Welcome to the PAXIS Trial Information Portal

The PAXIS Trial is an Investigational Clinical Trial in VEXAS Syndrome. This website is an educational resource for health care professionals to learn more about the trial and to determine the eligibility for patient enrollment.

Research Overview

The PAXIS Trial is an international, randomized, multicenter, double-blind, placebo-controlled phase 2 study designed to assess pacritinib in patients with VEXAS syndrome.*

PAXIS Trial Objectives:

Primary Objective

  • To evaluate the efficacy of two dose levels of pacritinib compared to placebo during the double-blind treatment period in patients with VEXAS syndrome by measuring the rate of overall clinical response (OCR), defined as having a flare-free interval ≥8 consecutive weeks following a glucocorticoid (GC) taper. The GC dose during the flare-free interval must be ≤10 mg/day

Secondary Objectives

  • To evaluate hematologic improvement (HI)
  • To evaluate change in health-related quality of life (HR-QoL)
  • To evaluate pharmacokinetics and pharmacodynamics
  • To evaluate safety and tolerability
Study Design & Eligibility Criteria

PAXIS Trial Site Locations

Enrollment is ongoing / proposed in the countries below. For a full list of PAXIS clinical sites and site enrollment status, visit ClinicalTrials.gov and EU CTIS.

PAXIS clinical trial map
Canada
France
Germany
Italy
Japan
Spain
United Kingdom
United States of America

*This trial is evaluating the investigational use of pacritinib in VEXAS syndrome. The safety and efficacy of pacritinib in VEXAS syndrome has not been established. Pacritinib is not approved by any Health Authority for this use.

Pacritinib (commercially known as VONJO®) was approved by U.S. Food and Drug Administration (FDA) on February 28, 2022 for the treatment of adults with myelofibrosis with a platelet count below 50 x 109/L, and has since been marketed in the US under the brand name VONJO®. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).