CI

At a glance

ClinicalIndex Comparison Record
Phase 2Recruiting· 78 target
Drug / intervention
Pacritinib +1 moredrug
Likely dose
Not stated in record
Key inclusion· 5
  • ✓Pathogenic UBA1 mutation at M41 or neighboring splice site (c.118-1, c.118-2) in peripheral blood or bone marrow
  • ✓Current or documented inflammatory involvement within 6 months in at least one organ system: cutaneous, vasculature, musculoskeletal, ocular, periorbital, genitourinary, or pulmonary
  • ✓Receiving ongoing stable glucocorticoid therapy with prednisone or prednisolone 15-45 mg/day
  • ✓Women of childbearing potential must have negative serum pregnancy test within 30 days and negative urine pregnancy test on Day 1
Key exclusion· 21
  • ✕Prior allogeneic hematopoietic stem cell transplant or solid organ transplant (except corneal)
  • ✕Current systemic glucocorticoids for conditions other than VEXAS syndrome that would interfere with GC taper or efficacy assessment
  • ✕More than one prior ICU admission for VEXAS flare within 6 months
  • ✕≥9 units of RBC transfusions in prior 90 days

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT06782373
NCT06782373Phase 2RecruitingOn TrackUpdated 2mo ago
CompareCT.gov

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

Swedish Orphan Biovitrum·interventional·Posted Jan 17, 2025·Updated Apr 24, 2026

In Brief

A Phase 2 clinical trial evaluating Pacritinib and Placebo for VEXAS and VEXAS Syndrome. Currently recruiting, targeting 78 participants across 39 sites in 8 countries.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsVEXAS, VEXAS Syndrome
CountriesCanada, France, Germany, Italy, Japan, Spain, United Kingdom, United States
CollaboratorsPSI CRO

Timeline

Phase 2Recruiting
2025202620272028
First PostedJan 17, 2025
Enrollment StartMay 28, 2025
Primary CompletionDec 30, 2026
Study CompletionMay 22, 2028
TodayJul 2, 2026
Enrollment to primary: 1.6 yearsPosted 1.5 years agoPrimary completion in 6 months

Interventions

Pacritinibdrug

Supplied in hard capsules.

Placebodrug

Supplied in hard capsules.