[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100576213":3},{"organization":4,"armGroups":7,"interventions":24,"overallOfficials":32,"centralContacts":37,"locations":42,"responsibleParty":423,"collaborators":425,"id":428,"slug":429,"hasResults":430,"nctId":431,"briefTitle":432,"officialTitle":433,"acronym":29,"eligibilityCriteria":434,"healthyVolunteers":430,"sex":435,"minAge":436,"maxAge":29,"enrollmentInfo":437,"targetDuration":29,"studyType":440,"phases":441,"briefSummary":443,"conditions":444,"keywords":447,"overallStatus":45,"whyStopped":29,"lastUpdateSubmitDate":457,"lastUpdatePostDateStruct":458,"startDateStruct":461,"completionDateStruct":463,"leadSponsor":465,"locationsCount":466},{"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",[43,57,67,77,86,96,105,115,125,135,147,157,167,177,188,197,203,212,221,230,242,252,262,272,282,292,303,312,321,330,341,350,359,370,379,388,399,408,414],{"facility":44,"status":45,"city":46,"state":47,"zip":48,"country":49,"countryCode":50,"cosmosGeoPoint":51,"geoPoint":56,"contacts":29},"Mayo Clinic - Scottsdale","RECRUITING","Scottsdale","Arizona","85259","United States","US",{"type":52,"coordinates":53},"Point",[54,55],-111.89903,33.50921,{"lat":55,"lon":54},{"facility":58,"status":45,"city":59,"state":60,"zip":61,"country":49,"countryCode":50,"cosmosGeoPoint":62,"geoPoint":66,"contacts":29},"Dana Farber Cancer Institute","Boston","Massachusetts","02215",{"type":52,"coordinates":63},[64,65],-71.05977,42.35843,{"lat":65,"lon":64},{"facility":68,"status":45,"city":69,"state":70,"zip":71,"country":49,"countryCode":50,"cosmosGeoPoint":72,"geoPoint":76,"contacts":29},"Mayo Clinic - Rochester","Rochester","Minnesota","55905",{"type":52,"coordinates":73},[74,75],-92.4699,44.02163,{"lat":75,"lon":74},{"facility":78,"status":45,"city":79,"state":79,"zip":80,"country":49,"countryCode":50,"cosmosGeoPoint":81,"geoPoint":85,"contacts":29},"NYU Langone Health","New York","10016",{"type":52,"coordinates":82},[83,84],-74.00597,40.71427,{"lat":84,"lon":83},{"facility":87,"status":45,"city":88,"state":89,"zip":90,"country":49,"countryCode":50,"cosmosGeoPoint":91,"geoPoint":95,"contacts":29},"Cleveland Clinic - Cleveland","Cleveland","Ohio","44195",{"type":52,"coordinates":92},[93,94],-81.69541,41.4995,{"lat":94,"lon":93},{"facility":97,"status":45,"city":98,"state":89,"zip":99,"country":49,"countryCode":50,"cosmosGeoPoint":100,"geoPoint":104,"contacts":29},"The James Cancer Hospital and Solove Research Institute","Columbus","43210",{"type":52,"coordinates":101},[102,103],-82.99879,39.96118,{"lat":103,"lon":102},{"facility":106,"status":45,"city":107,"state":108,"zip":109,"country":49,"countryCode":50,"cosmosGeoPoint":110,"geoPoint":114,"contacts":29},"UT MD Anderson Cancer Center","Houston","Texas","77030",{"type":52,"coordinates":111},[112,113],-95.36327,29.76328,{"lat":113,"lon":112},{"facility":116,"status":45,"city":117,"state":118,"zip":119,"country":49,"countryCode":50,"cosmosGeoPoint":120,"geoPoint":124,"contacts":29},"University of Utah Healthcare","Salt Lake City","Utah","84132",{"type":52,"coordinates":121},[122,123],-111.89105,40.76078,{"lat":123,"lon":122},{"facility":126,"status":45,"city":127,"state":128,"zip":129,"country":49,"countryCode":50,"cosmosGeoPoint":130,"geoPoint":134,"contacts":29},"Fred