A phase 3 clinical trial evaluating the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of satralizumab (Enspryng®) in children with aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD) is now recruiting participants.
The open-label, uncontrolled study will primarily evaluate the pharmacokinetics of the treatment in children aged 2 to 11 years. It aims to recruit 8 participants at Uniwersyteckie Centrum Kliniczne in Gdansk, Poland.
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Participants will be divided into 3 groups based on their body weight. Those in the first group (body weight 10-20 kg) will receive subcutaneous satralizumab every 6 weeks for at least 48 weeks. Those in the second (body weight 20-40 kg) and third (body weight of at least 40 kg) groups will receive subcutaneous satralizumab at weeks 0, 2, and 4 and every 4 weeks thereafter for a minimum of 48 weeks.
The primary outcome measures are the summary of observed serum concentration, apparent clearance, volume of distribution, and area under the concentration-time curve of satralizumab.
Secondary outcome measures include the proportion of relapse-free patients by week 48, annualized relapse rate, time to relapse requiring rescue therapy, and change from baseline in Expanded Disability Status Scale (EDSS) score, visual acuity, and FACES Pain Rating Scale score at weeks 24 and 48, as well as the incidence and severity of adverse events.
The trial is estimated to start on August 15, 2022, and be completed on February 28, 2027.
Satralizumab is a monoclonal antibody against interleukin 6 (IL-6), which is involved in the production of antibodies against AQP4. It has been approved by the US Food and Drug Administration (FDA) to treat adult patients with AQP4 antibody-positive NMOSD and by the European Medicines Agency (EMA) to treat patients aged 12 years and above.
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). ClinicalTrials.gov. January 20, 2022. Updated July 22, 2022. Accessed August 3, 2022.