[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"vexas-syndrome\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:vexas-syndrome":121},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,45,71,104,144,168],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":34,"lastUpdatePostDateStruct":35,"startDateStruct":38,"completionDateStruct":40,"leadSponsor":42,"locationsCount":4},"100636699","phase-2-a-study-evaluating-the-efficacy-and-safety-of-momelotinib-in-participants-with-vacuoles-e1-enzyme-x-linked-autoinflammatory-somatic-vexas-syndrome-100636699",false,"NCT07569081","A Study Evaluating the Efficacy and Safety of Momelotinib in Participants With Vacuoles, E1-enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome","A Randomized Phase 2\u002F3, Double-blind, Placebo Controlled Adaptive Study Evaluating the Efficacy and Safety of Momelotinib in Participants With VEXAS Syndrome","ATLAS","Inclusion Criteria:\n\n* Age greater than equal to (\\>=)18 years OR of legal age of consent in the jurisdiction in which the study is taking place, at the time of signing the Informed Consent Form.\n* Confirmed diagnosis of clinical VEXAS defined by:\n\n  1. Documented evidence of a canonical, pathogenic Ubiquitin-like modifier activating enzyme 1 (UBA1) mutation\n  2. Inflammatory manifestations: current or documented past involvement within 6 months of at least one organ system\n* Receiving glucocorticoid (GC) treatment (prednisone\u002Fprednisolone) for \\>=4 consecutive weeks for \\>=10 days prior to randomization.\n* A female participant is eligible to participate if she is not pregnant or breastfeeding and one of the following conditions applies:\n\n  1. Is a Participant of non-childbearing potential (PONCBP) OR\n  2. Is a Participant of childbearing potential (POCBP) and using a contraceptive method that is highly effective\n* Is capable of giving signed informed consent including compliance with the requirements and restrictions\n* Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2 at the time of screening.\n* Has adequate organ function\n\nExclusion Criteria:\n\n* More than 1 prior admission to an intensive care unit due to a VEXAS flare within the 6 months prior to randomization.\n* History of severe corticosteroid toxicity: uncontrolled concomitant cardiovascular, nervous system, pulmonary (including obstructive pulmonary disease), renal, hepatic, endocrine (including uncontrolled diabetes mellitus), psychiatric, osteoporosis\u002Fosteomalacia, glaucoma, corneal ulcers\u002Finjuries, nausea or vomiting or gastrointestinal disease.\n* High risk\u002Fvery high risk Myelodysplastic syndrome (MDS), according to the Revised International Prognostic Scoring System (IPSS-R) with overall risk score \\>3.5.\n* Peripheral blood blast counts \\>=10%.\n* Multiple myeloma (all stages) and other active plasma cell dyscrasias requiring treatment.\n* Malignancy (except disease under study including Lower-risk myelodysplastic syndrome \\[LR-MDS\\]) that has progressed or required active treatment within the past (24 months) except for basal cell or squamous cell carcinomas of the skin or in-situ carcinomas).\n* Uncontrolled intercurrent illness within 12 weeks prior to initiation of momelotinib.\n* Ongoing adverse reaction(s) from prior therapy that have not recovered to Grade \\\u003C=1 per NCI CTCAE v6.0 or to the Baseline status preceding prior therapy\n* Psychiatric illness, social situation, or any other condition that would limit informed consent and\u002For compliance with trial requirements or may interfere with the interpretation of study results, as judged by Investigator or Sponsor.\n* Has any clinically significant gastrointestinal conditions or abnormalities that may alter absorption or swallowing\n* Known contraindication or hypersensitivity to momelotinib and its metabolites, or any of their excipients.\n* Presence of peripheral neuropathy \\>=Grade 2 per NCI CTCAE v6.0.\n* Known history of disseminated mycobacterial infection.\n* Known positive status for human immunodeficiency virus (HIV).\n* Positive QuantiFERON (or other interferon gamma release assay) during Screening.\n* Unable to receive any Pneumocystis jiroveci pneumonia (PJP) medical prophylaxis\n* Known clinically significant anemia due to iron, vitamin B12 or folate deficiencies, or autoimmune or hereditary hemolytic anemia, gastrointestinal bleeding, or thalassemia.\n* More than 1 prior line of VEXAS directed therapy before or after VEXAS diagnosis or other medical condition.