A new clinical trial testing inebilizumab (Uplizna®) in children with neuromyelitis optica spectrum disorder (NMOSD) is now recruiting participants.

The phase 2, open-label, multicenter study aims to evaluate the safety, pharmacokinetics, and pharmacodynamics of inebilizumab in patients 2 to 17 years of age. 

The study will recruit 15 patients with recently active NMOSD who are seropositive for autoantibodies against aquaporin-4 in the US, UK, Canada, Argentina, Brazil, and Europe.

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Read more about Uplizna  for the treatment of NMOSD 

Participants will receive inebilizumab infusions for 28 weeks over 9 visits. This will be preceded by a 4-week screening period and a 52-week follow-up period during which participants will have to visit the trial site 4 times. The safety of the treatment will be evaluated throughout the study. 

Primary outcome measures include maximum observed concentration, systemic clearance, and terminal elimination half-life of inebilizumab, the number of patients with treatment-emergent events and adverse events of special interest, and change from baseline in serum chemistry and immunoglobulins, hematology, systolic and diastolic blood pressure, pulse and respiratory rates, and body temperature.

Secondary outcome measures include disease activity, health-related quality of life, change from baseline in visual acuity and Expanded Disability Status Scale score, and the rate of antidrug antibodies. 

The trial, which is sponsored by Horizon Therapeutics Ireland, started on August 25, 2022 and is estimated to be completed on September 13, 2024.

Inebilizumab is a cytolytic monoclonal antibody against CD19. CD19-positive B lymphocytes play an important role in the pathogenesis of NMOSD, producing autoantibodies against aquaporin-4,  which target aquaporin-4 found on astrocytes in the central nervous system causing damage. 

The treatment is already approved for the treatment of adult patients with NMOSD and marketed under the brand name Uplizna in the US.


Study of inebilizumab in pediatric subjects with neuromyelitis optica spectrum disorder. US National Library of Medicine. pdated September 22, 2022. Accessed October 11, 2022.