To prospectively study the clinical performance of ravulizumab, enhance knowledge on conventional MRI outcomes, assess for occult disease activity via contemporary imaging metrics, identify serum biomarkers related to disease progression, and to explore contemporary social factors that may impact neurological outcomes in people with neuromyelitis optica spectrum disorder (NMOSD).

NMOSD is a rare autoimmune condition of the central nervous system with aggressive involvement of the optic nerves and spinal cord caused by injury to aquaporin-4 water channels. Exacerbations are unpredictable and result in the accumulation of permanent disability. A comprehensive understanding of ideal practices for treatment and disease surveillance is needed to improve clinical outcomes. All pivotal trials involving FDA-approved treatments lacked comprehensive neuroimaging data capable of detecting neuroinflammatory or neurodegenerative activity below the resolution of conventional techniques, features that may relate to impending disease activity. The effectiveness of ravulizumab on such outcomes remains underexplored.

This single-center, observational, 78-week trial will include 35 subjects who fulfill the 2015 International Panel Criteria for aquaporin-4 immunoglobulin G positive NMOSD. General physical examinations, Expanded Disability Status Scale (EDSS) assessments, conventional and novel MRI sequences, and the collection of biological samples for biomarker analysis will be performed at baseline, and at intervals of 13-, 26-, 52-, and 78-weeks post-initiation of treatment with ravulizumab. Innovative social and environmental factors influencing health outcomes from patient reported data and external digital resources will also be captured and analyzed throughout the duration of the study.

Enrollment for this ongoing study began in July 2024 (ClinicalTrials.gov: NCT06398158). Preliminary data to be presented.

Following the completion of this study, more knowledge will be gained about the biological effects of ravulizumab on clinical, serological, and radiological measures of disease, providing additional data that will guide clinicians towards better practices aimed at optimizing care.