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NCT Number: 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)

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.

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Key information

Age range

2 year–11 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan, Ciudad Autonoma Buenos Aires, Argentina

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Primary outcomes

  1. Summary of observed serum concentration [Cthrough] of satralizumab

    Time frame: Week 48

  2. Apparent clearance [CL/F] of satralizumab

    Time frame: Week 48

  3. Apparent volume of distribution [V/F] of satralizumab

    Time frame: Week 48

  4. Area under the concentration-time curve [AUC] of satralizumab

    Time frame: Week 48

Secondary outcomes

  1. Proportion of relapse-free patients by Week 48

    Time frame: Week 48

  2. Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study

    Time frame: Week 48

  3. 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

  4. Time to relapse requiring rescue therapy

    Time frame: Week 48

  5. Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48

    Time frame: Baseline, Week 24, Week 48

  6. Change from baseline in visual acuity at Weeks 24 and 48

    Time frame: Baseline, Week 24, Week 48

  7. Change from baseline in FACES Pain Rating Scale at Weeks 24 and 48

    Time frame: Baseline, Week 24, Week 48

  8. 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

  9. Incidence and severity of adverse events

    Time frame: Week 48

Study contacts

Contact information is provided by the study sponsor or research team.

Fastest response: use the inquiry form. No email attachments. https://www.gene.com/contact-us/submit-medical-inquiry

CONTACT

[email protected]

Reference Study ID Number: WN41733 https://forpatients.roche.com/

CONTACT

[email protected]

888-662-6728 (U.S.)

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Registry information

Official study title

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)

Acronym: SAkuraSun

Important dates

Study start
2026
Primary completion
2027
Study completion
2029
First posted
Jan 20, 2022
Registry last updated
Jul 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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