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)

NCT05199688 | Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: WN417332019-004092-392023-507817-85-00
• Study Type: interventional
• Target: 8
• Locations: 9 countries
• Sites: 13

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.

Conditions

Neuromyelitis Optica Spectrum Disorder; NMOSD

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (9)

• 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

Study Arms (3)

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

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.

Drug: Satralizumab

Cohort 3 Participants with body weight ≥40kg

EXPERIMENTAL

Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.

Drug: Satralizumab

Interventions

Satralizumab DRUG

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.

Cohort 1: Participants with body weight ≥10kg to <20kg; Cohort 2 Participants with body weight ≥20kg to <40kg; Cohort 3 Participants with body weight ≥40kg

Eligibility Criteria

• Age: 2 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (13)

Children's Hospital Colorado.

Denver, Colorado, 80218-1007, United States

RECRUITING

Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan

Ciudad Autonoma Buenos Aires, C1249ABN, Argentina

RECRUITING

Clinica Universitaria Reina Fabiola

Córdoba, X5004FHP, Argentina

RECRUITING

Guangzhou Women and Children's Medical Center

Guangzhou, Guangdong, 510120, China

RECRUITING

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, 201102, China

RECRUITING

Centre Hospitalier Universitaire de Bicêtre

Le Kremlin-Bicêtre, 94275, France

RECRUITING

IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN

Rome, Lazio, 00165, Italy

RECRUITING

Fondazione Istituto Neurologico Mondino IRCCS

Pavia, Lombardy, 27100, Italy

RECRUITING

Grupo Medico Camino

DF, Mexico CITY (federal District), DUMMY_VALUE, Mexico

RECRUITING

Uniwersyteckie Centrum Kliniczne

Gda?sk, 80-214, Poland

ACTIVE NOT RECRUITING

Instytut "Pomnik - Centrum Zdrowia Dziecka"

Warsaw, 04-730, Poland

RECRUITING

Kocaeli University Research and Application Hospit

Kocaeli, 6810, Turkey (Türkiye)

RECRUITING

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

RECRUITING

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

satralizumab

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Central Study Contacts

Reference Study ID Number: WN41733 https://forpatients.roche.com/

CONTACT

888-662-6728 (U.S.); global-roche-genentech-trials@gene.com

Global Medical Information

CONTACT

global.medical_information@roche.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 3, 2021
• First Posted: January 20, 2022
• Study Start: April 30, 2026
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): September 12, 2029
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

PL (2); FR (1); AR (2); CN (2); US (1); GB (1); IT (2); TU (1); ME (1)