Hutchinson Cancer Center","Seattle","Washington","98109",{"type":52,"coordinates":131},[132,133],-122.33207,47.60621,{"lat":133,"lon":132},{"facility":136,"status":45,"city":137,"state":138,"zip":139,"country":140,"countryCode":141,"cosmosGeoPoint":142,"geoPoint":146,"contacts":29},"Vancouver Coastal Health Research Institute","Vancouver","British Columbia","V5Z 1M9","Canada","CA",{"type":52,"coordinates":143},[144,145],-123.11934,49.24966,{"lat":145,"lon":144},{"facility":148,"status":45,"city":149,"state":150,"zip":151,"country":140,"countryCode":141,"cosmosGeoPoint":152,"geoPoint":156,"contacts":29},"Queen Elizabeth II Health Sciences Center","Halifax","Nova Scotia","B3H 2Y9",{"type":52,"coordinates":153},[154,155],-63.57688,44.64269,{"lat":155,"lon":154},{"facility":158,"status":45,"city":159,"state":160,"zip":161,"country":140,"countryCode":141,"cosmosGeoPoint":162,"geoPoint":166,"contacts":29},"Princess Margaret Cancer Centre","Toronto","Ontario","M5G 2M9",{"type":52,"coordinates":163},[164,165],-79.39864,43.70643,{"lat":165,"lon":164},{"facility":168,"status":45,"city":169,"state":170,"zip":171,"country":140,"countryCode":141,"cosmosGeoPoint":172,"geoPoint":176,"contacts":29},"Hospital du Sacre-Coeur in Montreal","Montreal","Quebec","H4J 1C5",{"type":52,"coordinates":173},[174,175],-73.58781,45.50884,{"lat":175,"lon":174},{"facility":178,"status":45,"city":179,"state":29,"zip":180,"country":181,"countryCode":182,"cosmosGeoPoint":183,"geoPoint":187,"contacts":29},"Lille University Hospital Center","Lille","59037","France","FR",{"type":52,"coordinates":184},[185,186],3.05512,50.63391,{"lat":186,"lon":185},{"facility":189,"status":45,"city":190,"state":29,"zip":191,"country":181,"countryCode":182,"cosmosGeoPoint":192,"geoPoint":196,"contacts":29},"Saint-Antoine Hospital - APHP","Paris","75012",{"type":52,"coordinates":193},[194,195],2.3488,48.85341,{"lat":195,"lon":194},{"facility":198,"status":45,"city":190,"state":29,"zip":199,"country":181,"countryCode":182,"cosmosGeoPoint":200,"geoPoint":202,"contacts":29},"Tenon Hospital - APHP","75020",{"type":52,"coordinates":201},[194,195],{"lat":195,"lon":194},{"facility":204,"status":45,"city":205,"state":29,"zip":206,"country":181,"countryCode":182,"cosmosGeoPoint":207,"geoPoint":211,"contacts":29},"Hospices Civils de Lyon - Lyon Sud","Pierre-Bénite","69310",{"type":52,"coordinates":208},[209,210],4.82424,45.70359,{"lat":210,"lon":209},{"facility":213,"status":45,"city":214,"state":29,"zip":215,"country":181,"countryCode":182,"cosmosGeoPoint":216,"geoPoint":220,"contacts":29},"University Hospital Center of Poitiers","Poitiers","86000",{"type":52,"coordinates":217},[218,219],0.34348,46.58261,{"lat":219,"lon":218},{"facility":222,"status":45,"city":223,"state":29,"zip":224,"country":181,"countryCode":182,"cosmosGeoPoint":225,"geoPoint":229,"contacts":29},"IUCT-Oncopole","Toulouse","31100",{"type":52,"coordinates":226},[227,228],1.44367,43.60426,{"lat":228,"lon":227},{"facility":231,"status":45,"city":232,"state":233,"zip":234,"country":235,"countryCode":236,"cosmosGeoPoint":237,"geoPoint":241,"contacts":29},"University Hospital Tuebingen, Medical Clinic II, Hematology, Oncology, Clinical Immunology and