OncoMatch/Clinical Trials/NCT06782373
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
Is NCT06782373 recruiting? Yes, currently enrolling (Jun 2026). This Phase 2 trial studies Pacritinib for vexas.
Treatment: Pacritinib — 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.
Check if I qualifyExtracted eligibility criteria
Treatments studied
Targeted therapy
Biomarker criteria
Required: UBA1 pathogenic mutation at methionine-41 (M41)
pathogenic mutation at methionine-41 (M41) ... in UBA1 mutation based on myeloid next-generation sequencing (NGS) droplet digital polymerase chain reaction (ddPCR), or Sanger sequencing
Required: UBA1 neighboring splice site mutation (c.118-1, c.118-2)
neighboring splice site mutation (c.118-1, c.118-2) position in UBA1 mutation based on myeloid next-generation sequencing (NGS) droplet digital polymerase chain reaction (ddPCR), or Sanger sequencing
Prior therapy
Must have received: glucocorticoid (prednisone, prednisolone) — ongoing
Receiving ongoing GC therapy (stable prednisone or prednisolone dose of 15-45 mg/day) leading up to enrollment
Cannot have received: allogenic hematopoietic stem cell transplant
Prior allogenic hematopoietic stem cell transplant (allo-HSCT)
Cannot have received: solid organ transplant
Exception: corneal transplant allowed
solid organ transplant (other than corneal)
Cannot have received: hypomethylating agent
Exposure to hypomethylating agents (HMA) within 6 months prior to enrollment, or exposure to more than 6 cycles of HMAs at any time
Cannot have received: non-GC anti-inflammatory therapy
Exposure to non-GC anti-inflammatory therapy or hematologic support therapy within protocol defined timeframes prior to enrollment
Cannot have received: anti-platelet therapy
Exception: low-dose aspirin (≤100 mg daily) allowed
Exposure to anti-platelet therapy with the exception of low-dose aspirin (≤100 mg daily) within 28 days prior to enrollment
Cannot have received: experimental therapy
treatment with an experimental therapy within 28 days or five half-lives prior to enrollment, whichever is longer
Lab requirements
Blood counts
Absolute neutrophil count ≥500/μL; Platelet count ≥25 × 10^9/L (no platelet transfusion in prior 7 days); Peripheral blasts <5%
Kidney function
Creatinine clearance (CrCl) ≥30 mL/min based on Cockcroft-Gault formula
Liver function
AST and ALT ≤3 × ULN; total bilirubin ≤4 × ULN (≤8 × ULN in Gilbert's syndrome)
Cardiac function
QTcF ≤450 msec in males or ≤470 msec in females; QRS prolongation >100 msec allowed if QTcF ≤480 msec; PT/INR ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation); PTT/aPTT ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation)
Adequate organ function, meeting all the following criteria within 30 days prior to enrollment: AST and ALT ≤3 × 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%
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- Mayo Clinic - Scottsdale · Scottsdale, Arizona
- University of Maryland Medical Center Midtown Campus · Baltimore, Maryland
- Dana Farber Cancer Institute · Boston, Massachusetts
- Mayo Clinic - Rochester · Rochester, Minnesota
- NYU Langone Health · New York, New York
Showing up to 5 US sites.
See all sites on ClinicalTrials.gov →Frequently asked questions
Is NCT06782373 currently recruiting?
Yes, this trial is currently recruiting patients.
Are there prior therapy exclusions?
Yes. Prior allogenic hematopoietic stem cell transplant, solid organ transplant, hypomethylating agent disqualifies patients from enrollment.
Does this trial require UBA1?
Yes, UBA1 pathogenic mutation at methionine-41 (M41) is a required biomarker for enrollment.
Does this trial require UBA1?
Yes, UBA1 neighboring splice site mutation (c.118-1, c.118-2) is a required biomarker for enrollment.
Could you qualify for this trial?
Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.
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