[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"high-risk-neuroblastoma\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:high-risk-neuroblastoma":57},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,12,0,[8,43,69,98,167,197,232,257,283,307,330,353],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":27,"conditions":28,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100635179","phase-2-a-safety-and-efficacy-study-of-hu14-in-high-risk-neuroblastoma-patients-100635179",false,"NCT07549321","A Safety and Efficacy Study of hu14 in High-Risk Neuroblastoma Patients","A Phase 2\u002F3 Study to Characterize and Evaluate the Efficacy, Safety, and Tolerability of hu14.18K322A Treatment Given in Combination With Chemotherapy in Participants With High-Risk Neuroblastoma","SHINE","Inclusion Criteria\n\n1. Are ≥18 months to \\\u003C18 years of age at time of informed consent or assent.\n2. Are initially diagnosed with histologically proven HRNB with metastatic disease.\n3. Have evaluable or measurable disease per International Neuroblastoma Response Criteria (INRC)\n4. Have a Lansky performance status of ≥50 (≤16 years ) or Karnofsky performance status ≥50% (for \\>16 years).\n5. Have recovered from the toxic effects of prior chemotherapies\n6. Are at least 2 weeks beyond any major tumor surgery\n7. Meet the following organ function criteria, as measured within 1 week prior to Investigational Medicinal Product (IMP) dosing:\n\n   1. BM function: i. Platelets ≥50 × 109\u002FL ii. Absolute neutrophil count (ANC) ≥0.50 × 109\u002FL\n   2. Renal function: i. Age-adjusted serum creatinine ≤1.5 × upper limit of normal (ULN) for age. ii. Estimated glomerular filtration rate (eGFR) at least 60 mL\u002Fmin using the Schwartz formula.\n   3. Liver function: Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≤3 × ULN and total bilirubin ≤1.5 × ULN.\n   4. Cardiac function: i. Shortening fraction ≥27% or ejection fraction of ≥50% on echocardiogram. ii. Corrected QT Interval on electrocardiogram (ECG) using the Fridericia formula (QTcF) ≤ 480 msec.\n   5. Lung function: i. Pulse oximetry considered normal in room air. No evidence of dyspnea at rest.\n   6. Central nervous system (CNS) function: i. Participants with a history of CNS disease must have no clinical or radiological evidence of active CNS disease at the time of study enrollment.\n8. If a fertile and sexually active woman of child-bearing potential (WOCBP), have a negative serum pregnancy test at Screening and then either a negative serum or urine test prior to each cycle and agree to use an acceptable and highly effective contraception method during the study and for at least 6 months after the last day of study treatment .\n9. If a fertile and sexually active male, agree to use condoms during the study and for at least 6 months after the last dose of study treatment\n10. If applicable based on age, are willing and able to provide voluntary written informed assent for participation in the study (as per local Institutional Review Board \\[IRB\\]\u002FIndependent Ethics Committee \\[IEC\\] requirements and if applicable based on regional age of consent) and to comply with all protocol requirements.\n11. Their parent or legal guardian (if applicable based on regional age of consent) is willing and able to provide voluntary written informed consent for the participant's involvement in the study.\n\nExclusion Criteria\n\n1. Any active uncontrolled infection at the time of enrollment. Any known history of infection with human immunodeficiency virus (HIV), or active or chronic infection with hepatitis B virus (HBV) or hepatitis C virus (HCV).\n2. Any contraindications to any of the study treatments.\n3. Patients with \\>Grade 2 diarrhea.\n4. Patients with disease of any major organ system that would compromise their ability to withstand chemoimmunotherapy.\n5. Patients who have undergone a prior allogeneic stem cell transplant \\\u003C 6 months ago or have undergone a solid organ transplant.\n6. Patients who are on hemodialysis.\n7. Patients who require or are likely to require pharmacologic doses of systemic corticosteroids while receiving treatment on this study except to manage allergic reactions\n8. Patients on any other immunosuppressive medications\n9. Patients who have received enzyme-inducing anticonvulsants including phenytoin, phenobarbital, or carbamazepine for at least 7 days prior to study enrollment.\n10. Patients who have been diagnosed with any malignancy other than neuroblastoma.\n11. Patients with symptoms of congestive heart failure.\n12. Patients with a history of Grade 4 allergic reactions to anti-GD2 antibodies or reactions that required permanent discontinuation of the anti-GD2 therapy.\n13. Patients participating in or planning to participate in another study that is either blinded or involves an IMP\n14. If female, are breastfeeding, pregnant, or planning to become pregnant, or, if sexually active and of child-bearing potential, are unwilling to use an effective birth control method until 6 months after the last dose of study medication\n15. Do not meet the following required washout periods prior to the administration of the first dose of hu14.18K322A:\n\n    1. 14 days from prior systemic myelosuppressive chemotherapy.\n    2. 7 days from prior systemic biologic antineoplastic agents (e.g. anti-cancer agents not known to be myelosuppressive - not associated with reduced platelet or ANC counts).\n    3. 14 days from systemic steroids.\n    4. ≥12 weeks from large field radiation therapy (i.e., total body irradiation, whole abdominal, total lung, ≥50% pelvis).\n    5. 6 weeks from prior craniospinal radiotherapy or Iodine-131-metaiodobenzylguanidine (131I-MIBG) therapy.\n    6. 2 weeks from radiotherapy to the primary tumor bed.\n    7. ≥7 days from small treatment field radiation.\n    8. 14 days or 5 half-lives (whichever is longer) from last administration of an IMP.\n    9. \\>7 days prior to study enrollment for drugs that are strong inducers or inhibitors of Cytochrome P450 3A4 (CYP3A4).\n    10. ≥21 days and with recovery of all associated toxicities from receiving cellular therapy (e.g., modified T lymphocyte cells, Natural Killer (NK) cells, dendritic cells).\n16. Ongoing need for any medication known or suspected to interfere with study treatment.","ALL","18 Months","18 Years",{"count":21,"type":22},144,"ESTIMATED","INTERVENTIONAL",[25,26],"PHASE2","PHASE3","Neuroblastoma is the most common type of solid cancer found outside the brain in young children. Generally, it affects children younger than 5 years old, with the average age when it is found being just 2 years. Most patients have 'high-risk' disease, with spread of the disease to different sites (metastases). This multinational study aims to find out how effective and safe the treatment of a monoclonal anti-GD2 antibody hu14.18K322A (daretabart) is when used together with chemotherapy to treat children and young people who have high-risk neuroblastoma.",[29],"High-Risk Neuroblastoma","RECRUITING","2026-06-26",{"date":33,"type":34},"2026-06-30","ACTUAL",{"date":36,"type":34},"2026-03-30",{"date":38,"type":22},"2031-03",{"name":40,"class":41},"Renaissance Pharma Ltd.","INDUSTRY",5,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":50,"targetDuration":4,"studyType":23,"phases":52,"briefSummary":53,"conditions":54,"keywords":4,"overallStatus":58,"whyStopped":4,"lastUpdateSubmitDate":59,"lastUpdatePostDateStruct":60,"startDateStruct":62,"completionDateStruct":64,"leadSponsor":66,"locationsCount":4},"100582068","phase-2-comparing-123i-mibg-and-18f-mfbg-imaging-in-patients-with-newly-diagnosed-high-risk-neuroblastoma-100582068","NCT06858501","Comparing 123I-MIBG and 18F-MFBG Imaging in Patients With Newly Diagnosed, High Risk Neuroblastoma","Concordance in Response Assessment Incorporating Meta-Iodobenzylguanidine (123I-MIBG) and Meta-[18F]Fluorobenzylguanidine (18F-MFBG, IND#146379, NSC#853868) Imaging in Neuroblastoma","Inclusion Criteria:\n\n* Any age at diagnosis.