Neuromyelitis Optica Spectrum Disorder Inebilizumab Study in Children and Adolescents
Research type
Research Study
Full title
An Open-label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects with Neuromyelitis Optica Spectrum Disorder
IRAS ID
1004459
Contact name
Stephen Sainati
Contact email
Sponsor organisation
Horizon Therapeutics Ireland DAC
Eudract number
2021-003528-33
Clinicaltrials.gov Identifier
Research summary
Neuromyelitis optica spectrum disorder (NMOSD) is a rare neurological (brain) condition characterised by episodes of optic neuritis (inflammation or swelling of the optic nerve), transverse myelitis (inflammation or swelling of the spinal cord), together with one or more other diagnostic criteria including in some cases the presence of a specific antibody (AQP4). The purpose of this open-label multicentre study is to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of inebilizumab in eligible paediatric participants
2 to < 18 years of age with recently active NMOSD who have AQP4 antibody.Fifteen children between 2 and <18 years of age (at least 1 child from 2 to <6 years of age, at least 3 children from 6 to <12 years of age, and at least 5 children from 12 to <18 years of age) with NMOSD will take part in this study. This study will be conducted at approximately 15 sites around the world.
The study has the following periods: screening period (up to 4 weeks), a treatment period (28 weeks), and a follow-up period (52 weeks). The participants will be in the study for up to 84 weeks. The dose or amount of inebilizumab that the participants will receive will depend on their body weight.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
22/EE/0014
Date of REC Opinion
3 Mar 2022
REC opinion
Further Information Favourable Opinion