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Mael Heiblig practices practicing medicine in Lyon, France. Mr. Heiblig is rated as an Elite expert by MediFind in the treatment of VEXAS Syndrome. He is also highly rated in 8 other conditions, according to our data. His clinical expertise encompasses VEXAS Syndrome, Acute Myeloid Leukemia (AML), Leukemia, Myelodysplastic Syndrome (MDS), and Bone Marrow Transplant.
Arsene Mekinian practices practicing medicine in Paris, France. Mr. Mekinian is rated as an Elite expert by MediFind in the treatment of VEXAS Syndrome. He is also highly rated in 47 other conditions, according to our data. His clinical expertise encompasses VEXAS Syndrome, Takayasu Arteritis, Vasculitis, Bone Marrow Transplant, and Intrauterine Device Insertion.
New York University
David Beck is a Medical Genetics specialist and a Rheumatologist practicing medicine in New York, New York. Dr. Beck is rated as an Elite provider by MediFind in the treatment of VEXAS Syndrome. He is also highly rated in 2 other conditions, according to our data. His clinical expertise encompasses VEXAS Syndrome, Relapsing Polychondritis, Hypotonia, and Polyarteritis Nodosa.
Background: Allogeneic hematopoietic stem cell transplant involves taking blood stem cells from a donor and giving them to a recipient. The transplants are used to treat certain diseases and cancers. Researchers want to see if the transplant can treat VEXAS Syndrome.
Summary: 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. 78 participants will be enrolled, randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. Randomization will be stratified by prescribed GC dose on...