Actively Recruiting
A Phase 1 Study of Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome
Led by Washington University School of Medicine · Updated on 2025-07-16
15
Participants Needed
1
Research Sites
100 weeks
Total Duration
On this page
Sponsors
W
Washington University School of Medicine
Lead Sponsor
S
Swedish Orphan Biovitrum
Collaborating Sponsor
AI-Summary
What this Trial Is About
VEXAS syndrome is a recently identified disorder caused by changes in the UBA1 gene in blood-forming cells, leading to severe blood and inflammation problems. Patients often suffer from disabling inflammatory symptoms and currently have no standard treatments. Researchers are studying pacritinib, a drug that blocks specific enzymes (JAK2 and IRAK1), to see if it can safely reduce symptoms and improve blood counts in people with this syndrome. This phase 1 study involves a single group of patients treated with pacritinib by mouth twice daily in 28-day cycles for up to 12 cycles. The study starts with a small safety phase where 6 patients receive a 200 mg twice daily dose. If this dose is well tolerated, more patients will join to gather more safety and effectiveness data. If too many side effects occur, the dose may be lowered to 100 mg twice daily or the study may be stopped. Participants will be closely monitored throughout the treatment cycles with regular blood tests to measure blood cells, inflammation markers, and gene changes. Researchers will track side effects and improvements in symptoms using the VEXAS Disease Activity Index. After treatment, participants will be followed for up to 24 months to observe the time until next treatment is needed. The total study duration varies but includes treatment and long-term follow-up periods to assess safety and benefits.
CONDITIONS
Brief Title
Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must have a UBA1 gene mutation with a variant allele frequency of 2% or higher detected by next generation sequencing
- Must have at least one current or past VEXAS-related symptom such as skin rash, vasculitis, chondritis, eye inflammation, genitourinary inflammation, arthritis, lung inflammation, fever, blood clots, enlarged spleen or liver, heart inflammation, or low blood counts
- Patients who have never been treated with a JAK inhibitor or have had a washout period of 28 days if previously treated with a different JAK inhibitor (except pacritinib)
- Stable corticosteroid dose for at least 14 days before starting pacritinib
- At least 18 years old
- ECOG performance status 3 or less
- Adequate organ function including specific blood counts, liver, and kidney function
- QTcF less than 480 msec
- Agree to use effective contraception during and for 30 days after the study if of childbearing potential or male
- Ability to understand and sign informed consent
- Patients with myelodysplastic neoplasms or plasma cell disorders not on active treatment are eligible
You will not qualify if you...
- Prior use of pacritinib
- Use of other JAK inhibitors within 28 days before starting pacritinib
- Currently receiving other investigational drugs without a 28-day washout
- Blood clots within 60 days before enrollment
- Recent significant bleeding within 7 days before enrollment
- Active or acute infections
- History of cancer within 2 years except certain blood disorders or treated non-melanoma skin cancer
- Significant heart disease or abnormal heart rhythm
- Use of immunosuppressants (other than corticosteroids), DMARDs, or biologics without a 28-day washout
- Allergy to pacritinib or similar compounds
- Use of strong CYP3A4 inhibitors or inducers without washout
- Moderate or severe liver impairment
- Pregnant or breastfeeding women; negative pregnancy test required for women of childbearing potential
- Active HIV, hepatitis B, or hepatitis C infection
- Latent tuberculosis (must test negative)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 12 cycles of 28 days each
Participants receive pacritinib by mouth twice per day in repeated 28-day cycles. They can receive up to 12 cycles of treatment.
Visits at baseline and monthly during treatment for assessments
Duration - Up to 12 months after treatment completion
Participants are monitored for safety and treatment effects after completing treatment.
Follow-up visits approximately monthly up to 12 months
Trial Site Locations
Total: 1 location
1
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
Research Team
M
Meagan A Jacoby, M.D., Ph.D.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here