[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100576213":3},{"organization":4,"armGroups":7,"interventions":24,"overallOfficials":32,"centralContacts":37,"locations":42,"responsibleParty":415,"collaborators":417,"id":420,"slug":29,"hasResults":421,"nctId":422,"briefTitle":423,"officialTitle":424,"acronym":29,"eligibilityCriteria":425,"healthyVolunteers":421,"sex":426,"minAge":427,"maxAge":29,"enrollmentInfo":428,"targetDuration":29,"studyType":431,"phases":432,"briefSummary":434,"conditions":435,"keywords":438,"overallStatus":45,"whyStopped":29,"lastUpdateSubmitDate":448,"lastUpdatePostDateStruct":449,"startDateStruct":452,"completionDateStruct":454,"leadSponsor":456,"locationsCount":457},{"fullName":5,"class":6},"Swedish Orphan Biovitrum","INDUSTRY",[8,14,19],{"label":9,"type":10,"description":11,"interventionNames":12},"Pacritinib","EXPERIMENTAL","To receive oral administration of pacritinib dose A for up to 24 weeks.",[13],"Drug: Pacritinib",{"label":15,"type":10,"description":16,"interventionNames":17},"Pacritinib + placebo","To receive oral administration of pacritinib dose B plus placebo for up to 24 weeks",[13,18],"Drug: Placebo",{"label":20,"type":21,"description":22,"interventionNames":23},"Placebo","PLACEBO_COMPARATOR","To receive oral administration of placebo for up to 24 weeks.",[18],[25,30],{"type":26,"name":9,"description":27,"armGroupLabels":28,"otherNames":29},"DRUG","Supplied in hard capsules.",[9,15],null,{"type":26,"name":20,"description":27,"armGroupLabels":31,"otherNames":29},[15,20],[33],{"name":34,"affiliation":35,"role":36},"Study Physician","Sobi, Inc.","STUDY_DIRECTOR",[38],{"name":34,"role":39,"phone":40,"phoneExt":29,"email":41},"CONTACT","+4686972000","medical.info@sobi.com",[43,56,66,76,85,95,104,114,124,134,145,155,165,175,185,194,200,209,218,227,238,248,258,268,278,288,298,307,316,325,335,344,353,363,372,381,391,400,406],{"facility":44,"status":45,"city":46,"state":47,"zip":48,"country":49,"cosmosGeoPoint":50,"geoPoint":55,"contacts":29},"Mayo Clinic - Scottsdale","RECRUITING","Scottsdale","Arizona","85259","United States",{"type":51,"coordinates":52},"Point",[53,54],-111.89903,33.50921,{"lat":54,"lon":53},{"facility":57,"status":45,"city":58,"state":59,"zip":60,"country":49,"cosmosGeoPoint":61,"geoPoint":65,"contacts":29},"Dana Farber Cancer Institute","Boston","Massachusetts","02215",{"type":51,"coordinates":62},[63,64],-71.05977,42.35843,{"lat":64,"lon":63},{"facility":67,"status":45,"city":68,"state":69,"zip":70,"country":49,"cosmosGeoPoint":71,"geoPoint":75,"contacts":29},"Mayo Clinic - Rochester","Rochester","Minnesota","55905",{"type":51,"coordinates":72},[73,74],-92.4699,44.02163,{"lat":74,"lon":73},{"facility":77,"status":45,"city":78,"state":78,"zip":79,"country":49,"cosmosGeoPoint":80,"geoPoint":84,"contacts":29},"NYU Langone Health","New York","10016",{"type":51,"coordinates":81},[82,83],-74.00597,40.71427,{"lat":83,"lon":82},{"facility":86,"status":45,"city":87,"state":88,"zip":89,"country":49,"cosmosGeoPoint":90,"geoPoint":94,"contacts":29},"Cleveland Clinic - Cleveland","Cleveland","Ohio","44195",{"type":51,"coordinates":91},[92,93],-81.69541,41.4995,{"lat":93,"lon":92},{"facility":96,"status":45,"city":97,"state":88,"zip":98,"country":49,"cosmosGeoPoint":99,"geoPoint":103,"contacts":29},"The James Cancer Hospital and Solove