A Study to Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, and Pharmacodynamics of Satralizumab in Pediatric Patients with Aquaporin-4 (AQP4) Antibody Positive Neuromyelitis Optica Spectrum Disorder

Phase 1

Conditions: euromyelitis Optica Spectrum Disorder (NMOSD)
MedDRA version: 20.0Level: LLTClassification code 10029322Term: Neuromyelitis opticaSystem Organ Class: 10029205 - Nervous system disorders
MedDRA version: 21.1Level: PTClassification code 10077875Term: Neuromyelitis optica spectrum disorderSystem Organ Class: 10029205 - Nervous system disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: EUCTR2019-004092-39-PL

Lead Sponsor: F. Hoffmann-La Roche Ltd

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

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
? Neurological stability for >=30 days prior to both screening and baseline
?Normal or abnormal neurologic status as described by EDSS (0 to 6.5)

Are the trial subjects under 18? yes
Number of subjects for this age range: 8
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

? Pregnancy or lactation
? Evidence of other demyelinating disease mimicking NMOSD including but not limited to MS, myelin oligodendrocyte glycoprotein-IgG associated disease, or progressive multifocal leukoencephalopathy
? Active or presence of recurrent bacterial, viral, fungal, mycobacterial infection, or other infection (excluding fungal infection of nail beds or dental caries) 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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: ? To investigate the pharmacokinetics of satralizumab by evaluating<br>serum exposure over 24 weeks in patients aged 2-11 years;Secondary Objective: ?To evaluate the efficacy of subcutaneously administered satralizumab<br>for 48 weeks in pediatric patients (three weight-based cohorts).<br>? To evaluate the safety of subcutaneously administered satralizumab<br>for 48 weeks in pediatric patients (three weight-based cohorts)<br>? To evaluate the immune response to satralizumab<br>;Primary end point(s): 1. Summary of observed serum concentration of satralizumab at<br>specified trough timepoints (mean and standard deviation of trough<br>concentration [Ctrough] at each sampling timepoint)<br>2. Population and individual estimates of PK parameters using a<br>population-PK modeling approach<br>;Timepoint(s) of evaluation of this end point: 1-2. Baseline through Week 24

Secondary Outcome Measures

Name	Time	Method

Related Trials