Recruiting NCT05199688
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)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Satralizumab.
- Who it may be relevant to
- Registry conditions: Neuromyelitis Optica Spectrum Disorder, NMOSD. Basic parameters: 2 years — 11 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Argentina, China, France, Italy +4
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase III, Multicenter, Open-Label, Uncontrolled Study To Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, And Pharmacodynamics Of Satralizumab In Pediatric Patients With AQP4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD)
Overview
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.
Interventions
- Drug Satralizumab
Participants will receive satralizumab treatment for a minimum of 48 weeks and then will have the opportunity to enter an optional satralizumab extension (OSE) period.
Primary outcome measures
- Summary of observed serum concentration [Cthrough] of satralizumab [Time frame: Week 48]
- Apparent clearance [CL/F] of satralizumab [Time frame: Week 48]
- Apparent volume of distribution [V/F] of satralizumab [Time frame: Week 48]
- Area under the concentration-time curve [AUC] of satralizumab [Time frame: Week 48]
Secondary outcome measures (9)
- Proportion of relapse-free patients by Week 48 [Time frame: Week 48]
- Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study [Time frame: Week 48]
- Time to first relapse (TFR) after randomization, defined as the time from randomization until the first occurrence of relapse, as determined by the investigator [Time frame: Week 48]
- Time to relapse requiring rescue therapy [Time frame: Week 48]
- Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48 [Time frame: Baseline, Week 24, Week 48]
- Change from baseline in visual acuity at Weeks 24 and 48 [Time frame: Baseline, Week 24, Week 48]
- Change from baseline in FACES Pain Rating Scale at Weeks 24 and 48 [Time frame: Baseline, Week 24, Week 48]
- Change from baseline in EuroQol 5-Dimension, Youth (EQ-5D-Y) score and its proxy at Weeks 24 and 48 [Time frame: Baseline, Week 24, Week 48]
- Incidence and severity of adverse events [Time frame: Week 48]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Argentina · 2 centers
- Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan — Ciudad Autonoma Buenos Aires
- Clinica Universitaria Reina Fabiola — Córdoba
China · 2 centers
- Guangzhou Women and Children's Medical Center — Guangzhou
- Children's Hospital of Fudan University — Shanghai
Italy · 2 centers
- IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN — Rome
- Fondazione Istituto Neurologico Mondino IRCCS — Pavia
Poland · 2 centers
- Uniwersyteckie Centrum Kliniczne — Gdansk
- Instytut "Pomnik - Centrum Zdrowia Dziecka" — Warsaw
United States · 1 center
- Children's Hospital Colorado. — Denver
France · 1 center
- Centre Hospitalier Universitaire de Bicêtre — Le Kremlin-Bicêtre
Mexico · 1 center
- Grupo Medico Camino — DF
Turkey (Türkiye) · 1 center
- Kocaeli University Research and Application Hospit — Kocaeli
United Kingdom · 1 center
- Great Ormond Street Hospital for Children — London
Identifiers
NCT: NCT05199688 · WN41733 · 2019-004092-39 · 2023-507817-85-00