Clinical Trials

The objective of this study is to develop a biorepository of venous peripheral blood samples to carry out future multi-omic research on patients with VEXAS syndrome.

An adult auto-inflammatory syndrome caused by somatic mutations in the bone marrow myeloid precursors in the bone marrow has been recently identified and described by the National Institutes of Health (NIH). This condition has been termed VEXAS syndrome – which stands for Vacuoles, E1 enzyme, X-linked, Auto-inflammatory, Somatic

This trial is to assess the effectiveness and safety of pacritinib in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzyme, X-linked, autoinflammatory manifestations, and somatic) syndrome. In phase 2a, approximately 78 participants will be randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. In Phase 2b, approximately 78 participants will be randomized. Phase 2b participants will initially be randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. After dose selection, Phase 2b further participants will be randomized to either the selected pacritinib dose or placebo.

Randomization will be stratified by ...