PAXIS: A randomized, double-blind, placebo-controlled dose-finding phase 2 study (Part 1) followed by an open-label period (Part 2) to assess the efficacy and safety of pacritinib in patients with VEXAS syndrome

VEXAS Syndrome

1. The primary endpoint is Overall Clinical Response (OCR), defined as achieving Clinical Response or better at any time during the double-blind treatment period.

1. Best Response (Clinical Biochemical Response, Clinical Response, Partial Clinical Response, Stable Disease, or Non-response) during the double-blind treatment period. Note that Stringent Clinical Biochemical Response is not applicable during the double-blind treatment period (i.e., by Week 24) based on the fixed GC taper schedule, 2. Number of flare-free days with GC dose <10 mg during the double-blind treatment period., 3. Hematologic Improvement – Erythroid (HI-E) at any time during the double-blind treatment period among subjects with baseline hemoglobin <10 g/dL, per modified International Working Group (IWG) criteria., 4. Hematologic Improvement – Platelets (HI-P) at any time during the double-blind treatment period among subjects with baseline platelet count <100 × 10^9/L, per modified IWG criteria., 5. Change in health-related QOL as measured by Patient-Reported Outcomes Measurement Information System (PROMIS) short forms (fatigue, physical function, sleep disturbance), 36-Item Short Form Health Survey (SF-36), and the Patient Global Impression of Change (PGIC)., 6. PK of pacritinib., 7. PD inflammatory biomarkers (CRP, erythrocyte sedimentation rate [ESR], ferritin), 8. Safety and tolerability, assessed by adverse events (AEs), laboratory tests, electrocardiogram (ECG) results, and vital signs will be assessed throughout the double-blind and open-label treatment periods.

France, Germany, Italy, Spain