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Experimental drug pacritinib takes aim at rare inflammatory VEXAS syndrome

NCT ID NCT06538181

Recruiting now Disease control Sponsor: Washington University School of Medicine Source: ClinicalTrials.gov ↗

First seen Jul 15, 2026 · Last updated Jul 16, 2026 · Updated 1 time

Summary

This phase 1 trial tests the safety and tolerability of pacritinib, a pill that blocks JAK2 and IRAK1, in people with VEXAS syndrome—a rare, severe autoinflammatory disorder caused by a UBA1 gene mutation. Up to 15 participants will take pacritinib daily for up to 12 cycles, starting at 200 mg twice daily, with a lower dose option if needed. The study aims to find the best dose for future trials and to see if the drug can improve inflammatory and blood-related symptoms.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
pacritinib, a JAK2/IRAK1 inhibitor taken as a pill
What this could lead to
If successful, this could point toward a first approved treatment for VEXAS syndrome, reducing inflammation and blood-related problems.
What could go wrong
This is a very early, small phase 1 trial with only 15 participants, so results may not apply broadly. The drug may cause side effects or fail to improve symptoms.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

VEXAS syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

E1 UBIQUTIN-ACTIVATING ENZYME, X-LINKED, AUTOINFLAMMATORY, SOMATIC SYNDROME VEXAS VEXAS SYNDROME

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Washington University School of Medicine

    RECRUITING

    St Louis, Missouri, 63110, United States

    Contact

    Contact

    Contact

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

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