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
RECRUITINGGuangzhou Women and Children's Medical Center
Guangzhou, Guangdong, China
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_RECRUITING...and 3 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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