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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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  Data: 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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  Data: <searchLink fieldCode="AU" term="%22Beck+DB%22">Beck DB</searchLink>; Center for Human Genetics and Genomics, NYU School of Medicine, New York, NY 10016, USA.<br /><searchLink fieldCode="AU" term="%22Heiblig+M%22">Heiblig M</searchLink>; Hématologie Clinique, Lyon-Sud Hospital, Hospices Civils de Lyon, 69495 Pierre-Bénite, France.; Université Claude Bernard, 69100 Lyon, France.<br /><searchLink fieldCode="AU" term="%22Savic+S%22">Savic S</searchLink>; Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Leeds LS2 9JT, UK.<br /><searchLink fieldCode="AU" term="%22Ferrada+MA%22">Ferrada MA</searchLink>; Department of Medicine, University of Maryland School of Medicine, Baltimore, MD 21201, USA.<br /><searchLink fieldCode="AU" term="%22Mekinian+A%22">Mekinian A</searchLink>; Sorbonne Université, Service de Médecine Interne, AP-HP, Hôpital Saint-Antoine, CEREMAIAA, 75012 Paris, France.<br /><searchLink fieldCode="AU" term="%22Chowdhury+O%22">Chowdhury O</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Hammond+D%22">Hammond D</searchLink>; Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.<br /><searchLink fieldCode="AU" term="%22Weeks+LD%22">Weeks LD</searchLink>; Department of Medical Oncology, Division of Population Sciences, Dana Farber Cancer Institute, Boston, MA 02215, USA.<br /><searchLink fieldCode="AU" term="%22Gurnari+C%22">Gurnari C</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Kirino+Y%22">Kirino Y</searchLink>; Department of Stem Cell and Immune Regulation, Yokohama City University Graduate School of Medicine, Yokohama 236-0004, Japan.<br /><searchLink fieldCode="AU" term="%22Georgin-Lavialle+S%22">Georgin-Lavialle S</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Buckley+SA%22">Buckley SA</searchLink>; Medical Affairs and Clinical Development, Sobi, Inc., Waltham, MA 02451, USA.<br /><searchLink fieldCode="AU" term="%22Garcha+R%22">Garcha R</searchLink>; Medical Affairs and Clinical Development, Sobi, Inc., Waltham, MA 02451, USA.<br /><searchLink fieldCode="AU" term="%22Harder+BG%22">Harder BG</searchLink>; Medical Affairs and Clinical Development, Sobi, Inc., Waltham, MA 02451, USA.<br /><searchLink fieldCode="AU" term="%22Koster+MJ%22">Koster MJ</searchLink>; Department of Internal Medicine, Division of Rheumatology, Mayo Clinic, Rochester, MN 55905, USA.
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  Data: <searchLink fieldCode="JN" term="%22101606588%22">Journal of clinical medicine</searchLink> [J Clin Med] 2026 Feb 11; Vol. 15 (4). <i>Date of Electronic Publication: </i>2026 Feb 11.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22MDPI+AG%22">MDPI AG </searchLink><i>Country of Publication: </i>Switzerland <i>NLM ID: </i>101606588 <i>Publication Model: </i>Electronic <i>Cited Medium: </i>Print <i>ISSN: </i>2077-0383 (Print) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2220770383%22">20770383 </searchLink><i>NLM ISO Abbreviation: </i>J Clin Med <i>Subsets: </i>PubMed not MEDLINE
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