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

Summary

This trial is to assess the effectiveness and safety of pacritinib in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzyme, X-linked, autoinflammatory manifestations, and somatic) syndrome. 78 participants will be enrolled, randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. Randomization will be stratified by prescribed GC dose on the day of randomization.

Timeline

Start

2025-05-28

Primary completion

2026-12-30

Completion

2028-05-22

Publications

• Beck DB, Heiblig M, Savic S, Ferrada MA, Mekinian A, Chowdhury O, Hammond D, Weeks LD, Gurnari C, Kirino Y, Georgin-Lavialle S, Buckley SA, Garcha R, Harder BG, Koster MJ. 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. J Clin Med. 2026 Feb 11;15(4):1426. doi: 10.3390/jcm15041426.

Drugs

Indications