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Clinical Trials in the USA / NCT05199688
Recruiting Phase 3

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)

NCT05199688 · tracked via the Priya Life Science USA tracker
Sponsor
Hoffmann-La Roche
Phase
Phase 3
Started
2026-05-06
Last updated
2026-08-11

Condition(s) studied

Neuromyelitis Optica Spectrum DisorderNMOSD

Investigational drug(s) / intervention(s)

Satralizumab

Satralizumab: 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.

Study summary

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.

Eligibility

Sex
ALL
Min age
2 Years
Max age
11 Years
Healthy volunteers
No
Inclusion Criteria: * 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 Exclusion Criteria: * 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

Primary outcome measure(s)

Trial sites (13)

FacilityCityRegionStatus
Children's Hospital Colorado. Denver Colorado Recruiting
Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan Ciudad Autonoma Buenos Aires Argentina Recruiting
Clinica Universitaria Reina Fabiola Córdoba Argentina Recruiting
Guangzhou Women and Children's Medical Center Guangzhou Guangdong Recruiting
Children's Hospital of Fudan University Shanghai Shanghai Municipality Recruiting
Centre Hospitalier Universitaire de Bicêtre Le Kremlin-Bicêtre Val-de-Marne Recruiting
IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN Rome Lazio Recruiting
Fondazione Istituto Neurologico Mondino IRCCS Pavia Lombardy Recruiting
Grupo Medico Camino DF Mexico CITY (federal District) Recruiting
Uniwersyteckie Centrum Kliniczne Gda?sk Poland Active Not Recruiting
Instytut "Pomnik - Centrum Zdrowia Dziecka" Warsaw Poland Recruiting
Kocaeli University Research and Application Hospit Kocaeli Turkey (Türkiye) Recruiting
Great Ormond Street Hospital for Children London United Kingdom Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05199688 on ClinicalTrials.gov ↗ ← All trials in the USA