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)

Phase 3

Recruiting

• Conditions: Neuromyelitis Optica Spectrum Disorder; NMOSD
• Interventions: Drug: Satralizumab

• Registration Number: NCT05199688
• Lead Sponsor: Hoffmann-La Roche

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-12-03
• Study First Submitted QC: 2022-01-05
• Study First Posted: 2022-01-20
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-02
• Last Update Posted: 2025-05-06
• Study Start: 2025-06-30
• Primary Completion: 2027-03-31
• Study Completion: 2029-09-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 8

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort 1: Participants with body weight ≥10kg to <20kg	Satralizumab	Satralizumab will be administered SC Q6W in a cohort of at least 2 evaluable patients
Cohort 2 Participants with body weight ≥20kg to <40kg	Satralizumab	Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.
Cohort 3 Participants with body weight ≥40kg	Satralizumab	Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.

Primary Outcome Measures

Name	Time	Method
Apparent clearance [CL/F] of satralizumab	Week 48
Area under the concentration-time curve [AUC] of satralizumab	Week 48
Apparent volume of distribution [V/F] of satralizumab	Week 48
Summary of observed serum concentration [Cthrough] of satralizumab	Week 48

Secondary Outcome Measures

Name	Time	Method
Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study	Week 48
Change from baseline in visual acuity at Weeks 24 and 48	Baseline, Week 24, Week 48
Proportion of relapse-free patients by Week 48	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
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
Incidence and severity of adverse events	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
Change from baseline in FACES Pain Rating Scale at Weeks 24 and 48	Baseline, Week 24, Week 48

Trial Locations

Locations (8)

Children's Hospital Colorado.; 🇺🇸 Denver, Colorado, United States

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

Clinica Universitaria Reina Fabiola; 🇦🇷 Cordoba, Argentina

Centre Hospitalier Universitaire de Bicêtre; 🇫🇷 Le Kremlin-bicêtre, France

IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN; 🇮🇹 Roma, Lazio, Italy

Fondazione Istituto Neurologico Mondino IRCCS; 🇮🇹 Pavia, Lombardia, Italy

Uniwersyteckie Centrum Kliniczne; 🇵🇱 Gda?sk, Poland

Great Ormond Street Hospital for Children; 🇬🇧 London, United Kingdom