Neuromyelitis Optica Spectrum Disorder, NMOSD
Conditions
Brief 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.
Interventions
DRUGSatralizumab
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.
Sponsors
Hoffmann-La Roche
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 |
Secondary
| Measure | Time frame |
|---|---|
| Proportion of relapse-free patients by Week 48 | Week 48 |
| Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study | 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 | Week 48 |
| Time to relapse requiring rescue therapy | Week 48 |
| Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48 | Baseline, Week 24, Week 48 |
| Change from baseline in visual acuity at Weeks 24 and 48 | Baseline, Week 24, Week 48 |
| Change from baseline in FACES Pain Rating Scale at Weeks 24 and 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 | Baseline, Week 24, Week 48 |
| Incidence and severity of adverse events | Week 48 |
Countries
Argentina, China, France, Italy, Mexico, Poland, Turkey (Türkiye), United Kingdom, United States
Contacts
CONTACTReference Study ID Number: WN41733 https://forpatients.roche.com/
CONTACTGlobal Medical Information
STUDY_DIRECTORClinical Trials
Hoffmann-La Roche
Outcome results
None listed