[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100053839":3},{"organization":4,"armGroups":7,"interventions":15,"overallOfficials":28,"centralContacts":33,"locations":45,"responsibleParty":62,"collaborators":64,"id":67,"slug":68,"hasResults":69,"nctId":70,"briefTitle":71,"officialTitle":72,"acronym":73,"eligibilityCriteria":74,"healthyVolunteers":69,"sex":75,"minAge":76,"maxAge":77,"enrollmentInfo":78,"targetDuration":20,"studyType":81,"phases":82,"briefSummary":85,"conditions":86,"keywords":88,"overallStatus":97,"whyStopped":20,"lastUpdateSubmitDate":98,"lastUpdatePostDateStruct":99,"startDateStruct":102,"completionDateStruct":104,"leadSponsor":106,"locationsCount":107},{"fullName":5,"class":6},"Assistance Publique - Hôpitaux de Paris","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"FOXP3-T4 drug product","EXPERIMENTAL","FOXP3-T4 is a genetically modified cell therapy product consisting of autologous CD4+ T-cells transduced ex vivo with a self-inactivating bidirectional lentiviral vector (LV-EF1a-FOXP3-LNGFR) expressing the FOXP3 and LNGFR cDNAs.",[13,14],"Genetic: FOXP3-T4 drug product","Drug: ILT-101",[16,21],{"type":17,"name":9,"description":18,"armGroupLabels":19,"otherNames":20},"GENETIC","Each patient will receive a single IV infusion of FOXP3-T4, autologous gene-modified CD4+ T-transduced ex vivo with a self-inactivating lentiviral vector (LV-EF1a.FOXP3-LNGFR)",[9],null,{"type":22,"name":23,"description":24,"armGroupLabels":25,"otherNames":26},"DRUG","ILT-101","The first two patients included in the study will not receive IL-2 treatment. The others will receive the first injection the FOXP3-T4 treatment and if needed IL2-treatment. For IL2 treatment, patients will receive a daily dose for 5 days, followed by an administration per week for 3 months (ILT-101)",[9],[27],"Adeleuskin or IL2",[29],{"name":30,"affiliation":31,"role":32},"Emmanuelle SIX, MD, PhD","Institut Imagine","STUDY_CHAIR",[34,40],{"name":35,"role":36,"phone":37,"phoneExt":38,"email":39},"Marina CAVAZZANA, MD, PhD","CONTACT","01 44 49 50 68","+ 33","m.cavazzana@aphp.fr",{"name":41,"role":36,"phone":42,"phoneExt":43,"email":44},"Aline DECHANET, Project Manager","01 44 38 17 11","+33","aline.dechanet@aphp.fr",[46],{"facility":47,"status":20,"city":48,"state":49,"zip":50,"country":51,"countryCode":52,"cosmosGeoPoint":53,"geoPoint":58,"contacts":59},"Department of Biotherapy, Hopital Necker Enfants malades","Paris","Île-de-France Region","75015","France","FR",{"type":54,"coordinates":55},"Point",[56,57],2.3488,48.85341,{"lat":57,"lon":56},[60],{"name":61,"role":36,"phone":37,"phoneExt":43,"email":39},"Marina CAVAZZANA, MD, PHD",{"type":63,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR",[65],{"name":66,"class":6},"URC-CIC Paris Descartes Necker Cochin","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":79,"type":80},5,"ESTIMATED","INTERVENTIONAL",[83,84],"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.",[87],"The Immune Dysregulation Polyendocrinopathy Enteropathy X-linked Syndrome is a Primary Immunodeficiency Caused by Pathogenic Variants in Forkhead Box Protein 3",[89,90,91,92,93,94,95,96],"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":100,"type":101},"2026-07-13","ACTUAL",{"date":103,"type":80},"2026-09",{"date":105,"type":80},"2029-01",{"name":5,"class":6},1]