\n* Use of the following treatments within the noted time periods referenced from date of randomization:\n\n  1. VEXAS-directed therapies (washout period) up to 5 half-lives or up 14 days if half-life is \\\u003C3 days\n  2. Other non-GC anti-inflammatory therapies: for non-biologics): 14 days or five half-lives, whichever is longer; for biologics): 28 days or two half-lives whichever is longer.\n  3. Hematologic support therapy (e.g., ESAs, danazol, luspatercept, G-CSF): 4 weeks\n  4. Cell-depleting therapies such as anti-CD20 (rituximab): 12 months\n  5. Investigational agent from a class not otherwise specified: 5 half-lives or 60 days, whichever is longer\n* GC use for conditions other than VEXAS, which would interfere with adherence to the fixed GC taper regimen and\u002For to assessment of efficacy.\n* Chronic use of systemic corticosteroids for \\>4 years or inability to withdraw corticosteroid treatment\n* Planned allogeneic HSCT for MDS or VEXAS, within 1 year.\n* Any major surgery within 28 days prior to randomization.\n* Prior allogeneic\u002Fautologous stem cell transplant or solid organ transplant (other than corneal).\n* Presence of peripheral neuropathy \\>=Grade 2 per NCI CTCAE v6.0.\n* Hepatitis B or C active infection, unless protocol defined criteria are met.\n* Any of the following conditions within 6 months prior to randomization:\n\n  1. Unstable angina pectoris\n  2. Symptomatic congestive heart failure\n  3. Uncontrolled cardiac arrhythmia\n  4. QTc \\>450 msec or QTc \\>480 msec for participants with bundle branch block.","ALL","18 Years",{"count":20,"type":21},136,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE2","PHASE3","This study will assess the efficacy and safety of momelotinib in participants with a diagnosis of VEXAS.",[28],"VEXAS Syndrome",[28,30,31,32,15],"Momelotinib","Adaptive","Ubiquitin-like modifier activating enzyme 1 mutation","NOT_YET_RECRUITING","2026-05-29",{"date":36,"type":37},"2026-06-01","ACTUAL",{"date":39,"type":21},"2026-08-05",{"date":41,"type":21},"2031-06-04",{"name":43,"class":44},"GlaxoSmithKline","INDUSTRY",{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":49,"acronym":4,"eligibilityCriteria":50,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":51,"targetDuration":53,"studyType":54,"phases":4,"briefSummary":55,"conditions":56,"keywords":57,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":60,"lastUpdatePostDateStruct":61,"startDateStruct":63,"completionDateStruct":65,"leadSponsor":67,"locationsCount":70},"100545097","multicenter-interdisciplinary-national-vexas-registry-with-accompanying-biomaterial-collection-100545097","NCT06377462","Multicenter, Interdisciplinary National VEXAS Registry With Accompanying Biomaterial Collection","Inclusion Criteria:\n\n* Patients with established or suspected (clinical and hematological criteria) VEXAS Syndrome\n* Age ≥18 years\n* Signed informed consent form\n\nExclusion Criteria:\n\n* patients who are not in a position to understand the nature and scope of participation in this register",{"count":52,"type":21},500,"5 Years","OBSERVATIONAL","The aim is rapid collection of real-life data on the epidemiology, treatment and disease course in patients with VEXAS syndrome during routine clinical practice and collect biomaterials to evaluate genotype-phenotype associations, determine optimal treatment schedule, identify diagnostic features and biomarkers",[28],[58],"VEXAS","RECRUITING","2026-05-13",{"date":62,"type":37},"2026-05-15",{"date":64,"type":37},"2024-03-13",{"date":66,"type":21},"2030-12-31",{"name":68,"class":69},"Technische Universität Dresden","OTHER",17,{"id":72,"slug":73,"hasResults":11,"nctId":74,"briefTitle":75,"officialTitle":76,"acronym":4,"eligibilityCriteria":77,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":78,"targetDuration":4,"studyType":22,"phases":80,"briefSummary":81,"conditions":82,"keywords":83,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":94,"lastUpdatePostDateStruct":95,"startDateStruct":97,"completionDateStruct":99,"leadSponsor":101,"locationsCount":103},"100576213","phase-2-a-study-to-assess-the-effectiveness-and-safety-of-pacritinib-in-patients-with-vexas-syndrome-paxis-100576213","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.",{"count":79,"type":21},78,[24],"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.",[58,28],[84,85,86,87,88,89,90,91,92,93],"Vacuoles","E1 Ubiquitin-activating enzyme","X-linked","Autoinflammatory","Somatic syndrome","UBA1","Hematologic neoplasms","Myelodysplastic Syndromes","Pacritinib","Myeloid progenitors","2026-04-23",{"date":96,"type":37},"2026-04-24",{"date":98,"type":37},"2025-05-28",{"date":100,"type":21},"2028-05-22",{"name":102,"class":44},"Swedish Orphan