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Pacritinib for VEXAS Syndrome

This study is testing a medication called pacritinib for people with VEXAS syndrome (vacuoles, E1 ubiqutin-activating enzyme, X-linked, autoinflammatory, somatic syndrome). VEXAS is a recently identified condition causing severe blood and rheumatologic problems, and there isn't a standard treatment yet. Researchers want to see if pacritinib is safe and what dose works best. You might be able to join if you are 18 or older, have a specific genetic change (UBA1 mutation) detected in your blood, and have symptoms like skin rash, vasculitis (blood vessel inflammation), chondritis (cartilage inflammation), or eye inflammation. The study will look at how many participants experience side effects and help determine the recommended dose for future studies. This study is currently recruiting about 15 participants.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll about 15 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will track participants for dose-limiting toxicities and recommended dose determination through the completion of cycle 1, which is estimated to be 28 days.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06538181

Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome

Recruiting
PHASE1Ages 18+InterventionalTreatment
Washington University School of Medicine
~15 participants
Updated 2026-07-15 on ClinicalTrials.gov
What's tested:Pacritinib

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of participants with dose-limiting toxicities (DLTs)
Measured over Through completion of cycle 1 (estimated to be 28 days)
+1 more outcome measured

Conditions

E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome
VEXAS
Vexas Syndrome

Explore related trials

VEXAS SyndromeUbiquitin-Like Modifier-Activating Enzyme 1

Where it's being run

1 sites across 1 states
Missouri1

Study leadership

  • Meagan A Jacoby, M.D., Ph.D. · PRINCIPAL_INVESTIGATOR · Washington University School of Medicine

Who to contact

Meagan A Jacoby, M.D., Ph.D.
[email protected]
314-454-8306
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Patients must have UBA1 mutation with a variant allele frequency (VAF) of ≥ 2% detected on a next generation sequencing panel and have at least one of the following current or past clinical manifestation of VEXAS syndrome, as determined by the attending physician:
skin rash
vasculitis
chondritis
ocular/orbital inflammation (e.g., uveitis/iritis, episcleritis)
genitourinary inflammation (e.g., epididymitis/orchitis)
arthritis/arthralgias
pulmonary inflammation (e.g., alveolitis/pleural effusion,)
fever
thrombosis
splenomegaly
hepatomegaly
myocarditis or pericarditis
cytopenias (defined as hemoglobin \<11 g/dL, platelets \< 100 X 10\^9 /L, OR absolute neutrophil count \<1.0 X 10\^9 /L).
Patients with VEXAS syndrome who have never been treated with a JAK-I will be eligible to enroll on study. A stable corticosteroid dose must be maintained for at least 14 days prior to start of pacritinib.
Patients who have previously been treated with a JAK-I other than pacritinib, or who are currently being treated with a JAK-I other than pacritinib, may be eligible after a 28 day washout if either (i) their symptoms are not adequately controlled, as determined by the treating physician, or (ii) they have been unable to taper corticosteroids to an equivalent of \<10 mg prednisone/day, and in the opinion of the treating physician, may benefit from a change in JAK-I. A stable corticosteroid dose must be maintained for at least 14 days prior to start of pacritinib.
At least 18 years of age.
ECOG performance status ≤ 3.
Organ function as defined below:
Absolute neutrophil count ≥ 0.5 K/cumm
Platelets ≥ 25 K/cumm
PT/PTT \<2.5 X upper limit of normal (ULN)
Total bilirubin ≤ 1.5 x IULN
AST(SGOT)/ALT(SGPT) ≤ 3.0 x IULN
Creatinine clearance ≥ 30 mL/min by Cockcroft-Gault
QTcF \< 480 msec.
The effects of pacritinib on the developing human fetus are unknown. For this reason and because pacritinib was shown to be teratogenic in animal studies, women of childbearing potential and men must agree to use highly effective contraception prior to study entry, for the duration of study participation, and for 30 days after completion of study treatment. Hormonal contraception is no longer considered highly effective alone as pacritinib is a CYP3A4 inducer and accelerated progesterone metabolism. The contraceptive methods considered highly effective for WOCBP who receive pacritinib are intrauterine devices, bilateral tubal occlusion, vasectomized partner, or total sexual abstinence. Hormonal contraceptives (e.g., Depo-Provera) alone are not considered highly effective methods of contraception on their own when in treatment with pacritinib; such hormonal contraceptives must be combined with an additional barrier method (condom, diaphragm with spermicidal gel, or condoms with spermicides to be considered highly effective. Highly effective contraceptive methods in males include vasectomy, sexual abstinence, and condoms when combined with their partner using a highly effective method (including oral contraceptives).
Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.
Ability to understand and willingness to sign an IRB approved written informed consent document or that of legally authorized representative, if applicable.
Patients with myelodysplastic neoplasms (MDS) or plasma cell dyscrasias are eligible if they are not undergoing active treatment. Supportive care is permitted.

Exclusion

Prior use of pacritinib.
Use of another JAK inhibitor within 28 days of C1D1 of pacritinib.
Currently receiving any other investigational agents. Patients may be eligible after 28 day washout.
Thrombotic events (arterial or venous) within 60 days prior to enrollment.
Any recent clinically significant bleeding within at least 7 days prior to enrollment.
Any active or acute infection.
History of malignancy within the prior 2 years, with the exception of MDS and plasma cell dyscrasias, or non-melanoma skin cancers that have been treated.
History of clinically significant cardiovascular disease or clinically significant abnormalities in rhythm or conduction during screening EKG, including severe cardiac events, symptomatic congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmia, or heart failure.
Currently receiving immunosuppressants (other than corticosteroids), disease-modifying antirheumatic drugs (DMARDs), or biologic cytokine inhibitors. Patients may be eligible after 28 day washout.
A history of allergic reactions attributed to compounds of similar chemical or biologic composition to pacritinib.
Concurrent use of strong CYP3A4 inhibitors or inducers. Patients may be eligible after washout period of 28 days (or 5 half-lives, whichever is shorter).
Diagnosis or history of moderate (Child-Pugh B) and severe hepatic impairment (Child-Pugh C).
Pregnant and/or breastfeeding. Women of childbearing potential must have a negative serum pregnancy test within 7 days of C1D1 or negative urine pregnancy test within 3 days of C1D1.
Known active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV).
Patients with latent tuberculosis. Patients must have a negative T-Spot during screening to be eligible.

What this trial measures

  • Number of participants with dose-limiting toxicities (DLTs)Through completion of cycle 1 (estimated to be 28 days)

    Dose-limiting toxicities (DLTs) are adverse events defined by the protocol that occur during the DLT observation period (Cycle 1) of the study, unless clearly attributable to underlying disease or another cause unrelated to the study.

  • Recommended phase II dose (RP2D)Through completion of cycle 1 (each cycle is 28 days) for all treated participants

    The maximum tolerated dose (MTD) is defined as the dose level immediately below the dose level at which 2 patients of a cohort (of 2 to 6 patients) experience dose-limiting toxicity (DLT) during the first cycle. The recommended phase II dose (RP2D) will be less than or equal to the MTD. If the MTD is not reached, and in the opinion of the investigators, the toxicity profile is acceptable, Dose Level 1 will be the RP2D.