\n* Patients must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular, unfavorable subtype) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites.\n* Patients must have high-risk neuroblastoma defined as one of the following:\n\n  * Any age with International Neuroblastoma Risk Group (INRG) stage L2 or M and MYCN amplification.\n  * Age ≥ 547 days and INRG stage M regardless of biologic features.\n  * Age ≥ 547 days and INRG stage L2 with unfavorable histology.\n* Patients must have newly diagnosed disease.\n* Patients must have either measurable or evaluable disease by INRC.\n* Patients observed or treated with a single cycle of chemotherapy per a low- or intermediate-risk neuroblastoma regimen (e.g. as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high-risk disease, but subsequently found to meet high-risk criteria will be eligible. These patients must enroll prior to the start of high-risk therapy.\n* Patients who receive localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis will be eligible.\n* Patients initially recognized to have high-risk disease must enroll prior to or within the first week after starting high-risk induction chemotherapy.\n* Induction therapy as per a standard high-risk neuroblastoma induction regimen (examples include ANBL1531 arm A, ANBL2131 arm A, or ANBL2131 arm B) must be planned for patients to be eligible for this study.\n\nExclusion Criteria:\n\n* Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events of radiation. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use two effective methods of birth control, including a medically accepted barrier or contraceptive method (e.g., male or female condom) at least 48 hours prior to and following all imaging studies. Abstinence is an acceptable method of birth control.\n* Norepinephrine transporter (NET)-dependent agents: Many medications are known to interfere with uptake of NET-dependent agents. Investigators should use caution when prescribing these medications for patients undergoing procedures on this study. Medications that are known to substantially interfere with uptake of NET-dependent agents should be held if possible, based on patient condition 24 hours prior to each 18F-MFBG scan. These agents can be resumed immediately after each 18F-MFBG scan is completed. Patients who are receiving medications that are known to significantly interfere with uptake of NET-dependent agents (primarily tricyclic antidepressants, psychostimulants, and antihypertensives) and for whom these medications cannot be safely withheld before the start of study procedures will not be eligible.\n* The patient has a known or suspected history of significant allergic reaction or anaphylaxis to any components of the 18F-MFBG or 123I-MIBG imaging agents.\n* Patients who will require sedation or anesthesia only for 18F-MFBG imaging.\n\n  * Note: Patients who need anesthesia will be required to have 18F-MFBG imaging combined with other scans or procedures necessary for clinical care (ex: MRI, bone marrow aspirate\u002Fbiopsies, line placement).\n* Patients will be able to enroll prior to baseline 123I-MIBG imaging. However, patients who have had their baseline standard of care 123I-MIBG imaging prior to enrollment who have known MIBG non-avid disease are not eligible.\n* Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.",{"count":51,"type":22},84,[25],"This phase II trial evaluates whether an investigational scan (18F-MFBG positron emission tomography \\[PET\\]\u002Fcomputed tomography \\[CT\\] or PET\u002Fmagnetic resonance imaging \\[MRI\\]) can accurately detect tumors in patients with newly diagnosed, high-risk neuroblastoma as well as standard of care imaging with 123 I-MIBG. 18F-MFBG is a radioactive diagnostic agent that is injected into a vein and taken up by tumor cells. The cells can then be visualized using PET\u002FCT or PET\u002FMRI scans. A PET scan uses radioactive material injected into the blood to show the internal workings of the body. A CT scan uses x-rays and a computer to produce a 3-dimensional image of the body. MRI uses radiofrequency waves and a strong magnetic field rather than x-rays to provide clear and detailed pictures of internal organs and tissues. Combining PET with CT or MRI may help doctors better understand the extent and the exact location of disease. Diagnostic procedures, such as 18F-MFBG PET\u002FCT or PET\u002FMRI, may detect tumors as well as or better than the current standard imaging with 123 I-MIBG in patients with newly diagnosed, high-risk neuroblastoma.",[55,56,57],"Ganglioneuroblastoma","Ganglioneuroblastoma, Nodular","High Risk Neuroblastoma","NOT_YET_RECRUITING","2026-06-25",{"date":61,"type":34},"2026-06-29",{"date":63,"type":22},"2026-09-22",{"date":65,"type":22},"2028-12-20",{"name":67,"class":68},"Children's Oncology Group","NETWORK",{"id":70,"slug":71,"hasResults":11,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":4,"eligibilityCriteria":75,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":76,"targetDuration":4,"studyType":23,"phases":78,"briefSummary":79,"conditions":80,"keywords":84,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":89,"startDateStruct":91,"completionDateStruct":93,"leadSponsor":95,"locationsCount":97},"100520544","phase-2-a-study-of-a-vaccine-in-combination-with-beta-glucan-in-people-with-neuroblastoma-100520544","NCT06057948","A Study of a Vaccine in Combination With Beta-glucan in People With Neuroblastoma","Phase II Trial of a Bivalent Vaccine With the Immunological Adjuvant OPT-821 (QS-21), in Combination With Randomization of Oral β-glucan, for High-Risk Neuroblastoma","Inclusion Criteria:\n\n* Diagnosis of NB as defined by international criteria,102 i.e., histopathology (confirmed by the MSK Department of Pathology) or BM metastases plus high urine catecholamine levels, or positivity in MIBG scan.\n* HR-NB as defined by risk-related treatment guidelines and international criteria,102 i.e., metastatic\u002Fnon-localized disease with MYCN amplification (any age), metastatic disease \\>18 months old, MYCN-amplified localized disease (any age), or disease resistant to standard chemotherapy.\n* HR-NB (as defined above) and in 1) first CR at ≥ 6 months from initiation of immunotherapy using anti-GD2 antibody, or 2) second or subsequent CR (achieved after treatment for PD). CR is defined according to the International Neuroblastoma Response Criteria. Patients with positive MIBG scan but negative FDG-PET scan, and CR in BM, are eligible.\n* Patients with grade 3 toxicities or less using the Common Toxicity Criteria (Version 5.0) developed by the National Cancer Institute of the USA (CTCAE v5.0) related to hematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as determined by blood tests or physical exam. Plus:\n\n  * Absolute neutrophil count (ANC) ≥ 500\u002Fmcl\n  * Absolute lymphocyte count ≥ 500\u002Fmcl\n* \\>21 and \\\u003C180 days between completion of systemic therapy and 1st vaccination.