Biovitrum",39,{"id":105,"slug":106,"hasResults":11,"nctId":107,"briefTitle":108,"officialTitle":109,"acronym":110,"eligibilityCriteria":111,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":112,"targetDuration":114,"studyType":54,"phases":4,"briefSummary":115,"conditions":116,"keywords":132,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":134,"lastUpdatePostDateStruct":135,"startDateStruct":137,"completionDateStruct":139,"leadSponsor":141,"locationsCount":143},"100513772","clonal-hematopoiesis-of-immunological-significance-100513772","NCT05969821","Clonal Hematopoiesis of Immunological Significance","Immuno-inflammatory Manifestations With or Without Clonal Hematopoiesis: Ambispective Cohort Study","CHIS","Inclusion Criteria:\n\n* Age \\>=18 years old;\n* Confirmed dysimmune manifestations: clinical or biological abnormality or systemic disease;\n* Presence or absence of myeloid or lymphoid blood disease according to World Health Organization (WHO) classification\n\nExclusion Criteria:\n\n* Persons benefiting from special protection: adults under guardianship and curatorship;\n* People hospitalized without their consent and not protected by law; persons deprived of liberty;\n* Persons not affiliated to the social security system",{"count":113,"type":21},5000,"10 Years","Ambispective, national, multicenter observational cohort study aimed at characterizing the satellite dysimmune manifestations of clonal hematopoiesis, including Vexas (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic) syndrome.",[117,118,119,120,121,122,123,124,125,126,91,127,128,129,130,131],"Immune System Diseases","Autoimmune Diseases","Inflammation","Autoinflammatory Diseases","Vexas Syndrome","Hematopoiesis Clonal","Clonal Hematopoiesis of Indeterminate Potential","Hematologic Diseases","Myelodysplastic-Myeloproliferative Diseases","Leukemia Myelomonocytic Chronic","Myeloproliferative Disorders","Lymphoproliferative Disorders","Lymphoma","Leukemia","Monoclonal Gammopathy of Undetermined Significance",[119,120,133,118,123,125,128,131],"Vexas syndrome","2026-03-19",{"date":136,"type":37},"2026-03-23",{"date":138,"type":21},"2026-04",{"date":140,"type":21},"2045-09",{"name":142,"class":69},"Assistance Publique - Hôpitaux de Paris",1,{"id":145,"slug":146,"hasResults":11,"nctId":147,"briefTitle":148,"officialTitle":149,"acronym":4,"eligibilityCriteria":150,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":151,"targetDuration":4,"studyType":22,"phases":153,"briefSummary":155,"conditions":156,"keywords":158,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":159,"lastUpdatePostDateStruct":160,"startDateStruct":162,"completionDateStruct":164,"leadSponsor":166,"locationsCount":143},"100557443","phase-1-pacritinib-in-vacuoles-e1-ubiqutin-activating-enzyme-x-linked-autoinflammatory-somatic-vexas-syndrome-100557443","NCT06538181","Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome","A Phase 1 Study of Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome","Inclusion Criteria:\n\n* Patients must have UBA1 mutation with a variant allele frequency (VAF) of ≥ 2% detected on a next generation sequencing panel and have at least one of the following current or past clinical manifestation of VEXAS syndrome, as determined by the attending physician:\n\n  * skin rash\n  * vasculitis\n  * chondritis\n  * ocular\u002Forbital inflammation (e.g., uveitis\u002Firitis, episcleritis)\n  * genitourinary inflammation (e.g., epididymitis\u002Forchitis)\n  * arthritis\u002Farthralgias\n  * pulmonary inflammation (e.g., alveolitis\u002Fpleural effusion,)\n  * fever\n  * thrombosis\n  * splenomegaly\n  * hepatomegaly\n  * myocarditis or pericarditis\n  * cytopenias (defined as hemoglobin \\\u003C11 g\u002FdL, platelets \\\u003C 100 X 10\\^9 \u002FL, OR absolute neutrophil count \\\u003C1.0 X 10\\^9 \u002FL).\n* Patients with VEXAS syndrome who have never been treated with a JAK-I will be eligible to enroll on study. A stable corticosteroid dose must be maintained for at least 14 days prior to start of pacritinib.\n* Patients who have previously been treated with a JAK-I other than pacritinib, or who are currently being treated with a JAK-I other than pacritinib, may be eligible after a 28 day washout if either (i) their symptoms are not adequately controlled, as determined by the treating physician, or (ii) they have been unable to taper corticosteroids to an equivalent of \\\u003C10 mg prednisone\u002Fday, and in the opinion of the treating physician, may benefit from a change in JAK-I. A stable corticosteroid dose must be maintained for at least 14 days prior to start of pacritinib.\n* At least 18 years of age.\n* ECOG performance status ≤ 3.