A Phase III, Open-Label Study to Evaluate Satralizumab Pharmacokinetics, Efficacy, Safety, and Tolerability in Children Aged 2-11 Years with AQP4 Antibody Positive Neuromyelitis Optica Spectrum Disorder

What this Trial Is About

Researchers are evaluating the pharmacokinetics of satralizumab in children aged 2 to 11 years who have neuromyelitis optica spectrum disorder (NMOSD) with anti-aquaporin-4 (AQP4) antibody positivity. This study also descriptively assesses the drug's efficacy, safety, tolerability, and pharmacodynamics due to the small number of participants. The study is a phase III, open-label, uncontrolled trial sponsored by Hoffmann-La Roche. Satralizumab will be given as a subcutaneous injection with an initial dosing schedule at Weeks 0, 2, and 4, followed by injections every 4 weeks thereafter. Participants are grouped into cohorts based on body weight (10 to under 20 kg, 20 to under 40 kg, and 40 kg or more). Treatment will continue for at least 48 weeks, after which participants may opt to join an extension period to receive satralizumab further. Participants will undergo assessments including blood sampling to measure satralizumab concentration and pharmacokinetic parameters at Week 48. Other evaluations include relapse frequency, disability status, visual acuity, pain rating, quality of life, and monitoring for adverse events. The study involves regular visits and monitoring over the treatment period to track these outcomes and the drug's effects in pediatric patients with NMOSD.

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)