RECRUITING Phase 3 ClinicalTrials.gov

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)

Sponsor: Hoffmann-La Roche • ClinicalTrials.gov ID: NCT05199688

Registry updated: 2026-10-01

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Description

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.

Interventions

Satralizumab (Drug)
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.

Eligibility criteria

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: * Documented history of a positive anti-AQP4 antibody test or a positive anti-AQP4 antibody test at screening; * Neurological stability for \>=30 days prior to baseline * Expanded Disability Status Scale (EDSS) 0 to 6.5 * For participants 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 * IVIg or subcutaneous Ig (SCIg) within 4 weeks prior to enrollment/Day 1

Conditions

Neuromyelitis Optica Spectrum Disorder NMOSD

Locations

Children's Hospital Colorado. — Denver, Colorado, United States
Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan — Ciudad Autonoma Buenos Aires, Argentina
Clinica Universitaria Reina Fabiola — Córdoba, Argentina
Guangzhou Women and Children's Medical Center — Guangzhou, Guangdong, China
Children's Hospital of Fudan University — Shanghai, Shanghai Municipality, China
Centre Hospitalier Universitaire de Bicêtre — Le Kremlin-Bicêtre, Val-de-Marne, France
IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN — Rome, Lazio, Italy
Fondazione Istituto Neurologico Mondino IRCCS — Pavia, Lombardy, Italy
Grupo Medico Camino — DF, Mexico CITY (federal District), Mexico
Hospital Infantil de Mexico Federico Gomez — Mexico City, Mexico CITY (federal District), Mexico
Uniwersyteckie Centrum Kliniczne — Gdansk, Poland
Instytut "Pomnik - Centrum Zdrowia Dziecka" — Warsaw, Poland
Kocaeli University Research and Application Hospit — Kocaeli, Turkey (Türkiye)
Great Ormond Street Hospital for Children — London, United Kingdom

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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