[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"wilms-tumor\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:wilms-tumor":513},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,16,0,[8,49,89,117,146,177,207,249,275,298,319,348,375,405,427,452],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100054232","phase-1-trastuzumab-deruxtecan-ds-8201a-for-the-treatment-of-newly-diagnosed-recurrent-or-refractory-osteosarcoma-wilms-tumor-and-desmoplastic-small-round-cell-tumor-100054232",false,"NCT04616560","Trastuzumab Deruxtecan (DS-8201a) for the Treatment of Newly Diagnosed, Recurrent or Refractory Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor","A Phase 1\u002F2 Study of DS-8201a (NSC# 807708) in Children, Adolescents, or Young Adults With Recurrent Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor","Inclusion Criteria:\n\n* Phase 1 (Part A): Patients must be at least 2 years and less than 12 years of age at the time of study enrollment\n* Phase 2: Wilms tumor patients (Part B1): All Wilms tumor patients enrolled must be less than 18 years of age at enrollment\n\n  * Until the completion of the Phase 1 dose confirmation, patients must be at least 12 years of age and less than 18 years of age at the time of study enrollment\n  * Following dose confirmation of DS-8201a in children at least 2 to less than 12 years old in the Phase 1 component, Wilms tumor patients at least 2 to less than 18 years of age will be allowed on the Phase 2 component\n* Phase 2: DSRCT patients (Part B2): Until the completion of the Phase 1 component, patients enrolling on the Phase 2 component of the study must be from at least 12 to 39 years of age at the time of study enrollment\n\n  * Following dose confirmation of DS-8201a in children at least 2 to less than 12 years old in the Phase 1 component, DSRCT patients at least 2 to 39 years of age will be allowed on the Phase 2 component\n* Patients must have had histologic verification of Wilms tumor or desmoplastic small round cell tumor at original diagnosis or relapse\n* Solid tumors: Patients must have measurable disease according to Response Evaluation Criteria in Solid Tumors (RECIST) 1.1. Patients with clinically inactive brain metastases may be included in the study. Patients with treated brain metastases that are no longer symptomatic and who require no treatment with corticosteroids or anticonvulsants may be included in the study if they have recovered from the acute toxic effect of radiotherapy. Lastly, patient must have unresectable lesions or lesions with no intention to surgically remove the lesions in the 6 months following enrollment\n* Wilms tumor: WT patients must have either refractory disease or a very high risk relapse, defined as ANY of the following:\n\n  * Relapse after initial treatment with 4 or more chemotherapy agents (e.g. Regimens vincristine, dactinomycin, doxorubicin, cyclophosphamide, etoposide and radiation \\[M\\], vincristine, dactinomycin and doxorubicin, vincristine, and irinotecan \\[MVI\\], doxorubicin, vincristine, cyclophosphamide, carboplatin, etoposide and radiation \\[UH-1\\], doxorubicin, vincristine, cyclophosphamide, carboplatin, etoposide, vincristine, and irinotecan \\[UH-2\\], vincristine, irinotecan, cyclophosphamide, carboplatin, etoposide and doxorubicin \\[UH-3\\], and etoposide, carboplatin, cyclophosphamide, and doxorubicin \\[HR-1\\])\n  * Relapse with high risk histology (anaplasia, blastemal predominant)\n  * Multiple relapses\n* Desmoplastic small round cell tumor: DSRCT patients with relapsed or refractory disease are eligible\n* Patient's current disease state must be one for which they have received at least standard initial therapy, defined as systemic therapy combined with either radiation or surgery for local control of the primary tumor at diagnosis. Prior therapy after relapse is not required\n* Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0 or 1. Use Karnofsky for patients older than 16 years of age and Lansky for patients 16 years of age and younger\n* Patients must have recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g., blood count criteria, the patient is considered to have recovered adequately\n\n  * Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive: For agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned Research Coordinator prior to enrollment\n\n    * \\>= 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea)\n  * Anti-cancer agents not known to be myelosuppressive (e.g., not associated with reduced platelet or absolute neutrophil count \\[ANC\\] counts): \\>= 