[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100441512":3},{"organization":4,"outcomesModule":7,"designInfo":49,"detailedDescription":48,"studyPopulation":48,"armGroups":56,"interventions":63,"overallOfficials":71,"centralContacts":75,"locations":84,"responsibleParty":104,"collaborators":48,"id":108,"slug":48,"hasResults":109,"nctId":110,"briefTitle":111,"officialTitle":112,"acronym":48,"eligibilityCriteria":113,"healthyVolunteers":109,"sex":114,"minAge":115,"maxAge":116,"enrollmentInfo":117,"targetDuration":48,"studyType":120,"phases":121,"briefSummary":123,"conditions":124,"keywords":48,"overallStatus":86,"whyStopped":48,"lastUpdateSubmitDate":127,"lastUpdatePostDateStruct":128,"startDateStruct":131,"completionDateStruct":133,"leadSponsor":135,"locationsCount":136},{"fullName":5,"class":6},"Children's Hospital of Philadelphia","OTHER",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":48},[9],{"measure":10,"description":11,"timeFrame":12},"Two-year progression free survival","Survival without evidence of relapse or disease progression","2 years",[14,18,20,23,25,27,29,33,36,40,44],{"measure":15,"description":16,"timeFrame":17},"Disease-specific response\u002Fprogression endpoints: SSc cohort","o Pulmonary function: Change in forced vital capacity (FVC), total lung capacity (TLC) or diffusing capacity of the lung for carbon monoxide (DLCO) \\> 10%","24 months following transplant",{"measure":15,"description":19,"timeFrame":17},"o Skin condition: An improvement is indicated by a decrease on modified Rodan Skin Score (mRSS) of \\> 5 points",{"measure":21,"description":22,"timeFrame":17},"Disease-specific response\u002Fprogression endpoints: Systemic Lupus Erythematosus (SLE) cohort","o Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) \\\u003C 4",{"measure":21,"description":24,"timeFrame":17},"o Complete remission off therapy (BILAG D\u002FE only or SLEDAI=0 and no SLE treatment except hydroxychloroquine)",{"measure":21,"description":26,"timeFrame":17},"o Serologic response: presence of positive ANA, anti-dsDNA and anticardiolpin antibody titers",{"measure":21,"description":28,"timeFrame":17},"o Serologic response: abnormal complement C3 and C4 levels",{"measure":30,"description":31,"timeFrame":32},"Overall survival (OS)","Overall survival will be considered as time from transplant to death from any cause","2 and 5 years following transplant",{"measure":34,"description":35,"timeFrame":32},"Event free survival (EFS)","Events include death, and significant persistent organ damage\n\no An event based on organ dysfunction must be documented on at least two occasions, at least three months apart and include: respiratory failure (resting O2 saturation \\\u003C 88%), renal failure (chronic dialysis) and cardiomyopathy (clinical congestive heart failure New York Class III or IV, left ventricular ejection fraction (LVEF) \\\u003C 30% by echocardiogram despite therapy)",{"measure":37,"description":38,"timeFrame":39},"100 day treatment-related mortality","Defined as death from non-disease related causes in the 100 days from stem cell infusion","100 days from stem cell infusion",{"measure":41,"description":42,"timeFrame":43},"Time to engraftment","• Achieving an absolute neutrophil count (ANC) \\> 500 cells\u002FuL and an unsupported platelet count of \\> 20,000 cells\u002FuL for three consecutive days","3 days",{"measure":45,"description":46,"timeFrame":47},"Change in quality of life","* Quality of life will be measured based on the Patient-Reported Outcomes measurement Information System (PROMIS) that evaluates physical, mental and social health in adults and children.