Phase 1 Study of Pacritinib Treatment for Adults with VEXAS Syndrome, a Rare Autoinflammatory Disease

What this Trial Is About

VEXAS syndrome is a newly identified disorder characterized by severe blood and inflammatory symptoms caused by changes in the UBA1 gene in blood-forming cells. Patients often suffer from debilitating autoinflammatory symptoms, and currently, no standard treatment exists. This trial aims to study the safety and tolerability of pacritinib, a drug that blocks certain enzymes, to see if it can improve symptoms in patients with VEXAS syndrome. Participants will receive pacritinib by mouth in cycles lasting 28 days, up to 12 cycles total. The study starts with a safety phase where six patients receive 200 mg twice daily. If the drug is well tolerated, the study will expand to include up to 15 patients. If there are safety concerns, the dose may be lowered to 100 mg twice daily. The study will monitor for dose-limiting toxicities and adjust accordingly. Participants will undergo regular blood tests to monitor white blood cells, hemoglobin, platelets, and inflammation markers at baseline and at 1, 3, 6, and 12 months. Researchers will also assess disease activity and genetic markers linked to VEXAS syndrome. Safety and effectiveness data will be collected throughout the treatment and during a follow-up period lasting up to 24 months. The study is sponsored by Washington University School of Medicine.

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