\n* Organ function as defined below:\n\n  * Absolute neutrophil count ≥ 0.5 K\u002Fcumm\n  * Platelets ≥ 25 K\u002Fcumm\n  * PT\u002FPTT \\\u003C2.5 X upper limit of normal (ULN)\n  * Total bilirubin ≤ 1.5 x IULN\n  * AST(SGOT)\u002FALT(SGPT) ≤ 3.0 x IULN\n  * Creatinine clearance ≥ 30 mL\u002Fmin by Cockcroft-Gault\n* QTcF \\\u003C 480 msec.\n* The effects of pacritinib on the developing human fetus are unknown. For this reason and because pacritinib was shown to be teratogenic in animal studies, women of childbearing potential and men must agree to use highly effective contraception prior to study entry, for the duration of study participation, and for 30 days after completion of study treatment. Hormonal contraception is no longer considered highly effective alone as pacritinib is a CYP3A4 inducer and accelerated progesterone metabolism. The contraceptive methods considered highly effective for WOCBP who receive pacritinib are intrauterine devices, bilateral tubal occlusion, vasectomized partner, or total sexual abstinence. Hormonal contraceptives (e.g., Depo-Provera) alone are not considered highly effective methods of contraception on their own when in treatment with pacritinib; such hormonal contraceptives must be combined with an additional barrier method (condom, diaphragm with spermicidal gel, or condoms with spermicides to be considered highly effective. Highly effective contraceptive methods in males include vasectomy, sexual abstinence, and condoms when combined with their partner using a highly effective method (including oral contraceptives).\n* Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.\n* Ability to understand and willingness to sign an IRB approved written informed consent document or that of legally authorized representative, if applicable.\n* Patients with myelodysplastic neoplasms (MDS) or plasma cell dyscrasias are eligible if they are not undergoing active treatment. Supportive care is permitted.\n\nExclusion Criteria:\n\n* Prior use of pacritinib.\n* Use of another JAK inhibitor within 28 days of C1D1 of pacritinib.\n* Currently receiving any other investigational agents. Patients may be eligible after 28 day washout.\n* Thrombotic events (arterial or venous) within 60 days prior to enrollment.\n* Any recent clinically significant bleeding within at least 7 days prior to enrollment.\n* Any active or acute infection.\n* History of malignancy within the prior 2 years, with the exception of MDS and plasma cell dyscrasias, or non-melanoma skin cancers that have been treated.\n* History of clinically significant cardiovascular disease or clinically significant abnormalities in rhythm or conduction during screening EKG, including severe cardiac events, symptomatic congestive heart failure, unstable angina pectoris, uncontrolled cardiac arrhythmia, or heart failure.\n* Currently receiving immunosuppressants (other than corticosteroids), disease-modifying antirheumatic drugs (DMARDs), or biologic cytokine inhibitors. Patients may be eligible after 28 day washout.\n* A history of allergic reactions attributed to compounds of similar chemical or biologic composition to pacritinib.\n* Concurrent use of strong CYP3A4 inhibitors or inducers. Patients may be eligible after washout period of 28 days (or 5 half-lives, whichever is shorter).\n* Diagnosis or history of moderate (Child-Pugh B) and severe hepatic impairment (Child-Pugh C).\n* Pregnant and\u002For breastfeeding. Women of childbearing potential must have a negative serum pregnancy test within 7 days of C1D1 or negative urine pregnancy test within 3 days of C1D1.\n* Known active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV).\n* Patients with latent tuberculosis. Patients must have a negative T-Spot during screening to be eligible.",{"count":152,"type":21},15,[154],"PHASE1","VEXAS (vacuoles, E1 ubiqutin-activating enzyme, X-linked, autoinflammatory, somatic syndrome) is a recently described disorder with severe hematologic and rheumatologic manifestations caused by somatic variants in the ubiquitin- activating enzyme gene, UBA1, that is acquired in hematopoietic progenitor cells. Patients are often debilitated by autoinflammatory symptoms and there is currently no standard of care available. There is a clinically unmet need for better therapies in VEXAS Syndrome. There have been no prospective clinical trials of JAK-I in VEXAS syndrome. The investigators hypothesize that pacritinib, as a JAK2\u002FIRAK1 inhibitor with a manageable safety profile in myelofibrosis patients with thrombocytopenia, will improve the autoinflammatory and hematologic manifestations of VEXAS syndrome with a tolerable toxicity profile.