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

Active, not recruiting

Conditions: euromyelitis Optica Spectrum Disorder (NMOSD)
MedDRA version: 20.0Level: LLTClassification code: 10029322Term: Neuromyelitis optica Class: 10029205
MedDRA version: 21.1Level: PTClassification code: 10077875Term: Neuromyelitis optica spectrum disorder Class: 100000004852
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: CTIS2023-507817-85-00

Lead Sponsor: F. Hoffmann-La Roche AG

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Not Recruiting

Sex: All

Target Recruitment: 21

Inclusion Criteria

1. Age at screening 2-11 years, inclusive, 2. Body weight at screening =10 kg, 3. For female participants of childbearing potential (postmenarchal): agreement to either remain completely abstinent (refrain from heterosexual intercourse) or to use a reliable means of contraception, 4. Diagnosed as having NMOSD with AQP4 antibody seropositive status as defined by the Wingerchuk 2015 criteria, 5. Neurological stability for =30 days prior to both screening and baseline, 6. Normal or abnormal neurologic status as described by EDSS (0 to 6.5)

Exclusion Criteria

1. Pregnancy or lactation, 2. Evidence of other demyelinating disease mimicking NMOSD including but not limited to MS, myelin oligodendrocyte glycoprotein-IgG associated disease, or progressive multifocal leukoencephalopathy, 3. Active or presence of recurrent bacterial, viral, fungal, mycobacterial infection, or other infection (excluding fungal infection of nail beds or dental caries) at baseline, 4. Evidence of chronic active hepatitis B or C, 5. Evidence of untreated latent or active tuberculosis (TB), 6. 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: 1. To investigate the pharmacokinetics (PK) of satralizumab by evaluating serum exposure over 24 weeks in participants aged 2-11 years, inclusive;Secondary Objective: 1. To evaluate the efficacy of subcutaneously administered satralizumab for 48 weeks in pediatric patients (three weight-based cohorts)., 2. To evaluate the safety of subcutaneously administered satralizumab for 48 weeks in pediatric patients (three weight-based cohorts), 3. To evaluate the immune response to satralizumab;Primary end point(s): 1. Summary of observed serum concentration of satralizumab at specified trough timepoints (mean and standard deviation of trough concentration [Ctrough] at each sampling timepoint), 2. Population and individual estimates of PK parameters using a population-PK modeling approach

Secondary Outcome Measures