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
- Conditions
- euromyelitis Optica Spectrum Disorder (NMOSD)MedDRA version: 20.0Level: LLTClassification code 10029322Term: Neuromyelitis opticaSystem Organ Class: 10029205 - Nervous system disordersMedDRA version: 21.1Level: PTClassification code 10077875Term: Neuromyelitis optica spectrum disorderSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2019-004092-39-IT
- 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
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:
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
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
- Primary Outcome Measures
Name Time Method Main Objective: To investigate the pharmacokinetics of satralizumab in patients aged 2-11 years;Secondary Objective: To evaluate efficacy of satralizumab in pediatric patients aged 2-11 years<br>To evaluate safety of satralizumab in pediatric patients aged 2-11 years<br>To evaluate the immune response to satralizumab in pediatric patients aged 2-11 years;Primary end point(s): 1. Summary of observed serum concentration of satralizumab at specified trough timepoints <br>2. Population and individual estimates of PK parameters<br>;Timepoint(s) of evaluation of this end point: 1-2. Baseline through Week 24
- Secondary Outcome Measures
Name Time Method