\n* patient reported outcome measurement information system (PROMIS) will be administered to each patient (or proxy) prior to autologous stem cell transplant (ASCT) and three times\u002Fyear for the first two years post-transplant and then annually until five years post-transplant.","prior to autologous stem cell transplant (ASCT) until 5 years post-transplant",null,{"allocation":50,"interventionModel":51,"interventionModelDescription":52,"primaryPurpose":53,"observationalModel":48,"timePerspective":48,"maskingInfo":54},"NA","SINGLE_GROUP","open label single arm pilot study","TREATMENT",{"masking":55,"maskingDescription":48,"whoMasked":48},"NONE",[57],{"label":58,"type":59,"description":60,"interventionNames":61},"CD3\u002FCD19 depleted ASCT","EXPERIMENTAL","The test article is autologous stem cell transplant with a CD3\u002FCD19-depleted stem cell product.",[62],"Biological: Depletion of CD3\u002FCD19 in an autologous stem cell transplant",[64],{"type":65,"name":66,"description":67,"armGroupLabels":68,"otherNames":69},"BIOLOGICAL","Depletion of CD3\u002FCD19 in an autologous stem cell transplant","The purpose of this study is to determine the safety and feasibility of CD3\u002FCD19 depleted autologous stem cell transplant for the treatment of life threatening autoimmune disease. We will perform CD3\u002FCD19 depletion using the CliniMACs device as a means of purging autoreactive T and B cells from the transfused autologous stem cell product, while retaining some immune function, namely natural killer cells and monocytes in the product.",[58],[70],"CD3\u002FCD19 depletion using cliniMACs device",[72],{"name":73,"affiliation":5,"role":74},"Caitlin Elgarten, MD","PRINCIPAL_INVESTIGATOR",[76,81],{"name":77,"role":78,"phone":79,"phoneExt":48,"email":80},"Patricia M Hankins","CONTACT","(215) 590-5168","HANKINSP@chop.edu",{"name":73,"role":78,"phone":82,"phoneExt":48,"email":83},"2158079038","elgartenc@chop.edu",[85],{"facility":5,"status":86,"city":87,"state":88,"zip":89,"country":90,"countryCode":91,"cosmosGeoPoint":92,"geoPoint":97,"contacts":98},"RECRUITING","Philadelphia","Pennsylvania","19104","United States","US",{"type":93,"coordinates":94},"Point",[95,96],-75.16362,39.95238,{"lat":96,"lon":95},[99,101,103],{"name":100,"role":78,"phone":79,"phoneExt":48,"email":80},"Study Coordinator",{"name":102,"role":78,"phone":82,"phoneExt":48,"email":83},"Principal investigator",{"name":73,"role":74,"phone":48,"phoneExt":48,"email":48},{"type":105,"investigatorFullName":106,"investigatorTitle":107,"investigatorAffiliation":5,"oldNameTitle":48,"oldOrganization":48},"SPONSOR_INVESTIGATOR","Stephan Grupp MD PhD","Director of Cancer Immunotherapy Program","100441512",false,"NCT05029336","Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases","Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases","Inclusion Criteria:\n\n1. Age 8 ≤ 25 years at time of enrollment.\n2. Severe systemic sclerosis or systemic lupus erythematosus based on specific criteria\n3. Adequate organ function status\n4. No active, untreated infections.\n\nExclusion Criteria:\n\n1. Previous hematopoietic stem cell transplant (HSCT) or solid organ transplant\n2. Pregnancy\n3. Ongoing participation in a clinical trial testing an investigational drug or ongoing receipt of disallowed disease modifying anti-rheumatic drugs (DMARD)\n4. Severe comorbidity that jeopardizes the ability of the subject to tolerate therapy","ALL","8 Years","25 Years",{"count":118,"type":119},20,"ESTIMATED","INTERVENTIONAL",[122],"PHASE2","A subset of autoimmune diseases (ADs) in children and young adults are life-threatening and unresponsive to conventional treatments. In these patients, the delivery of high dose immunosuppressive therapy followed by autologous stem cell transplant (ASCT) offers a treatment strategy capable of purging the pathogenic, autoreactive immune system and an opportunity for \"immune reset.\" This strategy has been used in adults across a myriad of indications with evidence for efficacy. This study proposes a pilot study to evaluate this therapeutic strategy in children and young adults with systemic sclerosis (SSc) and systemic lupus erythematosis (SLE), two potentially life threatening autoimmune diseases that may response to this therapeutic approach.",[125,126],"Systemic Lupus Erythematosus","Systemic Sclerosis","2025-10-09",{"date":129,"type":130},"2025-10-14","ACTUAL",{"date":132,"type":119},"2026-03",{"date":134,"type":119},"2031-05",{"name":106,"class":6},1]