Research Institute","Columbus","43210",{"type":51,"coordinates":100},[101,102],-82.99879,39.96118,{"lat":102,"lon":101},{"facility":105,"status":45,"city":106,"state":107,"zip":108,"country":49,"cosmosGeoPoint":109,"geoPoint":113,"contacts":29},"UT MD Anderson Cancer Center","Houston","Texas","77030",{"type":51,"coordinates":110},[111,112],-95.36327,29.76328,{"lat":112,"lon":111},{"facility":115,"status":45,"city":116,"state":117,"zip":118,"country":49,"cosmosGeoPoint":119,"geoPoint":123,"contacts":29},"University of Utah Healthcare","Salt Lake City","Utah","84132",{"type":51,"coordinates":120},[121,122],-111.89105,40.76078,{"lat":122,"lon":121},{"facility":125,"status":45,"city":126,"state":127,"zip":128,"country":49,"cosmosGeoPoint":129,"geoPoint":133,"contacts":29},"Fred Hutchinson Cancer Center","Seattle","Washington","98109",{"type":51,"coordinates":130},[131,132],-122.33207,47.60621,{"lat":132,"lon":131},{"facility":135,"status":45,"city":136,"state":137,"zip":138,"country":139,"cosmosGeoPoint":140,"geoPoint":144,"contacts":29},"Vancouver Coastal Health Research Institute","Vancouver","British Columbia","V5Z 1M9","Canada",{"type":51,"coordinates":141},[142,143],-123.11934,49.24966,{"lat":143,"lon":142},{"facility":146,"status":45,"city":147,"state":148,"zip":149,"country":139,"cosmosGeoPoint":150,"geoPoint":154,"contacts":29},"Queen Elizabeth II Health Sciences Center","Halifax","Nova Scotia","B3H 2Y9",{"type":51,"coordinates":151},[152,153],-63.57688,44.64269,{"lat":153,"lon":152},{"facility":156,"status":45,"city":157,"state":158,"zip":159,"country":139,"cosmosGeoPoint":160,"geoPoint":164,"contacts":29},"Princess Margaret Cancer Centre","Toronto","Ontario","M5G 2M9",{"type":51,"coordinates":161},[162,163],-79.39864,43.70643,{"lat":163,"lon":162},{"facility":166,"status":45,"city":167,"state":168,"zip":169,"country":139,"cosmosGeoPoint":170,"geoPoint":174,"contacts":29},"Hospital du Sacre-Coeur in Montreal","Montreal","Quebec","H4J 1C5",{"type":51,"coordinates":171},[172,173],-73.58781,45.50884,{"lat":173,"lon":172},{"facility":176,"status":45,"city":177,"state":29,"zip":178,"country":179,"cosmosGeoPoint":180,"geoPoint":184,"contacts":29},"Lille University Hospital Center","Lille","59037","France",{"type":51,"coordinates":181},[182,183],3.05512,50.63391,{"lat":183,"lon":182},{"facility":186,"status":45,"city":187,"state":29,"zip":188,"country":179,"cosmosGeoPoint":189,"geoPoint":193,"contacts":29},"Saint-Antoine Hospital - APHP","Paris","75012",{"type":51,"coordinates":190},[191,192],2.3488,48.85341,{"lat":192,"lon":191},{"facility":195,"status":45,"city":187,"state":29,"zip":196,"country":179,"cosmosGeoPoint":197,"geoPoint":199,"contacts":29},"Tenon Hospital - APHP","75020",{"type":51,"coordinates":198},[191,192],{"lat":192,"lon":191},{"facility":201,"status":45,"city":202,"state":29,"zip":203,"country":179,"cosmosGeoPoint":204,"geoPoint":208,"contacts":29},"Hospices Civils de Lyon - Lyon Sud","Pierre-Bénite","69310",{"type":51,"coordinates":205},[206,207],4.82424,45.70359,{"lat":207,"lon":206},{"facility":210,"status":45,"city":211,"state":29,"zip":212,"country":179,"cosmosGeoPoint":213,"geoPoint":217,"contacts":29},"University Hospital Center of