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.

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Title: 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.
Authors: Beck DB; Center for Human Genetics and Genomics, NYU School of Medicine, New York, NY 10016, USA., Heiblig M; Hématologie Clinique, Lyon-Sud Hospital, Hospices Civils de Lyon, 69495 Pierre-Bénite, France.; Université Claude Bernard, 69100 Lyon, France., Savic S; Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Leeds LS2 9JT, UK., Ferrada MA; Department of Medicine, University of Maryland School of Medicine, Baltimore, MD 21201, USA., Mekinian A; Sorbonne Université, Service de Médecine Interne, AP-HP, Hôpital Saint-Antoine, CEREMAIAA, 75012 Paris, France., Chowdhury O; Department of Haematology, Oxford University Hospitals' NHS Foundation Trust, Oxford OX3 7LE, UK.; Weatherall Institute of Molecular Medicine, University of Oxford, Oxford OX3 9DS, UK., Hammond D; Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA., Weeks LD; Department of Medical Oncology, Division of Population Sciences, Dana Farber Cancer Institute, Boston, MA 02215, USA., Gurnari C; Department of Biomedicine and Prevention, University of Rome Tor Vergata, 00133 Rome, Italy.; Translational Hematology and Oncology Research Department, Taussig Cancer Center, Cleveland Clinic, Cleveland, OH 44106, USA., Kirino Y; Department of Stem Cell and Immune Regulation, Yokohama City University Graduate School of Medicine, Yokohama 236-0004, Japan., Georgin-Lavialle S; Sorbonne University, 75020 Paris, France.; Department of Internal Medicine, DMU3ID, Tenon Hospital, 75020 Paris, France.; CEREMAIA (French National Reference Center for Autoinflammatory Diseases), 75012 Paris, France.; ERN RITA (European Reference Network for Rare Immunodeficiency, Autoinflammatory and Autoimmune Disease Network), 75012 Paris, France., Buckley SA; Medical Affairs and Clinical Development, Sobi, Inc., Waltham, MA 02451, USA., Garcha R; Medical Affairs and Clinical Development, Sobi, Inc., Waltham, MA 02451, USA., Harder BG; Medical Affairs and Clinical Development, Sobi, Inc., Waltham, MA 02451, USA., Koster MJ; Department of Internal Medicine, Division of Rheumatology, Mayo Clinic, Rochester, MN 55905, USA.
Source: Journal of clinical medicine [J Clin Med] 2026 Feb 11; Vol. 15 (4). Date of Electronic Publication: 2026 Feb 11.
Publication Type: Journal Article
Journal Info: Publisher: MDPI AG Country of Publication: Switzerland NLM ID: 101606588 Publication Model: Electronic Cited Medium: Print ISSN: 2077-0383 (Print) Linking ISSN: 20770383 NLM ISO Abbreviation: J Clin Med Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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ISSN:2077-0383
DOI:10.3390/jcm15041426