8
Participants will receive satralizumab treatment for a minimum of 48 weeks and then will have the opportunity to enter an optional satralizumab extension (OSE) period.
Children's Hospital Colorado.
Denver, Colorado, United States
RECRUITINGHospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
Ciudad Autonoma Buenos Aires, Argentina
RECRUITINGClinica Universitaria Reina Fabiola
Córdoba, Argentina
RECRUITINGChildren's Hospital of Fudan University
Shanghai, Shanghai Municipality, China
RECRUITINGCentre Hospitalier Universitaire de Bicêtre
Le Kremlin-Bicêtre, France
RECRUITINGIRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
Rome, Lazio, Italy
RECRUITINGFondazione Istituto Neurologico Mondino IRCCS
Pavia, Lombardy, Italy
RECRUITINGGrupo Medico Camino
DF, Mexico CITY (federal District), Mexico
RECRUITINGUniwersyteckie Centrum Kliniczne
Gdansk, Poland
ACTIVE_NOT_RECRUITINGInstytut "Pomnik - Centrum Zdrowia Dziecka"
Warsaw, Poland
RECRUITING...and 2 more locations
Summary of observed serum concentration [Cthrough] of satralizumab
Time frame: Week 48
Apparent clearance [CL/F] of satralizumab
Time frame: Week 48
Apparent volume of distribution [V/F] of satralizumab
Time frame: Week 48
Area under the concentration-time curve [AUC] of satralizumab
Time frame: Week 48
Proportion of relapse-free patients by Week 48
Time frame: Week 48
Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study
Time frame: 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
Time frame: Week 48
Time to relapse requiring rescue therapy
Time frame: Week 48
Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48
Time frame: Baseline, Week 24, Week 48
Change from baseline in visual acuity at Weeks 24 and 48
Time frame: Baseline, Week 24, Week 48
Change from baseline in FACES Pain Rating Scale at Weeks 24 and 48
Time frame: Baseline, Week 24, Week 48
Change from baseline in EuroQol 5-Dimension, Youth (EQ-5D-Y) score and its proxy at Weeks 24 and 48
Time frame: Baseline, Week 24, Week 48
Incidence and severity of adverse events
Time frame: Week 48
Reference Study ID Number: WN41733 https://forpatients.roche.com/
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