Poitiers","Poitiers","86000",{"type":51,"coordinates":214},[215,216],0.34348,46.58261,{"lat":216,"lon":215},{"facility":219,"status":45,"city":220,"state":29,"zip":221,"country":179,"cosmosGeoPoint":222,"geoPoint":226,"contacts":29},"IUCT-Oncopole","Toulouse","31100",{"type":51,"coordinates":223},[224,225],1.44367,43.60426,{"lat":225,"lon":224},{"facility":228,"status":45,"city":229,"state":230,"zip":231,"country":232,"cosmosGeoPoint":233,"geoPoint":237,"contacts":29},"University Hospital Tuebingen, Medical Clinic II, Hematology, Oncology, Clinical Immunology and Rheumatology","Tübingen","Baden-Wurttemberg","72076","Germany",{"type":51,"coordinates":234},[235,236],9.05222,48.52266,{"lat":236,"lon":235},{"facility":239,"status":45,"city":240,"state":241,"zip":242,"country":232,"cosmosGeoPoint":243,"geoPoint":247,"contacts":29},"Hospital Rechts der Isar of the Technical University of Munich, Clinic and Polyclinic for Internal Medicine III: Hematology and Internal Oncology","Munich","Bavaria","81675",{"type":51,"coordinates":244},[245,246],11.57549,48.13743,{"lat":246,"lon":245},{"facility":249,"status":45,"city":250,"state":251,"zip":252,"country":232,"cosmosGeoPoint":253,"geoPoint":257,"contacts":29},"University Hospital Hamburg-Eppendorf","Hamburg","Free and Hanseatic City of Hamburg","20246",{"type":51,"coordinates":254},[255,256],9.99302,53.55073,{"lat":256,"lon":255},{"facility":259,"status":45,"city":260,"state":261,"zip":262,"country":232,"cosmosGeoPoint":263,"geoPoint":267,"contacts":29},"University Hospital Duesseldorf","Düsseldorf","North Rhine-Westphalia","40225",{"type":51,"coordinates":264},[265,266],6.77927,51.22319,{"lat":266,"lon":265},{"facility":269,"status":45,"city":270,"state":271,"zip":272,"country":232,"cosmosGeoPoint":273,"geoPoint":277,"contacts":29},"University Hospital Carl Gustav Carus Dresden, Medical Clinic and Polyclinic I","Dresden","Saxony","01307",{"type":51,"coordinates":274},[275,276],13.73832,51.05089,{"lat":276,"lon":275},{"facility":279,"status":45,"city":280,"state":281,"zip":282,"country":232,"cosmosGeoPoint":283,"geoPoint":287,"contacts":29},"University Hospital Schleswig-Holstein","Lübeck","Schleswig-Holstein","23538",{"type":51,"coordinates":284},[285,286],10.68729,53.86893,{"lat":286,"lon":285},{"facility":289,"status":45,"city":290,"state":29,"zip":291,"country":292,"cosmosGeoPoint":293,"geoPoint":297,"contacts":29},"Hospital San Raffaele, IRCCS, Unit of Immunology, Rheumatology, Allergy and Rare Diseases","Milan","20132","Italy",{"type":51,"coordinates":294},[295,296],9.18951,45.46427,{"lat":296,"lon":295},{"facility":299,"status":45,"city":300,"state":29,"zip":301,"country":292,"cosmosGeoPoint":302,"geoPoint":306,"contacts":29},"University Hospital of Padova, Rheumatology Unit, Department of Medicine - DIMED","Padova","35128",{"type":51,"coordinates":303},[304,305],11.14261,44.38225,{"lat":305,"lon":304},{"facility":308,"status":45,"city":309,"state":29,"zip":310,"country":292,"cosmosGeoPoint":311,"geoPoint":315,"contacts":29},"AUSL of Reggio Emilia - Hospital Arcispedale S. Maria Nuova, Complex Structure of Rheumatology","Reggio Emilia","42123",{"type":51,"coordinates":312},[313,314],10.63125,44.69825,{"lat":314,"lon":313},{"facility":317,"status":45,"city":318,"state":29,"zip":319,"country":292,"cosmosGeoPoint":320,"geoPoint":324,"contacts":29},"Foundation PTV - Polyclinic Tor Vergata Biomedicine and