Satralizumab for Pediatric NMOSD (Aquaporin-4 Antibody Positive)

This study is looking at a medicine called satralizumab for children aged 2 to 11 years old who have neuromyelitis optica spectrum disorder (NMOSD) that is aquaporin-4 (AQP4) antibody positive. NMOSD is a rare, lifelong autoimmune disease that affects the central nervous system. The main goal is to understand how the body processes satralizumab in these children. Researchers will also look at how well satralizumab works, its safety, and how well children tolerate it. Only a small number of children (8) will be included in this study. Participants will receive satralizumab for at least 48 weeks, with an option to continue longer.

Study design
This is an interventional study planning to enroll 8 participants. It is not specified if it is randomized, blinded, or its phase.
What's involved
Participants will receive satralizumab treatment for a minimum of 48 weeks and then have the opportunity to enter an optional extension period.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoints for measuring satralizumab levels are at Week 48. It is not specified how long participants are followed after the optional extension period.

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NCT05199688

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)

Recruiting
PHASE3Ages 2–11InterventionalTreatment
Hoffmann-La Roche
~8 participants
Updated 2026-08-11 on ClinicalTrials.gov
What's tested:Satralizumab

At a glance

Recruiting sites
12 of 13 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Summary of observed serum concentration [Cthrough] of satralizumab
Measured over Week 48
+3 more outcomes measured
Neuromyelitis Optica Spectrum Disorder
NMOSD
13 sites across 11 states
Argentina2
Poland2
Colorado1
Guangdong1
Shanghai Municipality1
Val-de-Marne1
Lazio1
Lombardy1
  • Clinical Trials · STUDY_DIRECTOR · Hoffmann-La Roche
Reference Study ID Number: WN41733 https://forpatients.roche.com/ No attachments to email below.
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Eligibility criteria

Inclusion

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

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
  • Summary of observed serum concentration [Cthrough] of satralizumabWeek 48
  • Apparent clearance [CL/F] of satralizumabWeek 48
  • Apparent volume of distribution [V/F] of satralizumabWeek 48
  • Area under the concentration-time curve [AUC] of satralizumabWeek 48