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Phase 1
A Phase 1 Study of Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome
VEXAS (vacuoles, E1 ubiqutin-activating enzyme, X-linked, autoinflammatory, somatic syndrome) is a recently described disorder with severe hematologic and rheu…
E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic SyndromeVEXASVexas Syndrome
Washington University School of MedicineNCT06538181
Natural History and Genetics of VEXAS Syndrome and Related Autoinflammatory Syndromes
This is an exploratory natural history protocol that will enroll patients with known genetic diseases, such as VEXAS syndrome, or as yet undiagnosed disorders …
VEXAS SyndomeAutoinflammatory DiseaseAutoimmune Disease+1 more
NYU Langone HealthNCT06004349
Phase 2
PAXIS: A Randomized, Double-blind, Placebo-controlled Dose-finding Phase 2 Study (Part 1) Followed by an Open-label Period (Part 2) to Assess the Efficacy and Safety of Pacritinib in Patients With VEXAS Syndrome
This trial is to assess the effectiveness and safety of pacritinib in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzym…
VEXASVEXAS Syndrome
Swedish Orphan BiovitrumNCT06782373