prevention","Roma","00133",{"type":51,"coordinates":321},[322,323],11.10642,44.99364,{"lat":323,"lon":322},{"facility":326,"status":45,"city":327,"state":29,"zip":328,"country":329,"cosmosGeoPoint":330,"geoPoint":334,"contacts":29},"Fukushima Medical University Hospital","Fukushima","960-1295","Japan",{"type":51,"coordinates":331},[332,333],140.46667,37.75,{"lat":333,"lon":332},{"facility":336,"status":45,"city":337,"state":29,"zip":338,"country":329,"cosmosGeoPoint":339,"geoPoint":343,"contacts":29},"Nagasaki University Hospital","Nagasaki","852-8501",{"type":51,"coordinates":340},[341,342],129.88333,32.75,{"lat":342,"lon":341},{"facility":345,"status":45,"city":346,"state":29,"zip":347,"country":329,"cosmosGeoPoint":348,"geoPoint":352,"contacts":29},"Yokohama City University Hospital","Yokohama","236-0004",{"type":51,"coordinates":349},[350,351],139.65,35.43333,{"lat":351,"lon":350},{"facility":354,"status":45,"city":355,"state":29,"zip":356,"country":357,"cosmosGeoPoint":358,"geoPoint":362,"contacts":29},"Hospital Clinic of Barcelona","Barcelona","08036","Spain",{"type":51,"coordinates":359},[360,361],2.15899,41.38879,{"lat":361,"lon":360},{"facility":364,"status":45,"city":365,"state":29,"zip":366,"country":357,"cosmosGeoPoint":367,"geoPoint":371,"contacts":29},"Catalan Institute of Oncology, Hospital Duran i Reynals, Department of Clinical Hematology","L'Hospitalet de Llobregat","08908",{"type":51,"coordinates":368},[369,370],2.10028,41.35967,{"lat":370,"lon":369},{"facility":373,"status":45,"city":374,"state":29,"zip":375,"country":357,"cosmosGeoPoint":376,"geoPoint":380,"contacts":29},"University Clinical Hospital of Salamanca","Salamanca","37007",{"type":51,"coordinates":377},[378,379],-5.66388,40.96882,{"lat":379,"lon":378},{"facility":382,"status":45,"city":383,"state":29,"zip":384,"country":385,"cosmosGeoPoint":386,"geoPoint":390,"contacts":29},"St James's University Hospital","Leeds","LS9 7TF","United Kingdom",{"type":51,"coordinates":387},[388,389],-1.54785,53.79648,{"lat":389,"lon":388},{"facility":392,"status":45,"city":393,"state":29,"zip":394,"country":385,"cosmosGeoPoint":395,"geoPoint":399,"contacts":29},"Royal Free Hospital","London","NW3 2QG",{"type":51,"coordinates":396},[397,398],-0.12574,51.50853,{"lat":398,"lon":397},{"facility":401,"status":45,"city":393,"state":29,"zip":402,"country":385,"cosmosGeoPoint":403,"geoPoint":405,"contacts":29},"King's College Hospital, Department of Hematology","SE5 9RS",{"type":51,"coordinates":404},[397,398],{"lat":398,"lon":397},{"facility":407,"status":45,"city":408,"state":29,"zip":409,"country":385,"cosmosGeoPoint":410,"geoPoint":414,"contacts":29},"Churchill Hospital","Oxford","OX3 7LE",{"type":51,"coordinates":411},[412,413],-1.25596,51.75222,{"lat":413,"lon":412},{"type":416,"investigatorFullName":29,"investigatorTitle":29,"investigatorAffiliation":29,"oldNameTitle":29,"oldOrganization":29},"SPONSOR",[418],{"name":419,"class":6},"PSI CRO","100576213",false,"NCT06782373","A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)","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","Key Inclusion Criteria:\n\n* Documented evidence of a pathogenic mutation at methionine-41 (M41) or neighboring splice site mutation (c.118-1, c.118-2) position in UBA1 mutation based on myeloid next-generation sequencing (NGS) droplet digital polymerase chain reaction (ddPCR), or Sanger sequencing in peripheral blood or bone marrow samples.