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NCT05199688PHASE3Recruiting

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)

Hoffmann-La Roche

Start Date

5/6/2026

Completion Date

9/12/2029

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 Criteria

Age Range: 2 years to 11 years

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

Interventions

DRUG

Satralizumab

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Conditions

Neuromyelitis Optica Spectrum DisorderNMOSD

Locations

Children's Hospital Colorado.

Denver, Colorado 80218-1007

United States

Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan

Ciudad Autonoma Buenos Aires, C1249ABN

Argentina

Clinica Universitaria Reina Fabiola

Córdoba, X5004FHP

Argentina

Guangzhou Women and Children's Medical Center

Guangzhou, Guangdong 510120

China

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality 201102

China

Centre Hospitalier Universitaire de Bicêtre

Le Kremlin-Bicêtre, 94275

France

IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN

Rome, Lazio 00165

Italy

Fondazione Istituto Neurologico Mondino IRCCS

Pavia, Lombardy 27100

Italy

Grupo Medico Camino

DF, Mexico CITY (federal District) DUMMY_VALUE

Mexico

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-214

Poland

Instytut "Pomnik - Centrum Zdrowia Dziecka"

Warsaw, 04-730

Poland

Kocaeli University Research and Application Hospit

Kocaeli, 6810

Turkey (Türkiye)

Great Ormond Street Hospital for Children

London, WC1N 3JH

United Kingdom