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)

Launched by HOFFMANN-LA ROCHE · Jan 5, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a medication called satralizumab in children aged 2 to 11 years who have a condition known as neuromyelitis optica spectrum disorder (NMOSD). NMOSD can cause inflammation in the nervous system and is linked to certain antibodies in the body. The trial aims to understand how satralizumab works in these young patients, including how it is processed by the body, its effectiveness in reducing symptoms, and its safety. Since the study involves a small number of participants, they will also evaluate how well children tolerate the treatment.

To be eligible for this study, children must be between 2 and 11 years old and weigh at least 10 kilograms. They should have been diagnosed with NMOSD and have experienced at least one attack in the previous year. Additionally, they need to be stable in their health for at least 30 days before joining the trial. Participants can expect to receive careful monitoring and support throughout the study. It’s important to note that certain conditions, such as other types of infections or a history of severe allergic reactions, may prevent participation. This trial is currently accepting participants.

Gender

ALL

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