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)

August 10, 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

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
8

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

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
    • Val-de-Marne
      • Le Kremlin-Bicêtre, Val-de-Marne, 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
      • Gda?sk, 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Hoffmann-La Roche

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 6, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 31, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 12, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 3, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 5, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 20, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 11, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 10, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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