7 days after the last dose of agent\n  * Antibodies: \\>= 4 weeks (28 days) must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =\\\u003C 1\n  * Corticosteroids\n  * Hematopoietic growth factors: \\>= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur\n  * Interleukins, interferons and cytokines (other than hematopoietic growth factors): \\>= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)\n  * Stem cell Infusions (with or without total body irradiation \\[TBI\\]):\n\n    * Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: \\>= 84 days after infusion and no evidence of graft versus host disease (GVHD)\n    * Autologous stem cell infusion including boost infusion: \\>= 30 days\n  * Cellular therapy: \\>= 30 days after the completion the infusion of any type of cellular therapy (e.g., modified T cells, natural killer \\[NK\\] cells, dendritic cells, etc.)\n  * Radiation therapy (XRT)\u002Fexternal beam irradiation including protons: \\>= 4 weeks (28 days) including palliative radiation therapy to the chest. \\>= 14 days after palliative local XRT to areas other than the chest or for whole brain radiotherapy\n  * Radiopharmaceutical therapy (e.g., radiolabeled antibody, samarium): \\>= 42 days after systemically administered radiopharmaceutical therapy\n  * Patients must not have received prior HER2 therapies including antibody drug conjugates (e.g. TDM-1 or DS-8201a), HER2 directed cellular therapies, HER2 receptor therapy (e.g. trastuzumab, pertuzumab, margetuximab, zanidatamab, zenocutuzumab) or small molecule antagonists of HER2 (e.g lapatinib, tucatinib, or neratinib). Prior exposure to antibody drug conjugates which do not target HER2 as well as prior treatment with topoisomerase 1 inhibitors (e.g. irinotecan, topotecan) are permitted\n* Patients must be at least 14 days from the date of last surgery\n* Peripheral absolute neutrophil count (ANC) \\>= 1000\u002FuL, (for patients with solid tumors without known bone marrow involvement)\n* Platelet count \\>= 100,000\u002FuL (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) (for patients with solid tumors without known bone marrow involvement)\n* Hemoglobin \\>= 8.0 g\u002FdL at baseline (Red Blood Cell transfusion is not allowed within 1 week prior to enrollment) (for patients with solid tumors without known bone marrow involvement)\n* For patients less than or equal to 17 years old, \"Bedside\" Schwartz formula (2009)\n* Estimated glomerular filtration rate (GFR) (eGFR) ≥ 70 mL\u002Fmin\u002F1.73 m\\^2 (\\> 70mL\u002Fmin\u002F1.73 m\\^2 for patients \\> 17 years old)\n\n  * For patients older than 17 years of age the Cockroft-Gault equation should be utilized to calculate creatinine clearance ≥ 70 ml\u002Fmin:\n  * OR for any age group:\n\n    * A 24 hour urine creatinine clearance ≥ 70 mL\u002Fmin\u002F1.73 m\\^2 (\\> 70mL\u002Fmin for patients ≥ 17 years old) OR\n    * A directly measured GFR ≥ 70 mL\u002Fmin\u002F1.73 m\\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard)\n* Bilirubin (sum of conjugated + unconjugated or total) =\\\u003C 1.5 x upper limit of normal (ULN) for age. For patients with documented Gilbert's syndrome (unconjugated hyperbilirubinemia) bilirubin must be \\\u003C 3 x ULN for age (patients with solid tumors)\n* Aspartate aminotransferase (AST) =\\\u003C 3 x ULN\n* Serum albumin \\>= 2.5 g\u002FdL (patients with solid tumors)\n* International normalized ratio (INR)\u002Fprothrombin time (PT) =\\\u003C 1.5 x ULN. Exception for patients receiving coumarin-derivative anticoagulants or other similar anticoagulant therapy, who must have INR\u002FPT within the therapeutic range as deemed appropriate by the investigator\n* Shortening fraction of \\>= 27% by echocardiogram, or ejection fraction of \\>= 50% by either an echocardiogram (ECHO) or multigated acquisition (MUGA) scan within 28 days before Step 1 enrollment\n* Corrected QT interval (QTc) prolongation to \\\u003C 480 ms based on average triplicate 12-lead electrocardiogram (ECG)\n* Pulse oximetry \\> 93% on room air\n* Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled as evidenced by no increase in seizure frequency in the prior 7 days\n* Nervous system disorders (Common Terminology Criteria for Adverse Events \\[CTCAE\\] version \\[v\\]5) resulting from prior chemotherapy, surgery, and\u002For radiation must be =\\\u003C grade 2, with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible\n* HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months of enrollment are eligible\n* All patients and\u002For their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines\n\nExclusion Criteria:\n\n* Pregnant, planning to become pregnant, or breast-feeding women will not be entered on this study. 