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

Required: UBA1 pathogenic mutation at methionine-41 (M41)

pathogenic mutation at methionine-41 (M41) ... in UBA1 mutation based on myeloid next-generation sequencing (NGS) droplet digital polymerase chain reaction (ddPCR), or Sanger sequencing

Required: UBA1 neighboring splice site mutation (c.118-1, c.118-2)

neighboring splice site mutation (c.118-1, c.118-2) position in UBA1 mutation based on myeloid next-generation sequencing (NGS) droplet digital polymerase chain reaction (ddPCR), or Sanger sequencing

Must have received: glucocorticoid (prednisone, prednisolone) — ongoing

Receiving ongoing GC therapy (stable prednisone or prednisolone dose of 15-45 mg/day) leading up to enrollment

Cannot have received: allogenic hematopoietic stem cell transplant

Prior allogenic hematopoietic stem cell transplant (allo-HSCT)

Cannot have received: solid organ transplant

Exception: corneal transplant allowed

solid organ transplant (other than corneal)

Cannot have received: hypomethylating agent

Exposure to hypomethylating agents (HMA) within 6 months prior to enrollment, or exposure to more than 6 cycles of HMAs at any time

Cannot have received: non-GC anti-inflammatory therapy

Exposure to non-GC anti-inflammatory therapy or hematologic support therapy within protocol defined timeframes prior to enrollment

Cannot have received: anti-platelet therapy

Exception: low-dose aspirin (≤100 mg daily) allowed

Exposure to anti-platelet therapy with the exception of low-dose aspirin (≤100 mg daily) within 28 days prior to enrollment

Cannot have received: experimental therapy

treatment with an experimental therapy within 28 days or five half-lives prior to enrollment, whichever is longer