\n* A negative pregnancy test is required for patients with child-bearing capability\n* Signed informed consent indicating awareness of the investigational nature of this program.\n\nExclusion Criteria:\n\n* Patients with grade 4 hematologic, cardiac, neurological, pulmonary, renal, hepatic or gastrointestinal function as determined by blood tests or physical exam, using the Common Toxicity Criteria (Version 5.0) developed by the National Cancer Institute of the USA.\n* History of allergy to KLH, QS-21, OPT-821, or glucan\n* Prior treatment with this vaccine.\n* Active life-threatening infection requiring systemic therapy.\n* Inability to comply with protocol requirements.",{"count":77,"type":22},94,[25],"The purpose of this study is to test which treatment schedule of β-glucan with bivalent vaccine is more effective for participants with high-risk neuroblastoma that is in complete remission.",[81,82,83],"Neuroblastoma","High-risk Neuroblastoma","Metastatic Neuroblastoma",[81,82,83,85,86,87],"HR-NB","Memorial Sloan Kettering Cancer Center","23-198","2026-06-04",{"date":90,"type":34},"2026-06-05",{"date":92,"type":34},"2023-09-21",{"date":94,"type":22},"2027-03-21",{"name":86,"class":96},"OTHER",7,{"id":99,"slug":100,"hasResults":11,"nctId":101,"briefTitle":102,"officialTitle":103,"acronym":104,"eligibilityCriteria":105,"healthyVolunteers":11,"sex":17,"minAge":106,"maxAge":4,"enrollmentInfo":107,"targetDuration":4,"studyType":23,"phases":109,"briefSummary":111,"conditions":112,"keywords":134,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":158,"lastUpdatePostDateStruct":159,"startDateStruct":160,"completionDateStruct":162,"leadSponsor":164,"locationsCount":166},"100562786","phase-1-study-in-children-and-adolescents-of-177lu-dotatate-lutathera-combined-with-the-parp-inhibitor-olaparib-for-the-treatment-of-recurrent-or-relapsed-solid-tumours-expressing-somatostatin-receptor-sstr-luparped-100562786","NCT06607692","Study in Children and Adolescents of 177Lu-DOTATATE (Lutathera®) Combined With the PARP Inhibitor Olaparib for the Treatment of Recurrent or Relapsed Solid Tumours Expressing Somatostatin Receptor (SSTR) (LuPARPed).","Single-arm Open-label Phase I\u002FII Study in Children and Adolescents of 177Lu-DOTATATE (Lutathera®) Combined With the PARP Inhibitor Olaparib for the Treatment of Recurrent or Relapsed Solid Tumours Expressing Somatostatin Receptor (SSTR) (LuPARPed).","LUPARPED","Inclusion criteria:\n\n* 18 months - 18 years of age at the time of the initial diagnosis.\n* ≥ 3 years at the moment of inclusion in the trial.\n* Diagnosis: relapsed\u002Frefractory solid tumours with positive uptake on SSTR-PET (PET-CT or PET-MRI), performed in the previous three months before entering the study.\n\nThe evaluation of SSTR expression will be classified according to a qualitative 4-point scale: SSTR expression V (visual score):\n\n* Score = 0: Below or equal to blood pool\n* Score = 1: Above blood pool and lower than liver\n* Score = 2: Equal to or above liver and lower than spleen\n* Score = 3: Equal to or above spleen\n\nPatients with scores ≥ 2 in the majority of the tumoral lesions will be considered to have a positive SSTR-PET and will be therefore eligible for the trial. Patients with a higher score are presumed to have a better response to the treatment.\n\nIt is admissible to have non-measurable disease only (e.g., HR-NB with bone-only or bone-marrow-only active disease).\n\n* Performance status ≥ 50% according to Lansky scale (\\\u003C16 years old) or Karnofsky scale (for ≥16 years old).\n* Life expectancy of at least 3 months.\n* Availability of ability to swallow tablets or capsules.\n* Adequate organ function within 28 days prior to enrolment, as defined by:\n\n  * Hb ≥10 g\u002Fdl (packed red blood transfusion is acceptable up to 24 hours prior starting treatment);\n  * White blood cell (WBC) count ≥ 2500\u002FμL (equivalent to 2.5 x 109\u002FL)\n  * Absolute Neutrophil Count (ANC) ≥ 1000\u002Fμl;\n  * Platelets ≥ 100.000\u002Fμl, without transfusion in the prior ≥7 days;\n  * Serum plasma creatinine ≤ 1.5 x upper limit of normal (ULN) OR estimated glomerular filtration rate (eGFR) ≥ 60 mL\u002Fmin\u002F1.73m2 (assessed by 2009-Schwartz formula).\n  * Total bilirubin ≤ 1.5 x the institutional ULN. For patients with known Gilbert's Syndrome ≤ 3.0 ULN is permitted.\n  * Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤ 3.0 ULN.\n  * Albumin \\>3.0 g\u002FdL (3.0 g\u002FdL is equivalent to 30 g\u002FL)\n* A negative serum or urine pregnancy test in women with onset of menses or ≥12 years of age.\n* Patients of reproductive potential must agree to use highly effective contraceptive methods for the entire study duration and up to 7 months, in case of females, and 4 months in case of males, after the last dose of Lutathera, or up to 6 months, in case of females, and 3 months in case of males, after the last dose of olaparib, whichever takes places later.\n* Have the ability to comprehend and willingness to provide written informed consent (ICF) for the study before patient registration or any trial-related screening procedures. If the patient is \\\u003C18 years old, the written informed consent must be signed by the parent(s) or legal guardian(s) according to national regulations. In the case of patients between 12 and 17 years, they must sign an assent form, and if the patient turns 18 during their participation in the study, they must sign an informed consent form.\n* Adequate recovery from major surgery prior to receiving study treatment.\n* Absence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before registration in the trial.\n\nExclusion criteria\n\n* Previous significant drug-induced hepatitis toxicity experienced in the past that has required treatment dose reductions, treatment discontinuation or that, at the investigator discretion, could infer a risk.\n* Having received more than one previous treatment with other radiolabelled somatostatin analogues.\n* Inability to swallow tablets or capsules.\n* Subjects who are currently receiving any other anticancer and\u002For investigational agents (e.g. chemotherapy, immunotherapy or biological therapy \\[including monoclonal antibodies\\]). There must be at least 28 days of washout from any prior treatment. In case of checkpoints inhibitors, there should be at least 4 months of washout. Palliative Radiation Therapy for symptom control (e.g. pain relief) could be acceptable, at the discretion of the investigator.\n* Treatment with long-acting somatostatin analogues within 28 days prior the administration of 177Lu-DOTATATE.\n* Known hypersensitivity to any of the excipients.\n* Subjects who have an uncontrolled infection.