[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"ipex\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:ipex":40},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,58],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":23,"conditions":24,"keywords":4,"overallStatus":45,"whyStopped":4,"lastUpdateSubmitDate":46,"lastUpdatePostDateStruct":47,"startDateStruct":50,"completionDateStruct":52,"leadSponsor":54,"locationsCount":57},"100431799","conception-of-a-diagnosis-prognosis-and-therapeutic-decision-tool-for-patients-with-autoimmunity-and-inflammation-100431799",false,"NCT04902807","Conception of a Diagnosis, Prognosis and Therapeutic Decision Tool for Patients With Autoimmunity and Inflammation","ATRACTion","Inclusion Criteria for controls (patients relatives and unrelated subjects):\n\n* Individuals aged\\\u003C18 y\u002Fo.\n* Individuals \\> 6 kg\n* Individuals not affected by an immune-related disease or not affected by cancer\n* Individuals whose parents have signed an enlightened consent.\n\nInclusion criteria for patients\n\n* Individuals with health insurance.\n* Patients treated at Necker hospital with PIDs and autoimmunity\u002Finflammation related to known genetic defects (cytopenia, Enteropathy Inflammatory bowel disease (IBD), Systemic Lupus Erythematosus (SLE), Juvenile Idiopathic Arthritis (JIA), Familial Hemophagocytic Lymphohistiocytosis (FHL), chronic EBV infection associated (Ca-EBV) with EBV-infected T and\u002For Natural Killer (NK) cells and with a high risk to develop macrophage activation syndrome similar to FHL. See table below for diagnosis inclusion criteria.\n* Individuals aged\\\u003C18 y\u002Fo.\n* Individuals \\> 9 kg\n* Patients whose parents have signed an enlightened consent.\n\nExclusion Criteria:\n\n* Intake of antibiotics within 2 weeks prior inclusion\n* Absence of parent's or child consent form\n* Cytotoxic cancer treatments\n* antiviral treatments (HIV, hepatitis …)\n* Short term life-threatening conditions\n* Individuals placed under judicial protection","ALL","1 Year","18 Years",{"count":20,"type":21},500,"ESTIMATED","OBSERVATIONAL","The main objective of this study is to generate diagnosis and therapeutic-decision tools through the identification of molecular causes of PIDs with autoimmunity\u002Finflammation and the variability in disease outcome at the transcriptional level using a combination of omics signatures (transcriptomics, epigenomics, proteomics, metagenomics, metabolomics and lipidomics).",[25,26,27,28,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44],"Autoimmune Lymphoproliferative Syndrome","Autoimmune Cytopenia","Autoimmune Diseases","Autoimmune Anemia","Autoimmune Thrombocytopenia","Autoimmune Hepatitis","Autoimmune Diabetes","Autoimmune Rheumatologic Disease","Systemic Lupus Erythematosus","Juvenile Idiopathic Arthritis","Hemophagocytic Lymphohistiocytoses","EBV Lymphoproliferation","RAS-Associated Autoimmune Leucoproliferative Disease","Primary Immunodeficiency","APECED","IPEX","BENTA","Enteropathy, Autoimmune","Combined Immunodeficiency","IBD","RECRUITING","2025-09-02",{"date":48,"type":49},"2025-09-08","ACTUAL",{"date":51,"type":49},"2021-09-07",{"date":53,"type":21},"2026-06",{"name":55,"class":56},"Institut National de la Santé Et de la Recherche Médicale, France","OTHER_GOV",1,{"id":59,"slug":60,"hasResults":11,"nctId":61,"briefTitle":62,"officialTitle":63,"acronym":4,"eligibilityCriteria":64,"healthyVolunteers":11,"sex":65,"minAge":66,"maxAge":67,"enrollmentInfo":68,"targetDuration":4,"studyType":70,"phases":71,"briefSummary":73,"conditions":74,"keywords":75,"overallStatus":45,"whyStopped":4,"lastUpdateSubmitDate":86,"lastUpdatePostDateStruct":87,"startDateStruct":89,"completionDateStruct":91,"leadSponsor":93,"locationsCount":57},"100457810","phase-1-cd4lvfoxp3-in-participants-with-ipex-100457810","NCT05241444","CD4^LVFOXP3 in Participants With IPEX","Phase 1 Study of Autologous CD4^LVFOXP3 in Participants With Immune Dysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) Syndrome","Inclusion Criteria:\n\n* Body weight greater than 8 kg, unless assessed as able to tolerate leukapheresis\n* FOXP3 gene mutation\n* Medical history of progressive symptoms of IPEX with persistency of some symptoms and\u002For signs requiring immune suppressive medication. The participant may or may not be on immunosuppression at time of starting the study.\n* Uncontrolled IPEX disease but unable to tolerate immune suppressive medication\n* Recurrent IPEX symptoms, requiring immune suppressive medications, in participants who have had prior allogeneic (allo) blood stem cell transplantation (HSCT).\n* ≥ 50% Performance rating on Lansky\u002FKarnofsky Scale\n* Organ and marrow function within acceptable levels of function\n* Absence of ongoing infections\n* Must be able to consent if an adult\n\nExclusion Criteria:\n\n* Medical instability\n* Less than 6 months life expectancy\n* Inability to meet limits for steroid dosing\n* Eligible for an HLA matched sibling or matched unrelated donor blood stem cell transplant, and be willing to undergo transplant.\n* Unrelated or comorbid disease\n* Allergy to any study medication, product, or intervention\n* Currently receiving another experimental treatment\n* History of malignancy, unless disease free for at least 2 years, with the exception of non melanoma skin cancer or carcinoma in situ","MALE","4 Months","35 Years",{"count":69,"type":21},30,"INTERVENTIONAL",[72],"PHASE1","This first-in-human, Phase 1 clinical trial will test the feasibility of the manufacturing and the safety of the administration of CD4\\^LVFOXP3 in up to 30 evaluable human participants with IPEX and evaluate the impact of the CD4\\^LVFOXP3 infusion on the disease.",[40],[76,77,78,79,80,81,82,83,84,85],"Treg cells","Regulatory T cells","Gene therapy","autoimmunity","Immune Dysregulation Polyendocrinopathy Enteropathy X-linked","FOXP3","Lentiviral","CD271","NGFR","immune suppression","2025-05-19",{"date":88,"type":49},"2025-05-22",{"date":90,"type":49},"2022-03-22",{"date":92,"type":21},"2037-02",{"name":94,"class":95},"Bacchetta, Rosa, MD","OTHER"]