OncoMatch/Clinical Trials/NCT06782373
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
Official title: PAXIS: A Phase 2a and 2b Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy and Safety of Pacritinib in Patients With VEXAS Syndrome
Is NCT06782373 recruiting? Yes, currently enrolling (Oct 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. In phase 2a, approximately 78 participants will be randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. In Phase 2b, approximately 78 participants will be randomized. Phase 2b participants will initially be randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. After dose selection, Phase 2b further participants will be randomized to either the selected pacritinib dose 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 m41 mutation
Required: UBA1 splice site mutation (c.118-1, c.118-2)
Prior therapy
Must have received: glucocorticoid therapy (prednisone, prednisolone)
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: other than corneal
Prior ... solid organ transplant (other than corneal)
Cannot have received: hypomethylating agent
Exception: more than 6 cycles at any time
Exposure to ... more than 6 cycles of HMAs at any time
Cannot have received: concurrent or prior enrollment in another prospective interventional trial of VEXAS-directed therapy
Concurrent or prior enrollment in another prospective interventional trial of VEXAS-directed therapy
Cannot have received: concurrent enrollment in another interventional trial of non-VEXAS-directed therapy
concurrent enrollment in another interventional trial of non-VEXAS-directed therapy
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 >100 msec allowed if QTcF ≤480 msec; ventricular paced rhythms allowed if QTcF ≤500 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 Gilbert's syndrome); CrCl ≥30 mL/min; ANC ≥500/μL; PT/INR ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation); PTT/aPTT ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation); Platelet count ≥25 × 10^9/L (no platelet transfusion in prior 7 days); Peripheral blasts <5%. QTcF ≤450 msec in males or ≤470 msec in females. QRS >100 msec allowed if QTcF ≤480 msec. Ventricular paced rhythms allowed if QTcF ≤500 msec.
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- Mayo Clinic - Scottsdale · Scottsdale, Arizona
- Dana Farber Cancer Institute · Boston, Massachusetts
- Mayo Clinic - Rochester · Rochester, Minnesota
- NYU Langone Health · New York, New York
- Cleveland Clinic - Cleveland · Cleveland, Ohio
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 m41 mutation is a required biomarker for enrollment.
Does this trial require UBA1?
Yes, UBA1 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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