\n* Lactating women.","3 Years",{"count":108,"type":22},25,[110,25],"PHASE1","Study in children and adolescents of 177Lu DOTATATE (Lutathera®) combined with the PARP inhibitor olaparib for treatment of recurrent or relapsed solid tumours expressing somatostatin receptors (SSTR) (LuPARPed)",[113,114,57,115,116,117,118,119,120,121,122,123,124,125,126,127,128,129,130,131,132,133],"Solid Tumor Cancer","Medulloblastoma","High Grade Gliomas","Meningioma","Paraganglioma","Pheochromocytoma","Neuroendocrine Tumours (NET)","Adrenal Tumours","DIPG","Glioblastoma","Glioblastoma (GBM)","Osteosarcoma Recurrent","Carcinoma Adrenal","Synovial Sarcomas","Esthesioneuroblastoma","Neuroblastoma Recurrent","Solid Tumor Refractory to Conventional Treatment","Medulloblastoma Recurrent","Paraganglioma\u002F Phaeochromocytoma","Ependymoma","Ependymoma Recurrent",[135,136,137,138,139,140,141,142,143,144,145,146,147,121,148,149,150,151,152,153,154,155,156,132,157],"solid tumours","somatostatin receptors","SSTR","LUTETIUM","OLAPARIB","MEDULLOBLASTOMA","NEUROBLASTOMA","HIGH GRADE GLIOMA","MEININGIOMA","PARAGANGLIOMA","PHEOCHROMOCYTOMA","NEUROENDOCRINE TUMOURS","ADRENAL TUMOR","GLIOBLASTOMA","OSTEOSARCOMA","CARCINOMA ADRENAL","SYNOVIAL SARCOMA","ESTHESIONEUROBLASTOMA","RECURRENT","RELAPSED","RELAPSE","REFRACTORY","Ependimoma Recurrent","2026-06-02",{"date":88,"type":34},{"date":161,"type":34},"2024-09-20",{"date":163,"type":22},"2029-12",{"name":165,"class":96},"Fundación de investigación HM",1,{"id":168,"slug":169,"hasResults":11,"nctId":170,"briefTitle":171,"officialTitle":172,"acronym":4,"eligibilityCriteria":173,"healthyVolunteers":11,"sex":17,"minAge":174,"maxAge":175,"enrollmentInfo":176,"targetDuration":4,"studyType":23,"phases":178,"briefSummary":179,"conditions":180,"keywords":181,"overallStatus":58,"whyStopped":4,"lastUpdateSubmitDate":188,"lastUpdatePostDateStruct":189,"startDateStruct":191,"completionDateStruct":193,"leadSponsor":195,"locationsCount":166},"100634262","phase-2-patients-between-the-ages-of-12-months-to-21-years-with-newly-diagnosed-high-risk-neuroblastoma-will-receive-childrens-oncology-group-cog-type-recommended-therapy-with-the-addition-of-naxitamab-and-granulocyte-macrophage-colony-stimulating-factor-gm-csf-to-induction-cycles-1-5-100634262","NCT07537400","Patients Between the Ages of 12 Months to 21 Years With Newly-Diagnosed High-Risk Neuroblastoma Will Receive Children's Oncology Group (COG) Type Recommended Therapy With the Addition of Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) to Induction Cycles 1-5","Phase II Trial of Naxitamab, Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Combination With Induction Chemotherapy for Patients With Newly-Diagnosed High-Risk Neuroblastoma","Inclusion Criteria:\n\n* Age - 12 months to 21 years at protocol enrollment\n* Clinical eligibility criteria:\n\n  1. Newly diagnosed high risk neuroblastoma.\n  2. BOTH stage M (INRG - International Neuroblastoma Risk Group) and age ≥547 days.\n  3. Patients ≥ 547 days of age who were initially diagnosed with INRG L1 or L2 disease but progress to Stage M without chemotherapy\n  4. Patients \\\u003C 547 days of age with INRG Stage M or MS disease and patients of any age with INRG L2 with MYCN amplification (v-myc avian myelocytomatosis viral related oncogene)\n* Pathology:\n\n  1. Neuroblastoma (NBL) or ganglioneuroblastoma (nodular) verified by tumor pathology analysis, or\n  2. demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamines\n* Molecular testing:\n\n  1. MYCN testing will be done by FISH (Fluorescence In Situ Hybridization) assessment of the FFPE (Formalin-Fixed Paraffin-Embedded) material submitted from the tumor mass. \\> 4-fold increase in MYCN signals as compared to reference signals will qualify as MYCN amplified.\n  2. ONCOMINE testing will evaluate ALK (Anaplastic Lymphoma Kinase) mutation\n* Timing of patient enrollment\n\n  1. Patients will be enrolled up to 6 weeks from primary diagnosis\n  2. Pre-study imaging tests are acceptable up to 3 weeks prior to study enrollment and only if done after any pre-protocol chemotherapy.\n* Pre-treatment functional status:\n\n  1. No active bacterial infection without adequate antibiotic therapy\n  2. No uncontrolled viral infection - decision will be made after infectious disease consultation\n  3. No uncontrolled organ dysfunction:\n\n  i. Renal function based on the Schwartz formula (Schwartz et al. J. Peds, 106:522, 1985). If creatinine level is abnormal chemotherapy treatment will be planned in consultation with Nephrology service. Naxitamab will be initiated if creatinine level is up to 1.2 of normal.\n\nii. Adequate liver function defined as: -\n\n1. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age,\n2. and SGPT (Serum Glutamic Pyruvic Transaminase - ALT) ≤ 10 x ULN\\* iii. Adequate cardiac function defined as: -\n\n1\\. Shortening fraction of ≥ 27% by echocardiogram, 2. or Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram.\n\nExclusion Criteria:\n\n* Subjects who have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and\u002For no more than 1 cycle of chemotherapy.\n* This will not restrict the emergency regimen at initial diagnosis.\n* Patients who are 365-546 days of age with INRG Stage M and MYCN non-amplified NBL, irrespective of additional biologic features.\n* Patients ≥547 days of age with INRG Stage L2, MYCN non-amplified NBL, regardless of additional biologic features.\n* Patients with known bone marrow failure syndromes.\n* Patients on chronic immunosuppressive medications (e.g., tacrolimus, cyclosporine, corticosteroids) for reasons other than prevention\u002Ftreatment of allergic reactions and adrenal replacement therapy are not eligible. Topical and inhaled corticosteroids are acceptable.\n* Patients with a primary immunodeficiency syndrome who require ongoing immune globulin replacement therapy.\n* Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required prior to enrollment for female patients of childbearing potential.\n* Lactating females who plan to breastfeed their infants.\n* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation.","12 Months","21 Years",{"count":177,"type":22},10,[25],"This clinical trial will evaluate the safety of chemoimmunotherapy with Naxitamab and COG-type induction chemotherapy in newly-diagnosed patients with high-risk neuroblastoma. We aim to recruit 10 patients over the next 2 years.",[29],[81,182,183,184,185,186,187],"High-risk","Resistant to treatment","Newly-diagnosed","Naxitamab","Anti-GD2 antibody","Chemotherapy","2026-04-16",{"date":190,"type":34},"2026-04-17",{"date":192,"type":22},"2026-05",{"date":194,"type":22},"2032-04",{"name":196,"class":96},"Shaare Zedek Medical Center",{"id":198,"slug":199,"hasResults":11,"nctId":200,"briefTitle":201,"officialTitle":202,"acronym":203,"eligibilityCriteria":204,"healthyVolunteers":11,"sex":17,"minAge":174,"maxAge":175,"enrollmentInfo":205,"targetDuration":4,"studyType":23,"phases":207,"briefSummary":208,"conditions":209,"keywords":212,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":224,"lastUpdatePostDateStruct":225,"startDateStruct":226,"completionDateStruct":228,"leadSponsor":230,"locationsCount":166},"100631561","phase-1-dual-target-gd2b7-h3-car-nk-cells-for-pediatric-relapsed-or-refractory-neuroblastoma-100631561","NCT07502287","Dual-Target GD2\u002FB7-H3 CAR-NK Cells for Pediatric Relapsed or Refractory Neuroblastoma","A Phase 1\u002FPhase 2, Open-Label Study of BiomarkerInformed, Allogeneic Dual-Target GD2\u002FB7-H3 (CD276) CAR-NK Cells in Children and Young Adults With Relapsed or Refractory Neuroblastoma","DUAL-NK-NB","Inclusion Criteria:\n\n* Age 12 months to 21 years at consent\u002Fassent.\n* Histologically confirmed neuroblastoma or ganglioneuroblastoma with relapsed, refractory, progressive, or persistent high-risk disease for which no curative standard option is available.\n* Measurable or evaluable disease according to revised International Neuroblastoma Response Criteria (rINRC), including MIBG-avid disease, CT\u002FMRI-evaluable soft-tissue disease, and\u002For bone marrow disease.\n* Tumor material available for central assessment of GD2 and B7-H3 expression; at least one target must be positive.