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) (SAkuraSun)

June 5, 2026 updated by: Hoffmann-La Roche

A Phase III, Multicenter, Open-Label, Uncontrolled Study To Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, And Pharmacodynamics Of Satralizumab In Pediatric Patients With AQP4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD)

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 Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

8

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Argentina
      • Ciudad Autonoma Buenos Aires, Argentina, C1249ABN
        • Recruiting
        • Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
      • Córdoba, Argentina, X5004FHP
        • Recruiting
        • Clinica Universitaria Reina Fabiola
  • China
    • Guangdong
      • Guangzhou, Guangdong, China, 510120
        • Recruiting
        • Guangzhou Women and Children's Medical Center
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, China, 201102
        • Recruiting
        • Children's Hospital of Fudan University
  • France
      • Le Kremlin-Bicêtre, France, 94275
        • Recruiting
        • Centre Hospitalier Universitaire de Bicêtre
  • Italy
    • Lazio
      • Rome, Lazio, Italy, 00165
        • Recruiting
        • IRCCS Ospedale Pediatrico Bambino Gesu - INCIPIT - PIN
    • Lombardy
      • Pavia, Lombardy, Italy, 27100
        • Recruiting
        • Fondazione Istituto Neurologico Mondino IRCCS
  • Mexico
    • Mexico CITY (federal District)
      • DF, Mexico CITY (federal District), Mexico, DUMMY_VALUE
        • Recruiting
        • Grupo Medico Camino
  • Poland
      • Gdansk, Poland, 80-214
        • Active, not recruiting
        • Uniwersyteckie Centrum Kliniczne
      • Warsaw, Poland, 04-730
        • Recruiting
        • Instytut "Pomnik - Centrum Zdrowia Dziecka"
  • Turkey (Türkiye)
      • Kocaeli, Turkey (Türkiye), 6810
        • Recruiting
        • Kocaeli University Research and Application Hospit
  • United Kingdom
      • London, United Kingdom, WC1N 3JH
        • Recruiting
        • Great Ormond Street Hospital For Children
  • United States
    • Colorado
      • Denver, Colorado, United States, 80218-1007
        • Recruiting
        • Children's Hospital Colorado.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 11 years (Child)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1: Participants with body weight ≥10kg to <20kg
Satralizumab will be administered SC Q6W in a cohort of at least 2 evaluable patients
Drug: 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.
Experimental: Cohort 2 Participants with body weight ≥20kg to <40kg
Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.
Drug: 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.
Experimental: Cohort 3 Participants with body weight ≥40kg
Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.
Drug: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Summary of observed serum concentration [Cthrough] of satralizumab
Time Frame: Week 48
Week 48
Apparent clearance [CL/F] of satralizumab
Time Frame: Week 48
Week 48
Apparent volume of distribution [V/F] of satralizumab
Time Frame: Week 48
Week 48
Area under the concentration-time curve [AUC] of satralizumab
Time Frame: Week 48
Week 48

Secondary Outcome Measures

Outcome Measure
Time Frame
Proportion of relapse-free patients by Week 48
Time Frame: Week 48
Week 48
Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study
Time Frame: Week 48
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
Week 48
Time to relapse requiring rescue therapy
Time Frame: Week 48
Week 48
Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48
Time Frame: Baseline, Week 24, Week 48
Baseline, Week 24, Week 48
Change from baseline in visual acuity at Weeks 24 and 48
Time Frame: Baseline, Week 24, Week 48
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
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
Baseline, Week 24, Week 48
Incidence and severity of adverse events
Time Frame: Week 48
Week 48

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Hoffmann-La Roche

Investigators

  • Study Director: Clinical Trials, Hoffmann-La Roche

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 6, 2026

Primary Completion (Estimated)

March 31, 2027

Study Completion (Estimated)

September 12, 2029

Study Registration Dates

First Submitted

December 3, 2021

First Submitted That Met QC Criteria

January 5, 2022

First Posted (Actual)

January 20, 2022

Study Record Updates

Last Update Posted (Actual)

June 9, 2026

Last Update Submitted That Met QC Criteria

June 5, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • WN41733
  • 2019-004092-39 (EudraCT Number)
  • 2023-507817-85-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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