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 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-FR
• Lead Sponsor: F. Hoffmann-La Roche Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-12-22
• Last Update Posted: 2024-07-22

Recruitment & Eligibility

• Status: Not Recruiting
• 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
? 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

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
? 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
? Intravenous immunoglobulin (IVIg) within 4 weeks prior to screening
? Plasma exchange (PLEX) within 4 weeks prior to screening
? Treatment with alemtuzumab, cyclophosphamide, total body irradiation, stem cell therapy, or bone marrow transplantation at any time
? Treatment with IL-6 inhibitor therapy at any time
? Treatment with anti-CD19 or CD20 depleting therapy within 6 months prior to baseline
? Treatment with eculizumab, belimumab, natalizumab, teriflunomide, fumaric acid esters (such as dimethyl fumarate), interferons, glatiramer acetate within 6 months prior to baseline
? Use of sphingosine-1-phosphate (S1P) receptor modulators within 6 months prior to baseline
? Treatment with methotrexate within 12 weeks prior to baseline
? Treatment with anti-CD4, cladribine, or mitoxantrone within 2 years prior to baseline
? Treatment with any investigational agent within 6 months prior to baseline

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified