[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"the-immune-dysregulation-polyendocrinopathy-enteropathy-x-linked-syndrome-is-a-primary-immunodeficiency-caused-by-pathogenic-variants-in-forkhead-box-protein-3\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:the-immune-dysregulation-polyendocrinopathy-enteropathy-x-linked-syndrome-is-a-primary-immunodeficiency-caused-by-pathogenic-variants-in-forkhead-box-protein-3":29},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":27,"conditions":28,"keywords":30,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":41,"startDateStruct":44,"completionDateStruct":46,"leadSponsor":48,"locationsCount":5},"100053839","phase-1-study-evaluating-a-gene-therapy-for-ipex-syndrome-through-the-expression-of-foxp3-on-deficient-t-cells-to-produce-tregs-like-100053839",false,"NCT07697118","Study Evaluating a Gene Therapy for IPEX Syndrome Through the Expression of FOXP3 on Deficient T Cells to Produce Tregs-like.","A Phase I\u002FII Open-label, Non-randomized, Monocentric, Single-arm Study Evaluating the Safety and Efficacy of Induced CD4+ Treg by LV Vector Transduction Expressing the FOXP3 cDNA (FOXP3-T4) in Patients With IPEX Syndrome","THERIPEX","Inclusion Criteria:\n\n* Male patients only\n* Patients aged from 1 - 45 years of age (the first three patients will be aged between 10 - 45 years of age)\n* Patient with IPEX syndrome caused by mutation of the FOXP3 gene\n* Patients are eligible from the second line of treatment onward, even those under controlled disease\n* Patient with recurrent IPEX symptoms, under immune suppressive medications\n* Patient for whom HSCT is not feasible or when no suitable compatible donor is available\n* Patients who have had prior allogeneic blood stem cell transplantation (HSCT) with engraftment failure defined as no intake of donor cells\n* Patient or parental, guardian's patient signed informed consent\n* Male participants of reproductive potential with a partner of childbearing potential (WOCBP): willing to use an effective method of contraception during the trial and for at least 12 months post-infusion\n* Affiliation to a French or European social security scheme\n\nExclusion Criteria:\n\n* Unwillingness to return for follow-up during the 2-year study and during the 15 years of long term follow up study.\n* Patient with short life expectancy\n* Patient on AME (state medical aid) (unless exemption from affiliation).\n* Diagnosis of a significant psychiatric disorder of the patient that could seriously impede the ability to participate in the study.\n* Eligible for an HLA matched sibling or matched unrelated donor blood stem cell transplant (HLA 10\u002F10) and be willing to undergo transplant.\n* Patients with uncontrolled or ongoing active infections.\n* HIV-1 or 2 or HTLV-1 infections.\n* Patients with severe IPEX clinical presentation needing a rapid allogeneic HSCT treatment within 3 months.\n* Patients with known history of hypersensitivity to IL-2 or any component of the formulation (mannitol, sodium lauryl sulfate, monosodium phosphate dehydrate, disodium phosphate dehydrate, glucose.","MALE","1 Year","45 Years",{"count":21,"type":22},5,"ESTIMATED","INTERVENTIONAL",[25,26],"PHASE1","PHASE2","The purpose of this study is to evaluate the safety and efficacy of FOXP3-T4 (an autologous gene therapy) alone or in combination with low-dose IL-2 for the treatment of IPEX syndrome. The therapy involves the transplantation of autologous CD4+ T-cells transduced ex vivo with the LV-EF1a-FOXP3-LNGFR lentiviral vector. The study follows a staggered approach: the first two patients will receive FOXP3-T4 monotherapy. Subsequent patients will receive FOXP3-T4 followed if needed by low-dose IL-2 treatment consisting of a daily dose for 5 days, then weekly administrations for 3 months.\n\nThe study aims to stabilize autoimmune manifestations and potentially cure the underlying disease, ultimately allowing for the discontinuation of ongoing immunosuppressive treatments.",[29],"The Immune Dysregulation Polyendocrinopathy Enteropathy X-linked Syndrome is a Primary Immunodeficiency Caused by Pathogenic Variants in Forkhead Box Protein 3",[31,32,33,34,35,36,37,38],"Autoimmune diseases","Genetic diseases","Gene therapy","Lentiviral vector","Immune dysregulation Polyendocrinopathy Enteropathy X-linked","Autoimmunity-Immunodeficiency Syndrome","Forkhead Box Protein 3","Low-dose IL-2","NOT_YET_RECRUITING","2026-07-06",{"date":42,"type":43},"2026-07-13","ACTUAL",{"date":45,"type":22},"2026-09",{"date":47,"type":22},"2029-01",{"name":49,"class":50},"Assistance Publique - Hôpitaux de Paris","OTHER"]