RecruitingPhase 2

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

United States78 participantsStarted 2025-05-28

Plain-language 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.

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3 × upper limit of normal (ULN)
✓. Total bilirubin ≤4 × ULN (≤8 × ULN in the setting of Gilbert's syndrome)
✓. Creatinine clearance (CrCl) ≥30 mL/min based on the Cockcroft-Gault formula
✓. Absolute neutrophil count ≥500/μL
✓. Prothrombin time (PT) or international normalized ratio (INR) ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation)
✓. Partial thromboplastic time (PTT) or activated PTT ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation)
✓. Platelet count ≥25 × 10\^9/L (value must be obtained in the absence of platelet transfusion in the prior 7 days)
✓. Peripheral blasts \<5%

Exclusion criteria

✕. QT corrected by the Fridericia method (QTcF) \> 480 msec within 30 days prior to enrollment; if QTcF is thought to be prolonged due to a modifiable factor (e.g., medication / electrolyte abnormality), QTcF may be re-evaluated
✕. Severe cardiac event (CTCAE grade ≥3) within 3 months prior to enrollment
✕. Heart failure resulting in limitations during ordinary activity.

What they're measuring

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

Timeframe: up to End of Week 24

Trial details

NCT IDNCT06782373

SponsorSwedish Orphan Biovitrum

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-12-30

Contact for this trial

Study Physician

+4686972000 medical.info@sobi.com

View on ClinicalTrials.gov More VEXAS trials