\n\nGD2 is treated as the core target and B7-H3 as the complementary target for correlative target-prioritization analyses.\n\n* Prior exposure to standard neuroblastoma therapy, including anti-GD2-based therapy, unless contraindicated, unavailable, or declined for a documented medical reason.\n* Lansky or Karnofsky performance score \\>= 50.\n* Life expectancy \\>= 8 weeks.\n* Recovery from clinically significant acute toxicities of prior therapy and protocol-defined washout from chemotherapy, biologics, radiation, and prior cell therapy.\n* Adequate organ function: hematologic, renal, hepatic, cardiac, and pulmonary function considered sufficient by protocoldefined laboratory and clinical thresholds.\n* Negative pregnancy test for patients of childbearing potential and agreement to use effective contraception during the protocol-defined period.\n* Written informed consent from parent\u002Flegal guardian and assent from the participant when appropriate.\n\nExclusion Criteria:\n\n* Active uncontrolled infection, including bacteremia, uncontrolled viral infection, or invasive fungal disease.\n* Pregnancy or breastfeeding.\n* Active grade \\>= 2 graft-versus-host disease, or systemic immunosuppression for treatment\u002Fprevention of graft-versushost disease within the protocol-defined washout period after prior allogeneic transplant.\n* Symptomatic or unstable central nervous system disease requiring urgent medical intervention.\n* Prior genetically modified cellular therapy within the protocol-defined washout window, or unresolved clinically significant toxicity from prior cell therapy.\n* Active autoimmune disease requiring systemic immunosuppressive therapy.\n* Clinically significant uncontrolled cardiovascular, pulmonary, hepatic, renal, or neurologic disorder that would increase study risk.\n* Known hypersensitivity to fludarabine, cyclophosphamide, study-product components, or supportive-care medications required by the protocol.\n* Known uncontrolled HIV infection or uncontrolled hepatitis B or C.\n* Any medical, psychosocial, or logistical condition that, in the investigator's judgment, would make study participation unsafe or would impair protocol adherence.",{"count":206,"type":22},36,[110,25],"This illustrative Phase 1\u002FPhase 2 study tests allogeneic dual-target GD2\u002FB7-H3 (CD276) CAR-NK cells in children and young adults with relapsed or refractory neuroblastoma. After lymphodepletion, participants receive IV CAR-NK cells;Part A defines the RP2D and Part B estimates preliminary activity",[210,211,29,55],"Relapsed Neuroblastoma","Refractory Neuroblastoma",[213,214,215,216,217,218,219,220,221,222,223],"pediatric neuroblastoma","CAR-NK","GD2","B7-H3","CD276","dual-targeting","relapsed\u002Frefractory","allogeneic","solid tumor immunotherapy","cell therapy","biomarkerinformed design","2026-03-25",{"date":36,"type":34},{"date":227,"type":34},"2026-03-02",{"date":229,"type":22},"2028-06-17",{"name":231,"class":41},"Beijing Biotech",{"id":233,"slug":234,"hasResults":11,"nctId":235,"briefTitle":236,"officialTitle":237,"acronym":238,"eligibilityCriteria":239,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":240,"enrollmentInfo":241,"targetDuration":4,"studyType":23,"phases":242,"briefSummary":244,"conditions":245,"keywords":246,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":247,"lastUpdatePostDateStruct":248,"startDateStruct":250,"completionDateStruct":252,"leadSponsor":254,"locationsCount":256},"100579996","resource-intervention-to-support-equity-rise-in-high-risk-neuroblastoma-100579996","NCT06831552","Resource Intervention to Support Equity (RISE) in High-Risk Neuroblastoma","A Randomized Phase II Trial Evaluation of the Addition of the Resource Intervention to Support Equity (RISE) to Usual Supportive Care for Children With Newly Diagnosed High-Risk Neuroblastoma","RISE in HR NBL","Inclusion Criteria:\n\nPatient cohort: The randomized Phase II multi-center RISE intervention will be conducted among a population of poverty-exposed children with high-risk neuroblastoma. Poverty will be a priori defined as parent-reported low-income (\\\u003C200% Federal Poverty Level). Children receiving treatment for cancer at study sites will serve as the study cohort, with parents\u002Fguardians as survey informants and intervention recipients on behalf of their minor children given that parents (not children) typically manage household finances.\n\n* Patient newly diagnosed with high-risk neuroblastoma\n* Patient has established care at study site and initiated cancer-directed therapy\n* Patient has not yet initiated Induction Cycle 3\n* Patient aged 0-17 years at the time of consent\n* Parent\u002Fguardian screened positive for self-reported low-income (\\\u003C200% Federal Poverty) \\*\n* Family primary residence in MA, PA, IL, CA, WA, CT, GA, WI and OH\n* Both patients co-enrolled on ANBL2131 or those receiving standard of care therapy at their center are eligible to participate\n* Patients of all languages are eligible to participate\n\nExclusion Criteria:\n\n* Foreign national family receiving care as an Embassy-pay patient.\n* Child or household member receiving SSI","17 Years",{"count":51,"type":22},[243],"NA","The goal of this study is to test if the addition of a novel income-poverty targeted supportive care intervention (Pediatric Resource Intervention to Support Equity \\[Pediatric RISE\\]) to usual supportive care for low-income children with high-risk neuroblastoma can improve parent- and child-centered outcomes.\n\nParticipants will be randomized to receive one of the following for 6-months:\n\n* Usual supportive care alone or\n* Usual supportive care plus Pediatric RISE",[81,82],[81,82],"2026-02-09",{"date":249,"type":34},"2026-02-10",{"date":251,"type":34},"2025-04-30",{"date":253,"type":22},"2029-06-01",{"name":255,"class":96},"Dana-Farber Cancer Institute",6,{"id":258,"slug":259,"hasResults":11,"nctId":260,"briefTitle":261,"officialTitle":262,"acronym":4,"eligibilityCriteria":263,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":264,"enrollmentInfo":265,"targetDuration":4,"studyType":23,"phases":267,"briefSummary":268,"conditions":269,"keywords":271,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":247,"lastUpdatePostDateStruct":277,"startDateStruct":278,"completionDateStruct":280,"leadSponsor":282,"locationsCount":166},"100556699","phase-2-n10-a-study-of-reduced-chemotherapy-and-monoclonal-antibody-mab-based-therapy-in-children-with-neuroblastoma-100556699","NCT06528496","N10: A Study of Reduced Chemotherapy and Monoclonal Antibody (mAb)-Based Therapy in Children With Neuroblastoma","N10: Reduced Therapy for High-Risk Neuroblastoma","Inclusion Criteria:\n\n* Diagnosis of NB as defined by histopathology, BM metastases plus high urine catecholamine levels, or positivity in MIBG scan.\n* HR-NB, defined as MYCN-amplified stage L2\u002FM\u002FMS at any age and stage M in patients \\>18 months old.\n* No more than one prior cycle of HR-NB chemotherapy\n* Age \\\u003C19 years.\n* Signed informed consent indicating awareness of the investigational nature of this treatment.