[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"vexas-syndome\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:vexas-syndome":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,45],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":28,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100600549","phase-2-momelotinib-in-vexas-syndrome-100600549",false,"NCT07098936","Momelotinib in VEXAS Syndrome","A Single-arm Phase II With safety-run-in Multicenter Study of Momelotinib in Patients With VEXAS Syndrome With or Without Associated Myelodysplastic Syndrome","Inclusion Criteria:\n\n* ECOG (Eastern Cooperative Oncology Group) performance status 0-2 at the time of screening\n* Age ≥ 18 years\n* Written informed consent\n* Diagnosis of VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic) syndrome with UBA1 (Ubiquitin Like Modifier Activating Enzyme 1) mutation and clinically symptomatic disease requiring immunosuppressive treatment and at least 10mg\u002Fd of glucocorticoids\n* Patients with uncontrolled symptoms related to VEXAS with prior treatment line(s) (including steroids)\n* Patients refractory\u002Fdependent to steroids\n* Single concomitant steroids therapy (e.g., prednisone or equivalent) at the time of inclusion is allowed\n* For patients treated with other immunosuppressive\u002Fimmunomodulatory therapy than glucocorticoids, a wash out period of 28 days is required prior momelotinib onset\n* Erythropoietin\u002Fluspatercept used as a growth factor treatment is not allowed 28 days prior enrollment\n* Adequate liver function (serum transaminases ≤ 3 x ULN (Upper Limits of Normal), Bilirubin ≤ 1.5 x ULN (isolated bilirubin \\> 1.5 x ULN is acceptable if bilirubin fractionated and direct bilirubin \\\u003C 35%)\n* Adequate renal function (creatinine clearance with MDRD (Modification of Diet in Renal Disease) formula \\> 30 ml\u002Fmin)\n* Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) must:\n\n  1. Have a negative serum or urine pregnancy test within 24 hours prior to beginning treatment on this study. Lactating patients are excluded.\n  2. Agree to use, and to be able to comply with, effective contraception without interruption, 4 weeks before starting study drug throughout the entire duration study drug therapy (including doses interruptions) and for 12 weeks after the end of the study drug therapy.\n  3. Agree to learn about the procedures for preservation of egg before starting treatment.\n* Male patients must:\n\n  1. Agree the need for the use of a condom if engaged in sexual activity with a woman of childbearing potential during the entire period of treatment, even if disruption of treatment and during 12 weeks after end of treatment.\n  2. Agree to learn about the procedures for preservation of sperm before starting treatment.\n\nExclusion Criteria:\n\n* Patients with MDS (Myelodysplastic syndrome) scheduled for allogeneic stem cell transplant or high risk MDS according to IWG (International Working Group) 2023\n* Patients who are or have been already treated with Janus Kinase (JAK) inhibitors for VEXAS syndrome or another indication\n* Patients who are unable to receive a starting daily dose of momelotinib of at least 100 mg\n* Subjects with any other active malignancies are not eligible, except for the following: adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer from which subject has been disease-free for at least 3 years\n* Uncontrolled congestive heart failure (New York Heart Association Classification 3 or 4), angina, myocardial infarction, cerebrovascular accident, coronary\u002Fperipheral artery bypass graft surgery, transient ischemic attack, or pulmonary embolism within 12 weeks prior to initiation of momelotinib\n* Known infection with acute and chronic active Human Immunodeficiency Virus, Hepatitis B Virus, Hepatitis C Virus\n* Any medical or psychiatric condition not allowing the informed consent of the subject\n* Presence of clinically meaningful active bacterial, fungal, parasitic or viral infection which requires therapy\n* Previous history of Progressive Multifocal Leuko-encephalopathy\n* Active gastrointestinal conditions that may affect absorption\n* No affiliation to a health insurance system\n* Known hypersensitivity to the study investigational medicinal product, the metabolites, or formulation excipients","ALL","18 Years",{"count":19,"type":20},57,"ESTIMATED","INTERVENTIONAL",[23],"PHASE2","Multicenter, phase II trial with safety run-in to evaluate the efficacy and safety of momelotinib in patients with VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic) syndrome with or without associated myelodysplastic syndrome (MDS).\n\nThe study will consist of two consecutive steps, a dose-finding safety run-in and a single-arm prospective phase II.\n\nDuring safety run-in phase, three fixed dose levels will be tested according to a 3+3 design, using cohorts of size 3 in order to establish the maximum tolerated dose.\n\nAfter this safety run-in phase, patients included in phase II will be treated with momelotinib at the maximum tolerated dose preliminary fixed.\n\nPatients included in the phase II will receive momelotinib continuously until disease progression or loss of response, at physician's discretion.