Study Details
Testing the Drug Pacritinib in People with VEXAS syndrome
(IRB#: IRB_00186453)
VEXAS syndrome is a severe disorder that affects the immune system making it hard to protect against infections. This study will test an already approved drug for other disorders called pacritinib to treat people with VEXAS syndrome. The study wants to see if the drug will help people with the syndrome and is safe. People in the study will be randomly chosen to participate in one of three groups to receive 200 mg pacritinib twice daily, 100 mg pacritinib + 1 placebo capsule twice daily, or 2 placebo capsules twice daily. A placebo looks like the study drug but does not have the medication. People in the study will write in a diary and complete surveys. Being in the study requires attending in person study visits at the study clinic for up to 52 weeks. Medical tests will be done during the study to track the health of participants.
- All genders
- Over 18 years old
- Volunteers with special conditions
- In Person
- Paid
Who can participate?
Gender: All genders
Age: Over 18 years old
Volunteers: Volunteers with special conditions
Location: In Person
Inclusion Criteria
- Ages 18 years and older
- Confirmed or likely diagnosis of VEXAS syndrome
- Currently experiencing a symptom of VEXAS syndrome, or have experienced symptoms within the last 6 months before participation
- Receiving ongoing treatment with steroids for VEXAS syndrome at least 4 weeks before participation
- Able to attend in person visits at the study clinic
Exclusion Criteria
- Prior all-HSCT (healthy blood forming cells from a donor are infused into a person to replace damaged cells) or solid organ transplant (other than corneal, the front part of the eye)
- More than one prior admission to an intensive care unit due to a VEXAS Syndrome issue within 6 months before participation
- Specific types of Malignancy within 1 year before participation
- Unstable disease
- Pregnant or breastfeeding
Will I be paid for my time?
Yes
IRB#: IRB_00186453
PI: Gopi Penmetsa
Department: INTERNAL MEDICINE
Approval Date: 2025-05-28 06:00:00
Specialties: Hematology/BMT, Rheumatology
I am Interested