JUL 01, 2020
This phase 3 study assessed the safety and efficacy of satralizumab monotherapy for neuromyelitis optica spectrum disorder (NMOSD).
The double-masked, placebo-controlled, parallel-group trial included 95 patients with at least 1 documented NMOSD attack or relapse in the past 12 months and exhibited a score of 6.5 or less on the expanded disability status scale. Both seropositive and seronegative participants were included using the 2006 Wingerchuk diagnostic criteria. Participants were randomized to either receive satralizumab 120 mg or placebo subcutaneously at weeks 0, 2, 4 and every 4 weeks thereafter, without any concomitant immunosuppressant therapy. Patients taking certain prior treatments or with recent clinical relapse were excluded. The primary endpoint was time to the first protocol-defined relapse.
Nineteen patients receiving medication had a protocol-defined relapse compared with 16 individuals receiving placebo (30% cs. 50%; P=0.018). Pain score and rate of adverse events were comparable between the 2 groups.
The study enrolled both AQP4-IgG seropositive and seronegative patients but limited the seronegative cohort to reflect typical population incidence rates, resulting in a relatively small sample size. Subgroup analysis for the study suggests efficacy for seropositive groups, however the analysis was not powerful enough to study the impact on disease modification in seronegative patients.
To date, there are 3 recent clinical trials for NMOSD: PREVENT (eculizumab, NCT01892345, approved by FDA), N-MOmentum (inebilizumab, NCT02200770), and SAkuraSky (satralizumab in combination with baseline immunosuppressants, NCT02028884). The findings from this study suggest that satralizumab monotherapy may be effective in patients with seropositive NMOSD.