[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100586087":3},{"organization":4,"armGroups":7,"interventions":19,"overallOfficials":18,"centralContacts":25,"locations":32,"responsibleParty":103,"collaborators":18,"id":105,"slug":18,"hasResults":106,"nctId":107,"briefTitle":108,"officialTitle":109,"acronym":110,"eligibilityCriteria":111,"healthyVolunteers":106,"sex":112,"minAge":113,"maxAge":114,"enrollmentInfo":115,"targetDuration":18,"studyType":118,"phases":119,"briefSummary":122,"conditions":123,"keywords":125,"overallStatus":35,"whyStopped":18,"lastUpdateSubmitDate":133,"lastUpdatePostDateStruct":134,"startDateStruct":137,"completionDateStruct":139,"leadSponsor":141,"locationsCount":142},{"fullName":5,"class":6},"Novartis","INDUSTRY",[8,14],{"label":9,"type":10,"description":11,"interventionNames":12},"Cohort 1 (DFT383)","EXPERIMENTAL","Treatment with DFT383",[13],"Genetic: DFT383",{"label":15,"type":16,"description":17,"interventionNames":18},"Cohort 0 (SoC)","NO_INTERVENTION","No study treatment, will continue with standard of care (cysteamine).",null,[20],{"type":21,"name":22,"description":23,"armGroupLabels":24,"otherNames":18},"GENETIC","DFT383","DFT383 is an autologous hematopoietic stem cell (HSC) gene therapy.",[9],[26,31],{"name":27,"role":28,"phone":29,"phoneExt":18,"email":30},"Novartis Pharmaceuticals","CONTACT","1-888-669-6682","novartis.email@novartis.com",{"name":27,"role":28,"phone":18,"phoneExt":18,"email":18},[33,55,71,87],{"facility":34,"status":35,"city":36,"state":37,"zip":38,"country":39,"cosmosGeoPoint":40,"geoPoint":45,"contacts":46},"University of California at San Diego - Rady Children's Hospital","RECRUITING","San Diego","California","92123","United States",{"type":41,"coordinates":42},"Point",[43,44],-117.16472,32.71571,{"lat":44,"lon":43},[47,52],{"name":48,"role":28,"phone":49,"phoneExt":50,"email":51},"Mieko Pretlow","858-966-1700","Ext 226010","mpretlow@rchsd.org",{"name":53,"role":54,"phone":18,"phoneExt":18,"email":18},"Nadine Benador nbenador@health.ucsd.edu","PRINCIPAL_INVESTIGATOR",{"facility":56,"status":35,"city":57,"state":37,"zip":58,"country":39,"cosmosGeoPoint":59,"geoPoint":63,"contacts":64},"Stanford University - Stanford Children's Health","Stanford","94305",{"type":41,"coordinates":60},[61,62],-122.16608,37.42411,{"lat":62,"lon":61},[65,69],{"name":66,"role":28,"phone":67,"phoneExt":18,"email":68},"Research Nurse","650-725-9032","scgt_clinical_trials_office@lists.stanford.edu",{"name":70,"role":54,"phone":18,"phoneExt":18,"email":18},"Alice Bertaina",{"facility":72,"status":35,"city":73,"state":74,"zip":75,"country":39,"cosmosGeoPoint":76,"geoPoint":80,"contacts":81},"Emory University School of Medicine - Children's Healthcare of Atlanta (recuiting Cohort 0)","Atlanta","Georgia","30322",{"type":41,"coordinates":77},[78,79],-84.38798,33.749,{"lat":79,"lon":78},[82,86],{"name":83,"role":28,"phone":84,"phoneExt":18,"email":85},"Laurence (Larry) Greenbaum","404-712-6374","lgreen6@emory.edu",{"name":83,"role":54,"phone":18,"phoneExt":18,"email":18},{"facility":88,"status":35,"city":89,"state":90,"zip":91,"country":39,"cosmosGeoPoint":92,"geoPoint":96,"contacts":97},"Baylor College of Medicine - Texas Children's Hospital (recuiting Cohort 0)","Houston","Texas","77030",{"type":41,"coordinates":93},[94,95],-95.36327,29.76328,{"lat":95,"lon":94},[98,102],{"name":99,"role":28,"phone":100,"phoneExt":18,"email":101},"Ewa Elenberg","832-824-3800","Email_elenberg@bcm.edu",{"name":99,"role":54,"phone":18,"phoneExt":18,"email":18},{"type":104,"investigatorFullName":18,"investigatorTitle":18,"investigatorAffiliation":18,"oldNameTitle":18,"oldOrganization":18},"SPONSOR","100586087",false,"NCT06910813","DFT383 in Pediatric Participants With Nephropathic Cystinosis","An Open-label, Multi-center, Phase I\u002FII Study to Assess Safety, Tolerability and Efficacy of DFT383 in Pediatric Participants With Nephropathic Cystinosis, Followed by a Long-term Extension Phase","CYStem","Key Inclusion Criteria:\n\nParticipants eligible for inclusion in this study must meet all the following criteria:\n\n1. Informed consent in writing from parent(s) or legal guardian(s) must be provided\n2. 2 to 5 years of age (including 5 years and 364 days old) at Screening\n3. Weight-for-stature is ≥ the third percentile, and is ≥ 10 kg\n4. Oral cysteamine therapy for at least 6 months\n5. Historic clinical diagnosis of nephropathic cystinosis\n6. Laboratory evidence of of renal fanconi syndrome (RFS)\n7. Relatively preserved kidney function (eGFR ≥ 60mL\u002Fmin\u002F1.73m2)\n8. Received all age-appropriate vaccinations\n\nKey exclusion Criteria for Cohort 1 and 0\n\n1. A history of kidney transplantation\n2. A prior or planned bone marrow or stem cell transplantation or prior treatment with gene therapy\n3. History of malignancy\n4. A severe or uncontrolled medical disorder\n5. Major surgery within 90 days\n\nAdditional Key exclusion criteria for Cohort 1 - The following exclusion criterion applies to Cohort 1 only as it is related to DFT383 treatment:\n\n1\\. Indomethacin within 2 weeks prior to Screening\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.","ALL","2 Years","5 Years",{"count":116,"type":117},30,"ESTIMATED","INTERVENTIONAL",[120,121],"PHASE1","PHASE2","An open-label, multi-center, phase I\u002FII study to assess the safety, tolerability and efficacy of DFT383 in pediatric participants with nephropathic cystinosis, followed by a long-term extension phase.\n\nThe purpose of this clinical study is to assess safety, tolerability, and efficacy of DFT383 in participants aged 2 to 5 years with nephropathic cystinosis. The study consists of a Core Phase and a long-term Extension Phase. DFT383 is a cellular gene therapy.\n\nThis study includes an active arm (Cohort 1) of participants treated with study treatment DFT383 and a concurrent reference arm (Cohort 0). Participants in Cohort 0 will not receive study treatment and will only participate in the Core Phase of the study. The study is not randomized and Cohort 0 aims to collect prospective and concurrent data in this rare disease.",[124],"Nephropathic Cystinosis",[126,127,128,129,22,130,131,132],"Cystinosis","Nephropathic cystinosis","Lysosomal storage disorder","CTNS gene","Cellular gene therapy","Cysteamine","Renal Fanconi syndrome","2026-05-28",{"date":135,"type":136},"2026-05-29","ACTUAL",{"date":138,"type":136},"2025-06-02",{"date":140,"type":117},"2044-03-14",{"name":27,"class":6},4]