\n\nExclusion Criteria:\n\n* Severe dysfunction of major organs, i.e., renal, cardiac, hepatic, neurologic, pulmonary, hematologic, or gastrointestinal toxicity \\>\u002F= to grade 3\n\n  °Organ dysfunction due to direct effects of primary tumor or metastatic disease, or due to paraneoplastic syndromes associated with neuroblastoma, will not be considered in exclusion criteria. Similarly, complications of interventions to control above direct effects (e.g., steroid- induced hyperglycemia or hypertension) will also not be considered in exclusion criteria\n* Inability to comply with protocol requirements\n* Pregnancy","19 Years",{"count":266,"type":22},45,[25],"The purpose of this study is to find out whether N10 chemotherapy is a safe and effective treatment for children with high-risk neuroblastoma.",[82,81,270],"Childhood Neuroblastoma",[272,273,274,275,86,276],"High-risk neuroblastoma","neuroblastoma","childhood neuroblastoma","N10","24-144",{"date":249,"type":34},{"date":279,"type":34},"2024-07-22",{"date":281,"type":22},"2029-07-22",{"name":86,"class":96},{"id":284,"slug":285,"hasResults":11,"nctId":286,"briefTitle":287,"officialTitle":288,"acronym":4,"eligibilityCriteria":289,"healthyVolunteers":11,"sex":17,"minAge":174,"maxAge":290,"enrollmentInfo":291,"targetDuration":4,"studyType":23,"phases":293,"briefSummary":294,"conditions":295,"keywords":296,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":297,"lastUpdatePostDateStruct":298,"startDateStruct":300,"completionDateStruct":302,"leadSponsor":304,"locationsCount":306},"100577867","phase-1-study-of-halkcar-t-cells-for-patients-with-relapsedrefractory-high-risk-neuroblastoma-100577867","NCT06803875","Study of hALK.CAR T Cells for Patients With Relapsed\u002FRefractory High-risk Neuroblastoma","A Phase 1\u002F2 Study of hALK.CAR T Cells for Patients With Relapsed\u002FRefractory High-risk Neuroblastoma","Inclusion Criteria:\n\n1. Age ≥ 12 months and \\\u003C 30 years at the time of consent. The first patient on each dose level will need to be age ≥ 6 years old\n2. Disease Status:\n\n   1. Patients must have histologic verification of neuroblastoma at diagnosis or at relapse\n   2. Patients must have high-risk neuroblastoma according to Children's Oncology Group (COG) risk classification at time of study enrollment\n   3. Patients must have persistent\u002Frefractory or relapsed disease for which standard curative measures are no longer effective, as defined in the protocol\n   4. Patients must have evaluable or measurable disease per the revised International Neuroblastoma Response Criteria (INRC)\n3. Adequate washout from prior treatment regimens\n4. Adequate organ function\n5. Adequate performance status defined as Lansky or Karnofsky performance score ≥50%\n6. Subjects of reproductive potential must agree to use acceptable birth control methods\n7. Signed informed consent\n\nExclusion Criteria:\n\n1. Pregnant or nursing (lactating) women\n2. Patients with uncontrolled active infection\n3. Patients who are concurrently receiving other investigational agents\n4. Patients who have received prior CART-cell or other gene-modified immune-effector cell therapy, are not eligible unless they are \\>8 weeks from time of infusion, have fully recovered from any associated toxicities and have documented lack of persistence of the product\n5. Patients with a known additional malignancy other than non-melanomatous skin cancer or carcinoma in situ, unless not requiring active treatment and stable or disease-free for at least 3 years\n6. Uncontrolled CNS metastasis\n7. CNS disorder such as cerebrovascular ischemia\u002Fhemorrhage, dementia, cerebellar disease, or autoimmune disease with CNS involvement which may impair the ability to evaluate neurotoxicity\n8. History of severe hypersensitivity reaction to compounds used in the study\n9. HIV\u002FHBV\u002FHCV infection\n10. Patients receiving systemic steroid therapy (physiologic replacement, inhaled steroids and premedication for blood products are allowed)\n11. Primary immunodeficiency or history of systemic autoimmune disease requiring systemic immunosuppression\u002Fdisease modifying agents within the last 2 years\n12. Uncontrolled intercurrent illness\n13. Inability to comply with the study requirements","29 Years",{"count":292,"type":22},42,[110,25],"This Phase 1\u002F2 trial aims to determine the safety and feasibility of administration of autologous chimeric antigen receptor (CAR) T cells targeting the human Anaplastic Lymphoma Kinase (ALK) receptor in pediatric subjects with relapsed or refractory neuroblastoma (NB).\n\nThe trial will be conducted in two phases:\n\nPhase 1 will determine the maximum tolerated dose (MTD) of autologous hALK.CAR T cells using a 3+3 dose escalation design. Phase 2 will be an expansion phase to determine rates of response to hALK.CAR T cells.",[210,211,82],[210,211,29],"2025-12-24",{"date":299,"type":34},"2025-12-26",{"date":301,"type":34},"2025-01-31",{"date":303,"type":22},"2029-12-31",{"name":305,"class":96},"Roberto Chiarle",2,{"id":308,"slug":309,"hasResults":11,"nctId":310,"briefTitle":311,"officialTitle":312,"acronym":313,"eligibilityCriteria":314,"healthyVolunteers":11,"sex":17,"minAge":315,"maxAge":4,"enrollmentInfo":316,"targetDuration":4,"studyType":23,"phases":317,"briefSummary":318,"conditions":319,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":297,"lastUpdatePostDateStruct":322,"startDateStruct":324,"completionDateStruct":326,"leadSponsor":328,"locationsCount":166},"100489256","phase-1-gpc2-car-t-cells-for-relapsed-or-refractory-neuroblastoma-and-metastatic-retinoblastoma-100489256","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.","1 Year",{"count":266,"type":22},[110],"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.",[211,210,82,320,321],"Retinoblastoma","Metastatic Retinoblastoma",{"date":323,"type":34},"2025-12-29",{"date":325,"type":34},"2023-05-23",{"date":327,"type":22},"2030-01-30",{"name":329,"class":96},"Stephan Grupp MD PhD",{"id":331,"slug":332,"hasResults":11,"nctId":333,"briefTitle":334,"officialTitle":334,"acronym":335,"eligibilityCriteria":336,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":175,"enrollmentInfo":337,"targetDuration":4,"studyType":23,"phases":339,"briefSummary":340,"conditions":341,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":343,"lastUpdatePostDateStruct":344,"startDateStruct":346,"completionDateStruct":348,"leadSponsor":350,"locationsCount":352},"100379473","phase-3-high-risk-neuroblastoma-study-2-of-siop-europa-neuroblastoma-siopen-100379473","NCT04221035","High-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN)","HR-NBL2","Inclusion Criteria:\n\nEnrollment in HR-NBL2 will be performed:\n\n* at diagnosis before the beginning of chemotherapy or\n* up to 21 days after one course of Carboplatin-Etoposide for patients with localized neuroblastoma or infants with metastatic neuroblastoma with MYCN amplification or patients with metastatic neuroblastoma treated in emergency or\n* up to 21 days after one course of the current protocol for R-I randomisation (RAPID COJEC\u002FGPOH) low\u002Fintermediate risk neuroblastoma in Germany\u002FNetherlands for patients with localized neuroblastoma or infants with metastatic neuroblastoma with MYCN amplification\n\nHR-NBL2 eligibility criteria:\n\n1. Established diagnosis of neuroblastoma according to the SIOPEN- modified International Neuroblastoma Risk Group (INRG) criteria, High-risk neuroblastoma defined as:\n\n   * Stage M neuroblastoma above 365 days of age at diagnosis (no upper age limit) and Ms neuroblastoma 12-18 months old, any MYCN status or\n   * L2, M or Ms neuroblastoma any age with MYCN amplification, or focal high level MYC or MYCL amplification.\n\n   In Germany, patients aged less than 18 months with stage M and without MYCN amplification will not be enrolled in HR-NBL2 trial.