[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100457810":3},{"organization":4,"armGroups":7,"interventions":18,"overallOfficials":26,"centralContacts":30,"locations":37,"responsibleParty":61,"collaborators":64,"id":67,"slug":68,"hasResults":69,"nctId":70,"briefTitle":71,"officialTitle":72,"acronym":35,"eligibilityCriteria":73,"healthyVolunteers":69,"sex":74,"minAge":75,"maxAge":76,"enrollmentInfo":77,"targetDuration":35,"studyType":80,"phases":81,"briefSummary":83,"conditions":84,"keywords":86,"overallStatus":40,"whyStopped":35,"lastUpdateSubmitDate":97,"lastUpdatePostDateStruct":98,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":105,"locationsCount":107},{"fullName":5,"class":6},"Stanford University","OTHER",[8,14],{"label":9,"type":10,"description":11,"interventionNames":12},"Cohort A (≥12 years)","EXPERIMENTAL","The first participant in Dose Level 1 will be administered 1.0 x 10\\^6 CD4\\^LVFOXP3 \u002Fkg (± 20%).\n\nIf there is no toxicity observed in the first participant, the following participants in Dose Level 1 will be administered the same dose of 1.0 x 10\\^6 CD4\\^LVFOXP3 \u002Fkg (± 20%).\n\nIf there is no toxicity observed in any participants in Dose Level 1, participants will be enrolled into Dose Level 2 and administered 3 x 10\\^6 CD4\\^LVFOXP3 \u002Fkg (± 20%).\n\nIf there is no toxicity observed in any participants in Dose Level 2, participants will be enrolled into Dose Level 3 and administered 10 x 10\\^6 CD4\\^LVFOXP3 \u002Fkg (± 20%).\n\nIf in any dose level 1 of 2 participants show toxicity, that dose level will be expanded to 6 participants.",[13],"Biological: CD4^LVFOXP3",{"label":15,"type":10,"description":16,"interventionNames":17},"Cohort B (\u003C12 years)","Participants in Cohort B will always follow treatment of participants in Cohort A for the same dose level.\n\nCohort B will start at Dose Level 2 and be administered 3 x 10\\^6 CD4\\^LVFOXP3 \u002Fkg (± 20%).\n\nIf there is no toxicity observed in any participants in Dose Level 2, participants will be enrolled into Dose Level 3 and administered 10 x 10\\^6 CD4\\^LVFOXP3 \u002Fkg (± 20%).\n\nIf in any dose level 1 of 2 participants show toxicity, that dose level will be expanded to 6 participants.",[13],[19],{"type":20,"name":21,"description":22,"armGroupLabels":23,"otherNames":24},"BIOLOGICAL","CD4^LVFOXP3","Infusion of autologous CD4+ T cells that have undergone lentiviral-mediated gene transfer of:\n\ni) healthy human FOXP3 gene leading to persistent high FOXP3 expression and acquisition of Treg-like cell function; and ii) human CD271 surface marker gene that allows tracking and quantification of the CD4\\^LVFOXP3 in the blood.",[9,15],[25],"CD4^LVFOXP3 Treg-like cells",[27],{"name":28,"affiliation":5,"role":29},"Jessie Alexander, MD","PRINCIPAL_INVESTIGATOR",[31],{"name":32,"role":33,"phone":34,"phoneExt":35,"email":36},"Rosa Bacchetta, MD","CONTACT","650-498-8369",null,"rosab@stanford.edu",[38],{"facility":39,"status":40,"city":41,"state":42,"zip":43,"country":44,"countryCode":45,"cosmosGeoPoint":46,"geoPoint":51,"contacts":52},"Lucile Packard Children's Hospital","RECRUITING","Palo Alto","California","94305","United States","US",{"type":47,"coordinates":48},"Point",[49,50],-122.14302,37.44188,{"lat":50,"lon":49},[53,56,59],{"name":54,"role":33,"phone":35,"phoneExt":35,"email":55},"SCGT Clinical Trials Program","DL-SCTIntakeCoordinators@stanfordchildrens.org",{"name":57,"role":58,"phone":35,"phoneExt":35,"email":35},"Rajni Agarwal, MD","SUB_INVESTIGATOR",{"name":60,"role":29,"phone":35,"phoneExt":35,"email":35},"Jessie Alexander",{"type":29,"investigatorFullName":62,"investigatorTitle":63,"investigatorAffiliation":5,"oldNameTitle":35,"oldOrganization":35},"Jessie L. Alexander","Professor of Pediatrics",[65],{"name":66,"class":6},"California Institute for Regenerative Medicine (CIRM)","100457810","phase-1-cd4lvfoxp3-in-participants-with-ipex-100457810",false,"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":78,"type":79},30,"ESTIMATED","INTERVENTIONAL",[82],"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.",[85],"IPEX",[87,88,89,90,91,92,93,94,95,96],"Treg cells","Regulatory T cells","Gene therapy","autoimmunity","Immune Dysregulation Polyendocrinopathy Enteropathy X-linked","FOXP3","Lentiviral","CD271","NGFR","immune suppression","2025-05-19",{"date":99,"type":100},"2025-05-22","ACTUAL",{"date":102,"type":100},"2022-03-22",{"date":104,"type":79},"2037-02",{"name":106,"class":6},"Bacchetta, Rosa, MD",1]