Rheumatology","Tübingen","Baden-Wurttemberg","72076","Germany","DE",{"type":52,"coordinates":238},[239,240],9.05222,48.52266,{"lat":240,"lon":239},{"facility":243,"status":45,"city":244,"state":245,"zip":246,"country":235,"countryCode":236,"cosmosGeoPoint":247,"geoPoint":251,"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":52,"coordinates":248},[249,250],11.57549,48.13743,{"lat":250,"lon":249},{"facility":253,"status":45,"city":254,"state":255,"zip":256,"country":235,"countryCode":236,"cosmosGeoPoint":257,"geoPoint":261,"contacts":29},"University Hospital Hamburg-Eppendorf","Hamburg","Free and Hanseatic City of Hamburg","20246",{"type":52,"coordinates":258},[259,260],9.99302,53.55073,{"lat":260,"lon":259},{"facility":263,"status":45,"city":264,"state":265,"zip":266,"country":235,"countryCode":236,"cosmosGeoPoint":267,"geoPoint":271,"contacts":29},"University Hospital Duesseldorf","Düsseldorf","North Rhine-Westphalia","40225",{"type":52,"coordinates":268},[269,270],6.77927,51.22319,{"lat":270,"lon":269},{"facility":273,"status":45,"city":274,"state":275,"zip":276,"country":235,"countryCode":236,"cosmosGeoPoint":277,"geoPoint":281,"contacts":29},"University Hospital Carl Gustav Carus Dresden, Medical Clinic and Polyclinic I","Dresden","Saxony","01307",{"type":52,"coordinates":278},[279,280],13.73832,51.05089,{"lat":280,"lon":279},{"facility":283,"status":45,"city":284,"state":285,"zip":286,"country":235,"countryCode":236,"cosmosGeoPoint":287,"geoPoint":291,"contacts":29},"University Hospital Schleswig-Holstein","Lübeck","Schleswig-Holstein","23538",{"type":52,"coordinates":288},[289,290],10.68729,53.86893,{"lat":290,"lon":289},{"facility":293,"status":45,"city":294,"state":29,"zip":295,"country":296,"countryCode":297,"cosmosGeoPoint":298,"geoPoint":302,"contacts":29},"Hospital San Raffaele, IRCCS, Unit of Immunology, Rheumatology, Allergy and Rare Diseases","Milan","20132","Italy","IT",{"type":52,"coordinates":299},[300,301],9.18951,45.46427,{"lat":301,"lon":300},{"facility":304,"status":45,"city":305,"state":29,"zip":306,"country":296,"countryCode":297,"cosmosGeoPoint":307,"geoPoint":311,"contacts":29},"University Hospital of Padova, Rheumatology Unit, Department of Medicine - DIMED","Padova","35128",{"type":52,"coordinates":308},[309,310],11.14261,44.38225,{"lat":310,"lon":309},{"facility":313,"status":45,"city":314,"state":29,"zip":315,"country":296,"countryCode":297,"cosmosGeoPoint":316,"geoPoint":320,"contacts":29},"AUSL of Reggio Emilia - Hospital Arcispedale S. Maria Nuova, Complex Structure of Rheumatology","Reggio Emilia","42123",{"type":52,"coordinates":317},[318,319],10.63125,44.69825,{"lat":319,"lon":318},{"facility":322,"status":45,"city":323,"state":29,"zip":324,"country":296,"countryCode":297,"cosmosGeoPoint":325,"geoPoint":329,"contacts":29},"Foundation PTV - Polyclinic Tor Vergata Biomedicine and prevention","Roma","00133",{"type":52,"coordinates":326},[327,328],11.10642,44.99364,{"lat":328,"lon":327},{"facility":331,"status":45,"city":332,"state":29,"zip":333,"country":334,"countryCode":335,"cosmosGeoPoint":336,"geoPoint":340,"contacts":29},"Fukushima Medical University Hospital","Fukushima","960-1295","Japan","JP",{"type":52,"coordinates":337},[338,339],140.46667,37.75,{"lat":339,"lon":338},{"facility":342,"status":45,"city":343,"state":29,"zip":344,"country":334,"countryCode":335,"cosmosGeoPoint":345,"geoPoint":349,"contacts":29},"Nagasaki