[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100489256":3},{"organization":4,"armGroups":7,"interventions":18,"overallOfficials":25,"centralContacts":29,"locations":39,"responsibleParty":62,"collaborators":66,"id":78,"slug":79,"hasResults":80,"nctId":81,"briefTitle":82,"officialTitle":83,"acronym":84,"eligibilityCriteria":85,"healthyVolunteers":80,"sex":86,"minAge":87,"maxAge":24,"enrollmentInfo":88,"targetDuration":24,"studyType":91,"phases":92,"briefSummary":94,"conditions":95,"keywords":24,"overallStatus":41,"whyStopped":24,"lastUpdateSubmitDate":101,"lastUpdatePostDateStruct":102,"startDateStruct":105,"completionDateStruct":107,"leadSponsor":109,"locationsCount":110},{"fullName":5,"class":6},"Children's Hospital of Philadelphia","OTHER",[8,14],{"label":9,"type":10,"description":11,"interventionNames":12},"Dose Escalation Arm","EXPERIMENTAL","The dose escalation arm will determine the maximum tolerated dose of GPC2 CAR T cells using a standard 3+3 trial design.",[13],"Biological: GPC2 CAR T cells",{"label":15,"type":10,"description":16,"interventionNames":17},"Dose Expansion Arm","If at least one dose from the dose expansion arm is determined to be safe, additional patients will be enrolled to the dose expansion arm to preliminarily evaluate the rate of response to GPC2 CAR T cells and further characterize the safety profile of GPC2 CAR T cells.",[13],[19],{"type":20,"name":21,"description":22,"armGroupLabels":23,"otherNames":24},"BIOLOGICAL","GPC2 CAR T cells","The GPC2 CAR T investigational product is comprised of autologous human T cells that have been genetically modified to express a GPC2-targeting chimeric antigen receptor (CAR) transgene.",[9,15],null,[26],{"name":27,"affiliation":5,"role":28},"Lisa Wray, MD","PRINCIPAL_INVESTIGATOR",[30,35],{"name":31,"role":32,"phone":33,"phoneExt":24,"email":34},"CART Nurse Navigator","CONTACT","445-942-5891","CARTNurseNavigator@chop.edu",{"name":36,"role":32,"phone":37,"phoneExt":24,"email":38},"Melissa Varghese, M.S.","8455535358","varghesem@chop.edu",[40],{"facility":5,"status":41,"city":42,"state":43,"zip":44,"country":45,"countryCode":46,"cosmosGeoPoint":47,"geoPoint":52,"contacts":53},"RECRUITING","Philadelphia","Pennsylvania","19104","United States","US",{"type":48,"coordinates":49},"Point",[50,51],-75.16362,39.95238,{"lat":51,"lon":50},[54,56,58,59],{"name":31,"role":32,"phone":33,"phoneExt":24,"email":55},"cartnursenavigator@chop.edu",{"name":36,"role":32,"phone":57,"phoneExt":24,"email":38},"845-553-5358",{"name":27,"role":28,"phone":24,"phoneExt":24,"email":24},{"name":60,"role":61,"phone":24,"phoneExt":24,"email":24},"Yael Mosse, MD","SUB_INVESTIGATOR",{"type":63,"investigatorFullName":64,"investigatorTitle":65,"investigatorAffiliation":5,"oldNameTitle":24,"oldOrganization":24},"SPONSOR_INVESTIGATOR","Stephan Grupp MD PhD","Chief, Cell Therapy and Transplant Section Director, Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy Medical Director, Cell and Gene Therapy Lab",[67,68,71,73,76],{"name":5,"class":6},{"name":69,"class":70},"Gilead Sciences","INDUSTRY",{"name":72,"class":6},"University of Pennsylvania",{"name":74,"class":75},"National Cancer Institute (NCI)","NIH",{"name":77,"class":70},"Kite, A Gilead Company","100489256","phase-1-gpc2-car-t-cells-for-relapsed-or-refractory-neuroblastoma-and-metastatic-retinoblastoma-100489256",false,"NCT05650749","GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma","Phase 1 Trial of GPC2-Directed Chimeric Antigen Receptor Autologous T Cells (GPC2 CAR T) for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma","GPC2","Neuroblastoma Inclusion Criteria:\n\n1. Patients must be ≥ 1 year of age\n2. Patients must have high-risk neuroblastoma according to COG risk classification at the time of study enrollment. Patients who were initially considered low- or intermediate-risk, but then reclassified as high-risk are also eligible.