Neuromyelitis Optica Spectrum DisorderPhase 3SAkuraSunNCT05199688
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)
A Phase 3 study testing Satralizumab in people with Neuromyelitis Optica Spectrum Disorder and NMOSD, run by Hoffmann-La Roche at 14 sites in 9 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 2 to 11 years
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorder, NMOSD
- What is being tested?
- Satralizumab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 14 sitesin 9 countries
- Study dates
- 6 May 2026to 12 Sep 2029
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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:
- Documented history of a positive anti-AQP4 antibody test or a positive anti-AQP4 antibody test at screening;
- Neurological stability for >=30 days prior to baseline
- Expanded Disability Status Scale (EDSS) 0 to 6.5
- For participants receiving a baseline immunosuppressant treatment and planning to continue on these therapies, treatment must be at stable dose for 4 weeks prior to baseline
Who cannot join
- 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
- IVIg or subcutaneous Ig (SCIg) within 4 weeks prior to enrollment/Day 1
Trial locations and contacts
14 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
Reference Study ID Number: WN41733 https://forpatients.roche.com/ No attachments to email below. contact
Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry contact
14 sites in 9 countries
Argentina2 sites, 2 recruiting
- Recruiting
Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
Ciudad Autonoma Buenos Aires, C1249ABN
- Recruiting
Clinica Universitaria Reina Fabiola
Córdoba, X5004FHP
China2 sites, 2 recruiting
- Recruiting
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong, 510120
- Recruiting
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102
Italy2 sites, 2 recruiting
- Recruiting
Fondazione Istituto Neurologico Mondino IRCCS
Pavia, Lombardy, 27100
- Recruiting
IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
Rome, Lazio, 00165
Mexico2 sites, 2 recruiting
- Recruiting
Grupo Medico Camino
DF, Mexico CITY (federal District)
- Recruiting
Hospital Infantil de Mexico Federico Gomez
Mexico City, Mexico CITY (federal District), 06720
Poland2 sites, 1 recruiting
- Active, not recruiting
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-214
- Recruiting
Instytut "Pomnik - Centrum Zdrowia Dziecka"
Warsaw, 04-730
United States1 site, 1 recruiting
- Recruiting
Children's Hospital Colorado.
Denver, Colorado, 80218-1007
France1 site, 1 recruiting
- Recruiting
Centre Hospitalier Universitaire de Bicêtre
Le Kremlin-Bicêtre, Val-de-Marne, 94275
Turkey (Türkiye)1 site, 1 recruiting
- Recruiting
Kocaeli University Research and Application Hospit
Kocaeli, 6810
United Kingdom1 site, 1 recruiting
- Recruiting
Great Ormond Street Hospital for Children
London, WC1N 3JH
About this study
Official 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)
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.
- Conditions
- Neuromyelitis Optica Spectrum Disorder, NMOSD
- Being tested
- Satralizumab
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SatralizumabDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Participants with body weight ≥10kg to <20kg | Experimental | Satralizumab will be administered SC Q6W in a cohort of at least 2 evaluable patients Receives: Drug: Satralizumab |
| Cohort 2 Participants with body weight ≥20kg to <40kg | Experimental | Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter. Receives: Drug: Satralizumab |
| Cohort 3 Participants with body weight ≥40kg | Experimental | Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter. Receives: Drug: Satralizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Summary of Observed Serum Concentration of Satralizumab
Measured: Week 48
Apparent Clearance [CL/F] of Satralizumab
Measured: Week 48
Apparent Volume of Distribution [V/F] of Satralizumab
Measured: Week 48
Area Under the Concentration-time Curve [AUC] of Satralizumab
Measured: Week 48
Secondary outcomes (9)
Proportion of Relapse-free Participants by Week 48
Measured: Week 48
Annualized Relapse Rate (ARR), defined as the average number of relapses for each year of the study
Measured: Week 48
Time to First Relapse (TFR) after enrollment, defined as the time from enrollment until the first occurrence of relapse, as determined by the investigator
Measured: Week 48
Time to Relapse Requiring Rescue Therapy
Measured: Week 48
Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48
Measured: Baseline, Week 24, Week 48
Change from Baseline in Visual Acuity at Weeks 24 and 48
Measured: Baseline, Week 24, Week 48
Change from Baseline in FACES Pain Rating Scale at Weeks 24 and 48
Measured: 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
Measured: Baseline, Week 24, Week 48
Incidence and Severity of Adverse Events
Measured: Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jan 2022
- Study start6 May 2026
- Primary completion31 Mar 2027 (estimated)
- Study completion12 Sep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis Optica Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05199688
- Sponsor's protocol ID
- WN41733
- Other IDs
- 2019-004092-39, 2023-507817-85-00, 2023, 2019
- Acronym
- SAkuraSun
- Status last verified
- Sep 2026
- First submitted
- 3 Dec 2021
- Last updated
- 1 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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