\n\nAll patients included in the study will receive glucocorticoids (prednisone\u002Fprednisolone equivalent) at baseline (at least \\> 10mg\u002Fday).\n\nResponse assessment regarding VEXAS related symptoms will be evaluated after 4, 12, 24 and 48 weeks. Response assessment regarding MDS features will be evaluated at 12 and 24 weeks.",[26,27],"VEXAS Syndome","Myelo Dysplastic Syndrome",[29,30,31],"VEXAS syndrome","Momelotinib","Myelodysplastic syndrome","RECRUITING","2025-12-03",{"date":35,"type":36},"2025-12-10","ACTUAL",{"date":38,"type":36},"2025-11-25",{"date":40,"type":20},"2028-11",{"name":42,"class":43},"Groupe Francophone des Myelodysplasies","OTHER",11,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":52,"sex":16,"minAge":53,"maxAge":4,"enrollmentInfo":54,"targetDuration":4,"studyType":56,"phases":4,"briefSummary":57,"conditions":58,"keywords":62,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":5},"100516425","autoimmune-and-autoinflammatory-genetics-study-100516425","NCT06004349","Autoimmune and Autoinflammatory Genetics Study","Natural History and Genetics of VEXAS Syndrome and Related Autoinflammatory Syndromes","Inclusion Criteria:\n\nIn order to be eligible to participate in this study as a subject with known or suspected autoinflammatory disease, an individual must meet all of the following criteria:\n\n* Stated willingness to participate in study procedures (which at the very least includes providing a mail-in blood or saliva sample for genetic analysis);\n* Regardless of sex assigned at birth, at least one month of age;\n* A medical history that, in the expert opinion of the PI and study team, is consistent with the possibility of autoinflammatory disease or known diagnosis of an autoinflammatory disease, such as VEXAS syndrome; and\n* Ability of the subject, parents (in the case of children), or Legally Authorized Representative to understand and the willingness to sign a written informed consent document.\n\nIn order to be eligible to participate in this study as a family member of a subject with known or suspected autoinflammatory disease, an individual must meet all of the following criteria:\n\n* Stated willingness to participate in study procedures (which at the very least includes providing a mail-in sample for genetic analysis);\n* Regardless of sex assigned at birth, at least one month of age;\n* Relationship, either by blood or marriage, to an individual enrolled or about to be enrolled in the study with known or suspected autoinflammatory disease;\n* Likelihood, in the expert opinion of the PI and study team, that analysis of a sample from the individual would advance genetic or functional analysis of the affected relative's possible autoinflammatory condition; and\n* Ability of the subject, parents (in the case of children), to understand and the willingness to sign a written informed consent document.\n\nIn order to be eligible to participate in this study as a healthy volunteer, an individual must meet all of the following criteria:\n\n* Stated willingness to participate in study procedures for healthy volunteers;\n* Regardless of sex assigned at birth, at least five years old, and not pregnant (by history of a missed menstrual period);\n* Likelihood, in the expert opinion of the PI that a sample from the individual would advance the functional analysis of an autoinflammatory condition under study; and\n* Ability of the subject to understand and the willingness to sign a written informed consent document by a capacity assessment provided by the PI and study team.\n\nExclusion Criteria:\n\nFor any of the three categories of subjects, an individual will be excluded from participation in this study for the following reasons:\n\nProbands: an individual will not be enrolled as a proband if the study team has a low suspicion of having an autoinflammatory disease or a genetic cause for an autoinflammatory disease.\n\nFamily Members: an individual will not be enrolled as a family member if the study team believes they may have an autoinflammatory disease, in which case, they will be enrolled as a proband.\n\nHealthy controls: an individual will not be enrolled as a healthy control if they have an autoinflammatory disease, or any condition that may mimic an autoinflammatory disease, such as hematologic malignancy, rheumatologic disease.",true,"1 Month",{"count":55,"type":20},1500,"OBSERVATIONAL","This is an exploratory natural history protocol that will enroll patients with known genetic diseases, such as VEXAS syndrome, or as yet undiagnosed disorders of inflammation with the goal of improving our understanding of disease processes. Blood, saliva, hair, nail, or buccal samples may be collected for genetic analysis, blood samples will be obtained for immunologic and other functional studies, and a small number of subjects may undergo skin biopsy.",[26,59,60,61],"Autoinflammatory Disease","Autoimmune Disease","Rheumatic Disease",[63],"VEXAS syndrome (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic)","2025-07-08",{"date":66,"type":36},"2025-07-09",{"date":68,"type":36},"2022-04-15",{"date":70,"type":20},"2032-04-05",{"name":72,"class":43},"NYU Langone Health"]