\n* Current or documented evidence of past inflammatory involvement within 6 months prior to enrollment of at least one of the following organ systems by VEXAS syndrome: cutaneous (e.g., neutrophilic dermatosis, cutaneous vasculitis), vasculature (e.g., vasculitis), musculoskeletal (e.g., chondritis, arthritis), ocular (e.g., uveitis, scleritis), periorbital (e.g. periorbital edema), genitourinary (e.g., epididymitis), or pulmonary (e.g., alveolitis).\n* Receiving ongoing GC therapy (stable prednisone or prednisolone dose of 15-45 mg\u002Fday) leading up to enrollment.\n* Karnofsky Performance Status ≥50%\n* Adequate organ function, meeting all the following criteria within 30 days prior to enrollment:\n\n  1. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3 × upper limit of normal (ULN)\n  2. Total bilirubin ≤4 × ULN (≤8 × ULN in the setting of Gilbert's syndrome)\n  3. Creatinine clearance (CrCl) ≥30 mL\u002Fmin based on the Cockcroft-Gault formula\n  4. Absolute neutrophil count ≥500\u002FμL\n  5. Prothrombin time (PT) or international normalized ratio (INR) ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation)\n  6. Partial thromboplastic time (PTT) or activated PTT ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation)\n  7. Platelet count ≥25 × 10\\^9\u002FL (value must be obtained in the absence of platelet transfusion in the prior 7 days)\n  8. Peripheral blasts \\\u003C5%\n* QT corrected by the Fridericia method (QTcF) ≤450 msec in males or ≤470 msec in females. Participants with QRS prolongation \\>100 msec may enroll if their QTcF is ≤480 msec. If QTcF is thought to be prolonged due to a modifiable factor (e.g., medication \u002F electrolyte abnormality), QTcF may be reevaluated.\n* Women of child-bearing potential (WOCBP) must have a negative serum pregnancy test within 30 days prior to enrollment and a negative urine pregnancy test on Day 1 prior to randomization and dosing.\n* WOCBP and male participants must agree to use a highly effective method of contraception starting at the first dose of trial therapy through 30 days after the last dose of trial therapy.\n\nKey Exclusion Criteria\n\n* Prior allogenic hematopoietic stem cell transplant (allo-HSCT) or solid organ transplant (other than corneal).\n* Current use of systemic GCs for conditions other than VEXAS syndrome, which, in the opinion of the Investigator, would interfere with adherence to a GC taper regimen and\u002For assessment of efficacy.\n* More than one prior admission to an intensive care unit due to a VEXAS Syndrome flare within the prior 6 months.\n* Received ≥9 units of intensive red blood cell (RBC) transfusions in the 90 days prior to enrollment.\n* Known concurrent myelodysplastic syndrome (MDS) requiring antineoplastic treatment, or allo-HSCT, or known high-risk or very high-risk MDS based on the Revised International Prognostic Scoring System (IPSS-R). Participants with MDS who do not meet these criteria may enroll.\n* Malignancy within 1 year prior to enrollment with the exception of MDS (per exclusion criterion), curatively treated non-melanoma skin cancer, or curatively treated carcinoma in situ. Participants with pre-malignant hematologic conditions (e.g., monoclonal gammopathy of unknown significance \\[MGUS\\], clonal cytopenia of unknown significance) may enroll.