\n3. Patients must have a previously histologically confirmed diagnosis of neuroblastoma:\n\n   1. That is recurrent\u002Frelapsed or refractory\u002Fpersistent according to INRC AND\n   2. For which standard curative measures do not exist or are no longer effective.\n   3. patients at first relapse are eligible as no known curative therapies exist for relapsed high-risk neuroblastoma.\n4. Patients must have evaluable or measurable disease at enrollment.\n5. In addition, patient must have experienced at least one of the following:\n\n   a. New disease site documented on at least one of the following: i. 123I-meta-iodobenzylguanidine (MIBG) or 18F-mFBG (meta-fluorobenzylguanidine) scan; OR ii. CT\u002FMRI; OR iii. FDG or Ga-68 Dotatate PET (in patients known to have MIBG non-avid tumor) and MRI findings consistent with tumor (i.e., bone lesions), OR iv. Biopsy confirmed neuroblastoma for any new or progressing lesion. b. Greater than 20% increase in a least one dimension of soft tissue mass documented by CT\u002FMRI and a minimum absolute increase of 5 mm in longest dimension in existing lesion(s). Previously irradiated lesions may be included.\n\n   c. Bone marrow biopsy shows progressive disease according to the revised INRC d. Stable persistent disease, such that response at the completion of upfront therapy or salvage therapy is less than partial response AND has a biopsy of at least one site showing viable neuroblastoma.\n\n   e. Responding persistent disease, defined as at least a partial response to frontline therapy (i.e., at least a partial response to frontline therapy but still has residual disease by MIBG scan, CT\u002FMRI, or bone marrow aspirations\u002Fbiopsies). Patients in this category are required to have histologic confirmation of viable neuroblastoma from at least one residual site (tumor seen on routine bone marrow morphology is sufficient).\n6. Patients must have a Lansky (≤ 16 years) or Karnofsky (\\> 16 years) score of ≥ 60\n7. Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age.\n8. Total bilirubin ≤ 1.5 x ULN (exception: total bilirubin ≤ 3 ULN for patients with Gilbert's Disease)\n9. Aspartate aminotransferase (AST) ≤ 2.5 ULN (exception: AST ≤ 5 x ULN for patients with liver metastases).\n10. Alanine aminotransferase (ALT) ≤ 2.5 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases).\n11. Patients must have a baseline pulse oximetry of at least 92% on room air. In addition, a DLCO ≥ 60% (corrected for anemia) is required if PFTs are clinically appropriate as determined by the treating investigator.\n12. Left ventricular shortening fraction (LVSF) ≥28% or ejection fraction (LVEF) ≥ 50% confirmed by Echo, or adequate ventricular function documented by a scan or a cardiologist.\n\nNeuroblastoma Exclusion Criteria:\n\n1. Patients with active hepatitis B or active hepatitis C.\n2. Patients with HIV infection.\n3. Patients with uncontrolled active infection.\n4. Patients with primary or acquired immunodeficiency disorder.\n5. Patients with a known hypersensitivity to DMSO.\n6. Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.\n7. Patients with actively progressing CNS metastases, including parenchymal or leptomeningeal involvement. (Note: CNS imaging at screening is only required if the there is a clinical indication of suspected CNS metastasis)\n8. Active medical disorder that, in the opinion of the investigator, would substantially increase the risk of uncontrollable CRS and\u002For neurotoxicity.\n9. Patients with congestive heart failure (as defined by New York Heart Association Functional Classification III or IV), unstable angina, serious uncontrolled cardiac arrhythmia, a myocardial infarction within 6 months prior to study entry or a history of myocarditis.\n10. Patients who have received any live vaccines within 30 days prior to enrollment.\n11. Patients who are pregnant or nursing (lactating).\n12. Patients who have a life expectancy \\\u003C 6 months at time of consent.\n\nRetinoblastoma Inclusion Criteria:\n\n1. Patient age ≥ 6 months.