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)
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.
Study 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). 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.
Eligibility
Sex
ALL
Min age
2 Years
Max age
11 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Summary of observed serum concentration [Cthrough] of satralizumab — Week 48
Apparent clearance [CL/F] of satralizumab — Week 48
Apparent volume of distribution [V/F] of satralizumab — Week 48
Area under the concentration-time curve [AUC] of satralizumab — Week 48
Trial sites (13)
Facility
City
Region
Status
Children's Hospital Colorado.
Denver
Colorado
Recruiting
Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
Kocaeli University Research and Application Hospit
Kocaeli
Turkey (Türkiye)
Recruiting
Great Ormond Street Hospital for Children
London
United Kingdom
Recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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