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) (SAkuraSun)

Roche

Status and phase: Enrolling; Phase 3

Conditions: NMOSD; Neuromyelitis Optica Spectrum Disorder

Treatments: Drug: Satralizumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT05199688

2019-004092-39 (EudraCT Number)

2023-507817-85-00 (EU Trial (CTIS) Number)

WN41733

Details and patient eligibility

About

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.

Enrollment: 8 estimated patients

Sex: All

Ages: 2 to 11 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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8 participants in 3 patient groups

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

Experimental group

Description:

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

Treatment:

Drug: Satralizumab

Cohort 2 Participants with body weight ≥20kg to <40kg

Experimental group

Description:

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

Treatment:

Drug: Satralizumab

Cohort 3 Participants with body weight ≥40kg

Experimental group

Description:

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

Treatment:

Drug: Satralizumab