PAC601

Hematological neoplasms
Other indications (e.g. myeloproliferative diseases)
Follow-Up Treatment
This is a randomized, multicenter, double-blind, placebo-controlled phase 2 study (Part 1) followed by an open-label treatment period (Part 2) designed to evaluate the efficacy and safety of pacritinib for the prevention of VEXAS flares after GC taper. The study will enroll patients age >18 years with inflammatory VEXAS syndrome receiving ongoing GC therapy for >4 consecutive weeks, requiring between 15 and 45 mg daily (of prednisone / prednisolone or equivalent) at the time of enrollment (randomization). The study will consist of a screening period, a double-blind treatment period up to End of Week 24, an open-label treatment period through End of Week 48, and a 30-day post-End of Treatment (EOT) follow-up period. Study termination is planned approximately 1 year from the first dose of the last patient.
This is a randomized, multicenter, double-blind, placebo-controlled phase 2 study (Part 1) followed by an open-label treatment period (Part 2) designed to evaluate the efficacy and safety of pacritinib for the prevention of VEXAS flares after GC taper. The study will enroll patients age >18 years with inflammatory VEXAS syndrome receiving ongoing GC therapy for >4 consecutive weeks, requiring between 15 and 45 mg daily (of prednisone / prednisolone or equivalent) at the time of enrollment (randomization). The study will consist of a screening period, a double-blind treatment period up to End of Week 24, an open-label treatment period through End of Week 48, and a 30-day post-End of Treatment (EOT) follow-up period. Study termination is planned approximately 1 year from the first dose of the last patient.