Study of Pacritinib to Evaluate Safety and Effectiveness in Adults With VEXAS Syndrome Randomized, Double-blind, Placebo-controlled Phase 2 Trial with Open-label Extension

What this Trial Is About

Researchers are evaluating the effectiveness and safety of pacritinib in people with VEXAS syndrome, a rare inflammatory condition caused by specific genetic mutations. This randomized, placebo-controlled phase 2 trial aims to prevent flares of VEXAS symptoms after reducing glucocorticoid GC treatment. The study will include adults aged 18 and older who have been on stable GC therapy for at least four weeks and have inflammatory involvement of various organs linked to VEXAS syndrome. Participants will be randomly assigned to one of three groups pacritinib dose A, pacritinib dose B plus placebo, or placebo alone, all taken orally for up to 24 weeks without knowing which they receive. After this double-blind period, all participants will have the option to receive open-label pacritinib for up to 48 weeks. Those benefiting from pacritinib may continue treatment for an additional year in an extension period. If a participant stops treatment, they will be followed for 30 days after. Throughout the study, participants will have regular assessments to monitor clinical response, flare frequency, blood parameters, and quality of life using patient-reported questionnaires. Blood tests will measure drug levels and inflammation markers like C-reactive protein, erythrocyte sedimentation rate, and ferritin. The main outcome is overall clinical response by week 24. Safety and efficacy data will be collected until about two years after the first dose of the last participant.

A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)