\n2. No previous chemotherapy or up to 21 days after one cycle of Carboplatin-Etoposide chemotherapy for patients with localized neuroblastoma or infants with metastatic neuroblastoma with MYCN amplification or patients with metastatic neuroblastoma treated in emergency or up to 21 days after one course of the current protocol for low\u002Fintermediate risk neuroblastoma in Germany\u002FNetherlands for patients with localized neuroblastoma or infants with metastatic neuroblastoma with MYCN amplification\n3. Females of childbearing potential must have a negative serum or urine pregnancy test within 7 days prior to initiation of treatment. Sexually active patients must agree to use acceptable and appropriate contraception while on HR-NBL2 study and for one year after stopping the study. Acceptable contraception is defined in CTFG Guidelines \"Recommendations related to contraception and pregnancy testing in clinical trials\" (Appendix 11). Female patients who are lactating must agree to stop breast-feeding.\n4. Written informed consent to enter the HR-NBL2 protocol from patient or parents\u002Flegal representative, patient, and age-appropriate assent.\n5. Patient affiliated to a social security regimen or beneficiary of the same according to local requirements.\n6. Patients should be able and willing to comply with study visits and procedures as per protocol\n\nR-I eligibility criteria:\n\n\\- Written informed consent to enter the R-I randomisation from patient or parents\u002Flegal representative, patient, and age- appropriate assent.\n\nIn case of parents'\u002Fpatient's refusal to R-I, or Organ toxicity exclusion criteria at diagnosis, patients can still be enrolled in HR- NBL2 trial with parents'\u002Fpatient's consent before or within 3 weeks from the beginning of chemotherapy\n\nR-HDC randomisation (Single HDC Bu-Mel\u002F Tandem HDC Thiotepa+Bu-Mel) Etoposide or one course of the current protocol for low\u002Fintermediate risk neuroblastoma in Germany\u002FNetherlands). Patients will be treated with the standard induction regimen per country (Rapid COJEC or GPOH) and will be potentially eligible for subsequent randomisations.\n\nRandomisation for HDC strategy will be performed at the end of induction after the disease evaluation and after surgery of the primary tumour for those patients who will receive surgery before HDC.\n\nR-HDC eligibility criteria:\n\n1. \\- Stage M neuroblastoma above 365 days of age at diagnosis, any MYCN status, EXCEPT patients with stage M or Ms 12-18 months old with numerical chromosomal alterations only, and in complete metastatic response at the end of induction: in this case, patients will have surgery and no further treatment.\n\n   OR\n\n   \\- L2, M or Ms neuroblastoma, any age, with MYCN amplification, or focal high level MYC or MYCL amplification\n2. Age \\\u003C 21 years at the time of randomization\n3. Complete response (CR) or partial response (PR) at metastatic sites:\n\n   * Bone disease: mIBG uptake completely resolved or SIOPEN score ≤ 3 and at least 50% reduction in mIBG score (or ≤ 3 bone lesions and at least 50% reduction in number of FDG- PET-avid bone lesions for mIBG-nonavid tumours).\n   * Bone marrow disease: CR and\u002For minimal disease (MD) according to International Neuroblastoma Response Criteria\n   * Other metastatic sites: CR. (after induction chemotherapy +\u002F- surgery), except for distant lymph nodes for which PR is accepted with a possible secondary surgery\n4. Acceptable organ function and performance status:\n\n   * Performance status ≥ 50%.\n   * Hematological status: ANC\\>0.5x109\u002FL, platelets \\> 20x 109\u002FL\n   * Cardiac function: (\\\u003C grade 2)\n   * Normal chest X-Ray and oxygen saturation.\n   * Absence of any toxicity ≥ grade 3. 4) Sufficient collected stem cells available; a total harvest of at least 6 x 106\u002Fkg CD34+ cells, to be stored in at least 4 separate bags to administer at least 3 x 106\u002Fkg CD34+ cells per rescue.\n5. Written informed consent, including agreement of patient or parents\u002Flegal guardian for minors, to enter the R-HDC randomisation.\n6. Patient affiliated to a social security regimen or beneficiary of the same according to local requirements.\n7. Patients should be able and willing to comply with study visits and procedures as per protocol.\n\nIn case of parents'\u002Fpatient's refusal, or insufficient stem cells, collection for tandem HDC but with a minimum of 3 x 106 CD34+ cells\u002Fkg body weight, or in case of patients older than 21 years, or organ toxicity, HDC will consist on the standard HD Bu-Mel and patients will be eligible for the subsequent randomisation.\n\nR-RTx randomisation (Local Radiotherapy) Chemoimmunotherapy arm\n\nR-RTx eligibility criteria:\n\nAn evaluation of the local disease will be performed after HDC\u002FASCR and surgery:\n\n* In case of no local macroscopic disease, all patients will receive 21,6-Gy radiotherapy to the pre-operative tumour bed\n* In case of local macroscopic residual disease, patients will be eligible to R-RTx if the following criteria are met:\n\n  1. No evidence of disease progression after HDC\u002FASCR.\n  2. Interval between the last ASCR and radiotherapy start between 60 and 90 days.\n  3. Performance status greater or equal 50%.\n  4. Hematological status: ANC \\>0.5x109\u002FL, platelets \\> 20x109\u002FL.\n  5. Written informed consent, including agreement of patient or parents\u002Flegal guardian for minors, to enter the R-RTx randomisation.\n  6. Patient affiliated to a social security regimen or beneficiary of the same according to local requirements.\n  7. Patients should be able and willing to comply with study visits and procedures as per protocol.\n\nIn case of parents'\u002Fpatient's refusal of the randomisation, the patient will receive 21.6 Gy radiotherapy to the pre-operative tumour bed.\n\nChemoimmunotherapy arm eligibility criteria:\n\n1. Insufficient metastatic response at the end of induction chemotherapy, defined as:\n\n   * SIOPEN score \\> 3 or less than 50% reduction in mIBG score (or \\> 3 bone lesions or less 50% reduction in number of FDG-PET-avid bone lesions for mIBG-non avid tumours) OR\n   * Bone marrow disease: SD according to International Neuroblastoma Response Criteria OR\n   * Other metastatic sites: PR or SD. For distant lymph nodes: PR and not resectable or SD.\n2. Performance status ≥ 50%.\n3. Hematological status: ANC\\>0.75x109\u002FL without G-CSF for at least 48 hours (or ANC ≥ 0.50 x 109 \u002FL in case of bone marrow involvement), platelets \\> 50x 109\u002FL and rising, without platelets transfusion for 72 hours.\n4. AST or ALT ≤7.5 ULN and total bilirubin ≤1.5 ULN. In patients with liver metastases, total bilirubin ≤2.5 ULN is allowed.\n5. No active infection;\n6. No grade \\>2 gastrointestinal toxicity.\n7. No grade ≥ 3 toxicity related to previous treatment.\n8. Oxygen saturation \\> 94%\n\nNon-inclusion criteria for HR-NBL2:\n\n1. Any negative answer concerning the HR-NLB2 inclusion criteria\n2. Patient under guardianship or deprived of his liberty by a judicial or administrative decision or incapable of giving his consent.\n3. Participating in another clinical study with an IMP while on study treatment.\n4. Chronic inflammatory bowel disease and\u002For bowel obstruction.\n5. Pregnant or breastfeeding women.