University Hospital","Nagasaki","852-8501",{"type":52,"coordinates":346},[347,348],129.88333,32.75,{"lat":348,"lon":347},{"facility":351,"status":45,"city":352,"state":29,"zip":353,"country":334,"countryCode":335,"cosmosGeoPoint":354,"geoPoint":358,"contacts":29},"Yokohama City University Hospital","Yokohama","236-0004",{"type":52,"coordinates":355},[356,357],139.65,35.43333,{"lat":357,"lon":356},{"facility":360,"status":45,"city":361,"state":29,"zip":362,"country":363,"countryCode":364,"cosmosGeoPoint":365,"geoPoint":369,"contacts":29},"Hospital Clinic of Barcelona","Barcelona","08036","Spain","ES",{"type":52,"coordinates":366},[367,368],2.15899,41.38879,{"lat":368,"lon":367},{"facility":371,"status":45,"city":372,"state":29,"zip":373,"country":363,"countryCode":364,"cosmosGeoPoint":374,"geoPoint":378,"contacts":29},"Catalan Institute of Oncology, Hospital Duran i Reynals, Department of Clinical Hematology","L'Hospitalet de Llobregat","08908",{"type":52,"coordinates":375},[376,377],2.10028,41.35967,{"lat":377,"lon":376},{"facility":380,"status":45,"city":381,"state":29,"zip":382,"country":363,"countryCode":364,"cosmosGeoPoint":383,"geoPoint":387,"contacts":29},"University Clinical Hospital of Salamanca","Salamanca","37007",{"type":52,"coordinates":384},[385,386],-5.66388,40.96882,{"lat":386,"lon":385},{"facility":389,"status":45,"city":390,"state":29,"zip":391,"country":392,"countryCode":393,"cosmosGeoPoint":394,"geoPoint":398,"contacts":29},"St James's University Hospital","Leeds","LS9 7TF","United Kingdom","UK",{"type":52,"coordinates":395},[396,397],-1.54785,53.79648,{"lat":397,"lon":396},{"facility":400,"status":45,"city":401,"state":29,"zip":402,"country":392,"countryCode":393,"cosmosGeoPoint":403,"geoPoint":407,"contacts":29},"Royal Free Hospital","London","NW3 2QG",{"type":52,"coordinates":404},[405,406],-0.12574,51.50853,{"lat":406,"lon":405},{"facility":409,"status":45,"city":401,"state":29,"zip":410,"country":392,"countryCode":393,"cosmosGeoPoint":411,"geoPoint":413,"contacts":29},"King's College Hospital, Department of Hematology","SE5 9RS",{"type":52,"coordinates":412},[405,406],{"lat":406,"lon":405},{"facility":415,"status":45,"city":416,"state":29,"zip":417,"country":392,"countryCode":393,"cosmosGeoPoint":418,"geoPoint":422,"contacts":29},"Churchill Hospital","Oxford","OX3 7LE",{"type":52,"coordinates":419},[420,421],-1.25596,51.75222,{"lat":421,"lon":420},{"type":424,"investigatorFullName":29,"investigatorTitle":29,"investigatorAffiliation":29,"oldNameTitle":29,"oldOrganization":29},"SPONSOR",[426],{"name":427,"class":6},"PSI CRO","100576213","phase-2-a-study-to-assess-the-effectiveness-and-safety-of-pacritinib-in-patients-with-vexas-syndrome-paxis-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":438,"type":439},78,"ESTIMATED","INTERVENTIONAL",[442],"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.",[445,446],"VEXAS","VEXAS Syndrome",[448,449,450,451,452,453,454,455,9,456],"Vacuoles","E1 Ubiquitin-activating enzyme","X-linked","Autoinflammatory","Somatic syndrome","UBA1","Hematologic neoplasms","Myelodysplastic Syndromes","Myeloid progenitors","2026-04-23",{"date":459,"type":460},"2026-04-24","ACTUAL",{"date":462,"type":460},"2025-05-28",{"date":464,"type":439},"2028-05-22",{"name":5,"class":6},39]