\n\nThe investigators propose a single arm, pilot Phase 1 study evaluating the safety and tolerability of pacritinib in patients with VEXAS syndrome with an initial safety run-in phase of 6 patients treated with pacritinib 200mg twice daily (BID) on days 1-28 of a continuous 28 day cycle. If no more than 1 patient experiences a dose-limiting toxicity (DLT), the investigators will enroll an expansion cohort to gain additional toxicity and efficacy data, for a total enrollment of 15 patients. If more than 1 patient experiences a DLT during the safety run-in phase, the investigators will decrease the dose to 100 mg BID, and if no more than 1 of 6 patients experiences a DLT, the investigators will complete the expansion cohort as above for up to a total enrollment of 15 patients. If more than 1 patient experiences a DLT at 100 mg BID, the investigators will discontinue the study. Patients will be treated for up to 12 cycles.",[157,58,121],"E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome",[58,87,89,92],"2025-07-11",{"date":161,"type":37},"2025-07-16",{"date":163,"type":37},"2025-02-13",{"date":165,"type":21},"2029-02-28",{"name":167,"class":69},"Washington University School of Medicine",{"id":169,"slug":170,"hasResults":11,"nctId":171,"briefTitle":172,"officialTitle":173,"acronym":174,"eligibilityCriteria":175,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":176,"targetDuration":114,"studyType":54,"phases":4,"briefSummary":178,"conditions":179,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":190,"lastUpdatePostDateStruct":191,"startDateStruct":193,"completionDateStruct":195,"leadSponsor":197,"locationsCount":199},"100454682","autoinflammatory-disease-alliance-registry-aida-100454682","NCT05200715","AutoInflammatory Disease Alliance Registry (AIDA)","Development of an International Multicenter Registry of Patients With Monogenic and Polygenic Autoinflammatory Diseases Aimed at Clinical and Therapeutical Data Collection and Analysis","AIDA","Inclusion Criteria:\n\n* to be diagnosed with a monogenic AID according to the clinical phenotype and the detection of a confirmative genotype;\n* to be diagnosed with clinical familial Mediterranean fever or Behçet's disease or Still disease or PFAPA syndrome or Schnitzler's disease or CRMO according to the corresponding clinical diagnostic and\u002For classification criteria;\n* to be diagnosed with undifferentiated systemic AID;\n* to be diagnosed with non-infectious uveitis according to the standardization for uveitis nomenclature (SUN) criteria;\n* to be diagnosed with anterior or posterior non-infectious scleritis;\n* to be diagnosed with spondyloarthritis according to ASAS and\u002For New York criteria;\n* to be diagnosed with Castleman disease;\n\nExclusion Criteria:\n\n\\- informed consent\u002Fassent not provided by the patient and\u002For his\u002Fher legal representative.",{"count":177,"type":21},3500,"Autoinflammatory diseases (AID) are clinical entities characterized by recurrent inflammatory attacks in absence of infection, neoplasm or deregulation of the adaptive immune system. Among them, hereditary periodic syndromes, also known as monogenic AID, represent the prototype of this disease group, caused by mutations in genes involved in the regulation of innate immunity, inflammation and cell death. Based on recent experimental acquisitions in the field of monogenic AID, several immunologic disorders have been reclassified as polygenic\u002Fmultifactorial AID, sharing pathogenetic and clinical features with hereditary periodic fevers. This has paved the way to new treatment targets for patients suffering from rare diseases of unknown origin, including Behçet's disease, Still disease, Schnitzler's disease, PFAPA (periodic fever, aphthous stomatitis, pharyngitis and cervical adenitis) syndrome, chronic recurrent multifocal osteomyelitis (CRMO), non-infectious uveitis and scleritis. Gathering information on such rare conditions is made difficult by the small number of patients, along with the difficulty of obtaining an accurate diagnosis in non-specialized clinical settings.\n\nIn this context, the AIDA project promotes international collaboration among clinical centres to develop a permanent registry aimed at collecting demographic, genetic, clinical and therapeutic data of patients affected by monogenic and polygenic AID, in order to expand the current knowledge of these rare conditions.",[180,181,182,183,184,185,186,187,121,188,189],"Hereditary Autoinflammatory Diseases","Schnitzler Syndrome","Behcet Syndrome","PFAPA Syndrome","Still Disease","Autoinflammatory Syndrome, Unspecified","Uveitis","Scleritis","Spondyloarthritis (SpA)","Castleman Disease","2025-07-07",{"date":192,"type":37},"2025-07-10",{"date":194,"type":37},"2020-08-06",{"date":196,"type":21},"2030-08-06",{"name":198,"class":69},"University of Siena",112]