\n* Exposure to hypomethylating agents (HMA) within 6 months prior to enrollment, or exposure to more than 6 cycles of HMAs at any time.\n* Exposure to non-GC anti-inflammatory therapy or hematologic support therapy within protocol defined timeframes prior to enrollment\n* Exposure to anti-platelet therapy with the exception of low-dose aspirin (≤100 mg daily) within 28 days prior to enrollment.\n* Known concomitant multiple myeloma, or serum M-protein ≥3 g\u002FdL, involved-to uninvolved free light chain (FLC) ratio ≥100, or involved FLC level ≥100 mg\u002FdL. Participants with MGUS may enroll.\n* Systemic treatment with a strong cytochrome P450 3A4 (CYP3A4) inhibitor or inducer within 5 half-lives prior to enrollment.\n* Significant recent bleeding history defined as National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) grade ≥2 within 3 months prior to enrollment, unless precipitated by an inciting event.\n* History of clinically significant cardiovascular disease, or clinically significant abnormalities in rhythm or conduction during Screening ECG, including:\n\n  1. QT corrected by the Fridericia method (QTcF) \\> 480 msec within 30 days prior to enrollment; if QTcF is thought to be prolonged due to a modifiable factor (e.g., medication \u002F electrolyte abnormality), QTcF may be re-evaluated\n  2. Severe cardiac event (CTCAE grade ≥3) within 3 months prior to enrollment\n  3. Heart failure resulting in limitations during ordinary activity.\n* Arterial or venous thrombotic or embolic events, including deep vein thrombosis, pulmonary embolism, and cerebrovascular accident (including transient ischemic attacks), within 60 days prior to enrollment.\n* Moderate or severe hepatic impairment that meets criteria for Child-Pugh Class B or C, or active viral hepatitis.\n* Uncontrolled human immunodeficiency virus (HIV) off antiretrovirals, or on antiretrovirals with detectable viral load.\n* Positive Quantiferon (or other interferon gamma release assay) during Screening.\n* Known history of disseminated mycobacterial infection.\n* Concurrent enrollment in another interventional trial, or treatment with an experimental therapy within 28 days or five half-lives prior to enrollment, whichever is longer.\n* Pregnant, intending to become pregnant during the trial, or currently breastfeeding\u002Flactating.\n* Participants with any acute, active infection requiring systemic antimicrobial treatment at the time of enrollment. Exceptions are made for prophylactic antibiotics or chronic antibiotic therapy for non-acute conditions.\n* Known hypersensitivity to pacritinib or any of the following inactive ingredients: microcrystalline cellulose, polyethylene glycol, and magnesium stearate.","ALL","18 Years",{"count":429,"type":430},78,"ESTIMATED","INTERVENTIONAL",[433],"PHASE2","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 the day of randomization.",[436,437],"VEXAS","VEXAS Syndrome",[439,440,441,442,443,444,445,446,9,447],"Vacuoles","E1 Ubiquitin-activating enzyme","X-linked","Autoinflammatory","Somatic syndrome","UBA1","Hematologic neoplasms","Myelodysplastic Syndromes","Myeloid progenitors","2026-04-23",{"date":450,"type":451},"2026-04-24","ACTUAL",{"date":453,"type":451},"2025-05-28",{"date":455,"type":430},"2028-05-22",{"name":5,"class":6},39]