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)

NCT ID: NCT05199688

Last Updated: 2025-12-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-12-31

Study Completion Date

2029-09-12

Brief Summary

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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

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Conditions

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Neuromyelitis Optica Spectrum Disorder NMOSD

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1: Participants with body weight ≥10kg to <20kg

Satralizumab will be administered SC Q6W in a cohort of at least 2 evaluable patients

Group Type EXPERIMENTAL

Satralizumab

Intervention Type 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 2 Participants with body weight ≥20kg to <40kg

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

Group Type EXPERIMENTAL

Satralizumab

Intervention Type 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 3 Participants with body weight ≥40kg

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

Group Type EXPERIMENTAL

Satralizumab

Intervention Type 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.

Interventions

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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.

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

2 Years

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hoffmann-La Roche

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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Children's Hospital Colorado.

Denver, Colorado, United States

Site Status RECRUITING

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

Ciudad Autonoma Buenos Aires, , Argentina

Site Status RECRUITING

Clinica Universitaria Reina Fabiola

Córdoba, , Argentina

Site Status RECRUITING

Centre Hospitalier Universitaire de Bicêtre

Le Kremlin-Bicêtre, , France

Site Status RECRUITING

IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN

Rome, Lazio, Italy

Site Status RECRUITING

Fondazione Istituto Neurologico Mondino IRCCS

Pavia, Lombardy, Italy

Site Status RECRUITING

Grupo Medico Camino

DF, Mexico CITY (federal District), Mexico

Site Status RECRUITING

Uniwersyteckie Centrum Kliniczne

Gda?sk, , Poland

Site Status ACTIVE_NOT_RECRUITING

Kocaeli University Research and Application Hospit

Kocaeli, , Turkey (Türkiye)

Site Status RECRUITING

Great Ormond Street Hospital for Children

London, , United Kingdom

Site Status RECRUITING

Countries

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Ukraine United States Argentina France Italy Mexico Poland Turkey (Türkiye) United Kingdom

Central Contacts

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

Role: CONTACT

Phone: 888-662-6728 (U.S.)

Email: [email protected]

Global Medical Information

Role: CONTACT

Email: [email protected]

Other Identifiers

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2019-004092-39

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2023-507817-85-00

Identifier Type: CTIS

Identifier Source: secondary_id

WN41733

Identifier Type: -

Identifier Source: org_study_id