\n2. Patients must have metastatic retinoblastoma according to International\n\n   Retinoblastoma Staging System (IRSS) risk classification (4) at the time of study enrollment:\n\n   a. Retinoblastoma Cohort 1 (Extra-CNS metastasis) i. Stage IVa disease ii. Extra-CNS disease must be confirmed as retinoblastoma by histology (either at diagnosis or recurrence) iii. Measurable disease: Defined as \\> 1cm2 or biopsy-proven bone marrow disease iv. Prior treatment: Recurrent or refractory disease following treatment with COG ARET0321-like or equivalent regimen as part of upfront or recurrent therapy b. Retinoblastoma Cohort 2 (CNS disease) i. Stage IVb disease ii. Histologic confirmation is not required iii. CNS disease defined as measurable disease \\>1cm2, non-measurable, or CSF positivity alone c. Prior treatment: i. Stage IVb.1 and IVb.2: Recurrent after treatment with COG ARET0321-like or equivalent regimen as part of upfront or recurrent therapy i. Stage IVb.3: Prior treatment is not required (i.e., eligible at initial diagnosis or recurrence)\n3. Patients must have a Lansky (≤ 16 years) or Karnofsky (\\> 16 years) score of ≥ 60\n4. Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN .\n5. Total bilirubin ≤ 1.5 x ULN (exception: total bilirubin ≤ 3 ULN for patients with Gilbert's Disease)\n6. Aspartate aminotransferase (AST) ≤ 2.5 ULN (exception: AST ≤ 5 x ULN for patients with liver metastases).\n7. Alanine aminotransferase (ALT) ≤ 2.5 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases).\n8. Patients must have a baseline pulse oximetry of at least 92% on room air. In addition, a DLCO ≥ 60% (corrected for anemia) is required if PFTs are clinically appropriate as determined by the treating investigator.\n9. Left ventricular shortening fraction (LVSF) ≥28% or ejection fraction (LVEF) ≥ 50% confirmed by Echo, or adequate ventricular function documented by a scan or a cardiologist.\n\nRetinoblastoma Exclusion Criteria:\n\n1. Patients with active hepatitis B or active hepatitis C.\n2. Patients with HIV infection.\n3. Patients with uncontrolled active infection.\n4. Patients with primary or acquired immunodeficiency disorder.\n5. Patients with a known hypersensitivity to DMSO.\n6. Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.\n7. Active medical disorder that, in the opinion of the investigator, would substantially increase the risk to the subject.\n8. Patients with congestive heart failure (as defined by New York Heart Association Functional Classification III or IV), unstable angina, serious uncontrolled cardiac arrhythmia, a myocardial infarction within 6 months prior to study entry or a history of myocarditis.\n9. Patients who have received any live vaccines within 30 days prior to enrollment.\n10. Patients who are pregnant or nursing (lactating).\n11. Patients who have a life expectancy \\\u003C 6 months at time of consent.\n12. Retinoblastoma Cohort 1 (Extra-CNS disease):\n\n    1. Concurrent CNS disease (they may be eligible for Retinoblastoma Cohort 2)\n    2. Stage III disease (orbital or lymph node regional extension without other hematogenous metastases).\n13. Retinoblastoma Cohort 2 (CNS disease):\n\n    1. \"Bulky\" disease (\\>5 cm in diameter) within or compressing the brainstem or thalamus. Note: Tumors touching the brainstem\u002Fthalamus without evidence of compression and\u002For tumors in other CNS locations do not have a maximal size criterion.\n    2. If evidence of clinically significant increased intracranial pressure at time of relapse, patient must demonstrate improvement by time of enrollment.","ALL","1 Year",{"count":89,"type":90},45,"ESTIMATED","INTERVENTIONAL",[93],"PHASE1","This is a first in human dose escalation trial to determine the safety of administering GPC2 CAR T cells in patients with advanced neuroblastoma or retinoblastoma.",[96,97,98,99,100],"Refractory Neuroblastoma","Relapsed Neuroblastoma","High-risk Neuroblastoma","Retinoblastoma","Metastatic Retinoblastoma","2025-12-24",{"date":103,"type":104},"2025-12-29","ACTUAL",{"date":106,"type":104},"2023-05-23",{"date":108,"type":90},"2030-01-30",{"name":64,"class":6},1]