VEXAS syndrome

Displaying 2 studies

  • VEXAS Biobank Rochester, MN

    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

     

  • A study to assess the effectiveness and safety of pacritinib in patients with VEXAS syndrome (PAXIS) Scottsdale/Phoenix, AZ; Rochester, MN

    The purpose of this stduy is to evaluate the efficacy of two dose levels of pacritinib compared to placebo during the double-blind treatment period in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzyme, X-linked, autoinflammatory manifestations, and somatic) syndrome.

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