\n6. Known hypersensitivity to the active substance or to any of the excipients of the study drugs\n7. Concomitant self-medication medicine that in the investigator opinion could interact with study treatments, including herbal medicine (e.g. St John's Wort (Hypericum Perforatum).\n\nNon-inclusion criteria specific to the R-I randomisation (RAPID COJEC\u002FGPOH):\n\n1. Urinary tract obstruction ≥ grade 3\n2. Heart failure or myocarditis ≥ grade 2, any arrhythmia or myocardial infection\n3. Peripheral motor or sensory neuropathy ≥ grade 3\n4. Demyelinating form of Charcot-Marie-Tooth syndrome\n5. Hearing impairment ≥ grade 2\n6. Concurrent prophylactic use of phenytoin\n7. Cardiorespiratory disease that contraindicates hyperhydration\n\nNon-inclusion criteria common to all randomisations (R-I, R-HDC, and R-RTx):\n\n1. Any negative answer concerning the inclusion criteria of R-I or R- HDC or R-RTx will render the patient ineligible for the corresponding therapy phase randomisation. However, these patients may remain on study and be considered to receive standard treatment of the respective therapy phase, and may be potentially eligible for subsequent randomisations.\n2. Liver function: Alanine aminotransferase (ALT) \\> 3.0 x ULN and blood bilirubin \\> 1.5 x ULN (toxicity ≥ grade 2). In case of toxicity ≥ grade 2, call national principal investigator study coordinator to discuss the feasibility.\n3. Renal function: Creatinine clearance and\u002For GFR \\\u003C 60 ml\u002Fmin\u002F1.73m² (toxicity ≥ grade 2). If GFR \\\u003C 60ml\u002Fmin\u002F1.73m², call national principal investigator study coordinator to discuss about the treatment.\n4. Dyspnea at rest and\u002For pulse oximetry \\\u003C95% in air (only for R-HDC, and R-RTx)\n5. Any uncontrolled intercurrent illness or infection that in the investigator opinion would impair study participation.\n6. Concomitant use with yellow fever vaccine and with live virus or bacterial vaccines.\n7. Patient allergic to peanut or soya.\n\nNon-inclusion criteria to R-HDC:\n\n\\- Any negative answer concerning the R-HDC inclusion criteria\n\nNon-inclusion criteria to chemoimmunotherapy arm:\n\n\\- Any negative answer concerning the inclusion criteria of chemoimmunotherapy arm.",{"count":338,"type":22},800,[26],"This is an international multicenter, open-label, randomized phase III trial including three sequential randomizations to assess efficacy of induction and consolidation chemotherapies and radiotherapy for patients with high-risk neuroblastoma.",[29,342],"Patient With Insufficient Response Chemoimmunotherapy","2025-06-25",{"date":345,"type":34},"2025-06-26",{"date":347,"type":34},"2019-11-05",{"date":349,"type":22},"2032-11",{"name":351,"class":96},"Gustave Roussy, Cancer Campus, Grand Paris",142,{"id":354,"slug":355,"hasResults":11,"nctId":356,"briefTitle":357,"officialTitle":357,"acronym":358,"eligibilityCriteria":359,"healthyVolunteers":11,"sex":17,"minAge":315,"maxAge":19,"enrollmentInfo":360,"targetDuration":4,"studyType":23,"phases":362,"briefSummary":363,"conditions":364,"keywords":365,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":367,"lastUpdatePostDateStruct":368,"startDateStruct":370,"completionDateStruct":372,"leadSponsor":374,"locationsCount":166},"100460188","phase-1-immunotherapy-with-dinutuximab-beta-in-combination-with-chemotherapy-for-the-treatment-of-patients-with-primary-neuroblastoma-refractory-to-standard-therapy-and-with-relapsed-or-progressive-disease-100460188","NCT05272371","Immunotherapy With Dinutuximab Beta in Combination With Chemotherapy for the Treatment of Patients With Primary Neuroblastoma Refractory to Standard Therapy and With Relapsed or Progressive Disease","ChIm-NB-PL","Inclusion Criteria:\n\n1. Diagnosis of NBL according to international criteria (International Neuroblastoma Risk Group, INRG).\n2. Patients 1-18 years of age with HR-NBL with primary refractory disease, disease progression or recurrence.\n3. Adequate function of vital organs (if abnormal, dysfunction below grade 4 according to the CTC AE WHO classification, except for disorders defined in the exclusion criteria).\n4. Life expectancy ≥6 months.\n5. Obtaining the informed written consent of the patient and\u002For statutory representative for the treatment.\n6. Female patients of childbearing potential must consent to the use of effective contraception; Breastfeeding patients must consent to the termination of breastfeeding.\n7. Patients who have previously received immunotherapy with DB or other anti-GD2 specific antibodies may be eligible for this study.\n\nExclusion Criteria:\n\n1. Patients with toxicities of ≥3 CTCAE WHO grade, except hearing impairment, hematological disorders, liver and kidney disorders.\n2. Patients with neurological toxicities of ≥2 CTCAE WHO grade.\n3. Active life-threatening infection until stabilization of the patient's condition.\n4. Pregnancy and \u002F or lactation.\n5. Sexually active patients who refuse to use an effective method of contraception.\n6. Current treatment with experimental drugs or use of such treatment within 2 weeks before signing the informed consent to participate in the study.\n7. Radiotherapy within 3 weeks prior to the start of the study.\n8. Participation in another clinical trial within 6 months before signing the informed consent to participate in the trial (not applicable to clinical trials in 1st line of treatment in HR-NBL).\n9. Lack of informed written consent to treatment.",{"count":361,"type":22},20,[110],"Safety evaluation and initial efficacy evaluation will be conducted in a group of patients as a non-commercial, open-label clinical trial of dinutuximab beta (Qarziba) phase IIa.\n\nThe investigational medicinal product will be dinutuximab beta (anti-GD2 antibodies against GD2 disialoganglioside on NBL cells) at a dose of 10 mg \u002F m2 \u002F day by continuous infusion for 5 days in combination with irinotecan \u002F temozolomide, topotecan \u002F temozolomide or N5 \u002F N6 chemotherapy GPOH protocol.\n\nThe study group will be patients with recurrent \u002F progression of NBL or disease resistant to first-line treatment, for whom there are currently no standards of management, and the treatment methods used so far do not give a chance to achieve a permanent remission of the disease. After diagnosis of relapse \u002F progression or resistance to treatment, it is permissible to administer 2 cycles of standard chemotherapy prior to enrollment in the study.\n\nThe study plans to recruit 20 patients who will receive 5-7 cycles of DB with chemotherapy. The choice of an appropriate chemotherapy regimen will depend on the patient's prior treatment and tolerance.\n\nThe safety assessment will be conducted based on the registration of the types and frequency of adverse reactions identified on the basis of the registration of clinical parameters, symptoms and \u002F or diseases reported by the patient or identified during the intervention and \u002F or follow-up visits, abnormal laboratory and \u002F or imaging test results.\n\nThe initial assessment of the effectiveness will consist in comparing the percentage of objective responses obtained and the annual EFS and PFS (imaging tests, including scintigraphy, bone marrow examination and tumor markers). The study also included an exploratory evaluation of the usefulness of immunological, genetic and other studies.",[29],[366],"neuroblastoma dinutuksymab beta chemoimmunotherapy","2022-03-08",{"date":369,"type":34},"2022-03-09",{"date":371,"type":34},"2021-12-01",{"date":373,"type":22},"2026-12-31",{"name":375,"class":96},"Jagiellonian University"]