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

ConditionsVEXASVEXAS Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorSwedish Orphan Biovitrum

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Documented evidence of a pathogenic mutation at methionine-41 (M41) or 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 in peripheral blood or bone marrow samples.

Current or documented evidence of past inflammatory involvement within 6 months prior to enrollment of at least one of the following organ systems by VEXAS syndrome: cutaneous (e.g., neutrophilic dermatosis, cutaneous vasculitis), vasculature (e.g., vasculitis), musculoskeletal (e.g., chondritis, arthritis), ocular (e.g., uveitis, scleritis), periorbital (e.g. periorbital edema), genitourinary (e.g., epididymitis), or pulmonary (e.g., alveolitis).

Receiving ongoing GC therapy (stable prednisone or prednisolone dose of 15-45 mg/day) leading up to enrollment.

Karnofsky Performance Status ≥50%

Disqualifiers

Prior allogenic hematopoietic stem cell transplant (allo-HSCT) or solid organ transplant (other than corneal).

Current use of systemic GCs for conditions other than VEXAS syndrome, which, in the opinion of the Investigator, would interfere with adherence to a GC taper regimen and/or assessment of efficacy.

More than one prior admission to an intensive care unit due to a VEXAS Syndrome flare within the prior 6 months.

Received ≥9 units of intensive red blood cell (RBC) transfusions in the 90 days prior to enrollment.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Pacritinib

    Drug

    Supplied in hard capsules.

  • Placebo

    Drug

    Supplied in hard capsules.

Treatment groups

78 Participants
are divided into 3 treatment groups
Group A: PacritinibExperimental treatment 1 intervention
Group B: Pacritinib + placeboExperimental treatment 2 interventions
Group C: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

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

Difference in the proportion of participants achieving OCR for the pairwise comparison of pacritinib dose A vs. placebo and pacritinib dose B plus placebo vs. placebo

Time frame
up to End of Week 24

Secondary outcomes

1

Proportion of participants on each arm achieving each Best Response category (Clinical Biochemical Response, Clinical Response, Partial Clinical Response, Stable Disease, or Non-response).

Time frame
up to End of Week 24
2

Number of flare-free days with GC dose <10 mg

Time frame
up to End of Week 24
3

Hematologic Improvement - Erythroid

Erythroid (HI-E) at any time among participants with baseline hemoglobin \<10 g/dL per modified International Working Group (IWG) criteria

Time frame
up to End of Week 24
4

Hematologic Improvement - Platelets

Platelets (HI-P) at any time among participants with baseline platelet count \<100 × 10\^9/L per modified IWG criteria

Time frame
up to End of Week 24

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Swedish Orphan Biovitrum

Lead sponsor

PSI CRO

Collaborator

Source ClinicalTrials.gov
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