Alexion Pharmaceuticals is conducting a Phase 2/3 study to evaluate the safety and efficacy of eculizumab (Soliris) specifically in pediatric participants with relapsing neuromyelitis optica spectrum disorder (NMOSD).
This study is an interventional, open-label, single-arm trial for pediatric individuals aged 2 to 17 years with anti-aquaporin-4 antibody-positive NMOSD. It is open to participants in Japan, Korea, Spain, and the United States. All locations are currently recruiting except Korea at the time of this writing.
Dosing of eculizumab will be based on the participant’s body weight, and as such, as weight may change, so may the participant’s weight cohort and dosing. Beginning with a weight-based weekly dose of eculizumab during an induction phase, participants will receive intravenous infusion every two weeks during the Primary Treatment Period for a total of 52/53 weeks. After completing the Primary Treatment Period, participants may continue to receive eculizumab in the Extension Treatment Period for 104 weeks.
Notable inclusion criteria in addition to the anti-AQP4 antibody positivity and a confirmed NMOSD diagnosis, participants must be age 2 to 17 years (must be less than 18 years) and with a bodyweight greater than or equal to 10 kilograms (or 22.0462 pounds). Participants should have experienced at least two relapses in the past two years, with at least 1 of the relapses occurring in the past year before screening. Those who participate in the study and also receive supportive immunosuppressive therapies to prevent relapse, whether in combination or as a single therapy, must be on a stable dosing regimen for a long enough amount of time.
Exclusion criteria include those with unresolved or chronic infections that have not been appropriately treated with antibiotics, or prior to screening have used rituximab or other biologicals within six months, mitoxantrone within three months, IVIG or plasma exchange (PLEX) within three weeks, or immunomodulatory therapies for multiple sclerosis (MS) within three months.