Citations 1
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
Beck, David B. , Heiblig, Maël , Savic, S., Ferrada, Marcela A. , Mekinian, Arsène, Chowdhury, Onima , Hammond, Danielle E., Weeks, Lachelle D., Gurnari, Carmelo , Kirino, Yohei , Georgin-Lavialle, Sophie, Buckley, Sarah A., Garcha, Raman, Harder, Bryan G., Harder, Bryan , Weeks, Lachelle D. , Mékinian, A. , Savic, Sinisa , Hammond, Danielle , Georgin‐Lavialle, Sophie , Savic, Sinisa , Mékinian, Arsène , Hammond, Danielle , Weeks, Lachelle Dawn , Georgin-Lavialle, Sophie , Buckley, Sarah A , Harder, Bryan G , Koster, Matthew J , Gurnari, Carmelo , Kirino, Yohei , Heiblig, Mael , Ferrada, Marcela A , Koster, Matthew J.
Journal of Clinical Medicine | February 11, 2026 | Volume: 15 | Issue: 4 | Pages: 1426
VEXAS (Vacuoles, E1 ubiquitin-activating enzyme, X-linked, Autoinflammatory, Somatic) syndrome is a systemic disorder characterized by an overlap of hematologic and inflammatory features. Most patients require chronic use of moderate-to-high doses of glucocorticoids (GCs) to maintain disease control. Data on GC-sparing therapies is limited, and there have been no prospective pharmacotherapeutic trials in VEXAS syndrome published to date. Pacritinib, an oral inhibitor of IRAK1, JAK2, and ACVR1, has emerged as a promising therapeutic option for VEXAS syndrome. The PAXIS trial is the first prospective, randomized pharmacotherapeutic study conducted in this rare and severe disease. Utilizing a novel study design and disease-specific endpoints, the trial will evaluate the efficacy and safety of two dose levels of pacritinib compared with placebo in patients with VEXAS syndrome (NCT06782373, EUCTR: 2024-516347-41-00).