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) for the duration of the study and upon completion of the study and for at least 7 months for females and 4 months for males after the last dose of study drug. Abstinence is an acceptable method of birth control\n\n  * Methods considered as highly effective methods of contraception include:\n\n    * Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:\n\n      * Oral\n      * Intravaginal\n      * Transdermal\n    * Progestogen-only hormonal contraception associated with inhibition of ovulation:\n\n      * Oral\n      * Injectable\n      * Implantable\n    * Intrauterine device (IUD)\n    * Intrauterine hormone-releasing system (IUS)\n    * Bilateral tubal occlusion\n    * Vasectomized partner\n    * Complete sexual abstinence defined as refraining from heterosexual intercourse. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not an acceptable method of contraception\n* Non-child-bearing potential defined as pre-menopausal females with a documented tubal ligation or hysterectomy; or postmenopausal defined as 12 months of spontaneous amenorrhea (in questionable cases, a blood sample with simultaneous follicle-stimulating hormone \\[FSH\\] \\> 40 mIU\u002FmL and estradiol \\\u003C 40 pg\u002FmL \\[\\\u003C 147 pmol\u002FL\\] is confirmatory). Females on hormone replacement therapy (HRT) and whose menopausal status is in doubt will be required to use one of the contraception methods outlined for women of child-bearing potential if they wish to continue their HRT during the study. Otherwise, they must discontinue HRT to allow confirmation of post-menopausal status prior to study enrollment. For most forms of HRT, at least 2-4 weeks will elapse between the cessation of therapy and the blood draw; this interval depends on the type and dosage of HRT. Following confirmation of their post-menopausal status, they can resume use of HRT during the study without use of a contraceptive method\n* Male patients must not freeze or donate sperm starting at enrollment and throughout the study period, and at least 4 months after the final study drug administration. Preservation of sperm should be considered prior to enrolment in this study\n* Female patients must not donate, or retrieve for their own use, ova from the time of enrollment and throughout the study treatment period, and for at least 7 months after the final study drug administration\n* Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. If used to modify immune adverse events related to prior therapy, \\>= 14 days must have elapsed since last dose of corticosteroid\n* Patients who are currently receiving another investigational drug are not eligible\n* Patients who are currently receiving other anti-cancer agents are not eligible\n* Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial\n* Patients who are receiving chloroquine or hydroxychloroquine within 14 days are not eligible for this trial\n* Patients who received a live, attenuated vaccine (messenger ribonucleic acid \\[mRNA\\] and replication deficient adenoviral vaccines are not considered attenuated live vaccines) within 30 days prior to enrollment are not eligible for this trial\n\n  * Note: Participants, if enrolled, should not receive live vaccines during the study and up to 90 days after the last dose of study intervention. It is recommended that patients receive a yearly influenza killed vaccination and additional killed vaccinations based on local or national recommendations. Consider vaccination against viral pathogens that cause pneumonias according to local or national guidelines\n* Patients who have received a prior solid organ transplantation are not eligible\n* Patients with a medical history of myocardial infarction within 180 days before enrollment, symptomatic congestive heart failure (CHF) (New York Heart Association Class II to IV) or troponin levels consistent with myocardial infarction as defined according to the manufacturer 28 days prior to enrollment are not eligible\n* Additionally, patients with a history of any of the following congenital heart disease are not eligible:\n\n  * Single ventricle heart defects (hypoplastic left heart syndrome, unbalanced atrioventricular septal defects, double inlet left ventricle, tricuspid atresia, the presence of superior cavopulmonary anastomosis or Fontan palliation);\n  * Unpalliated defects with significant hemodynamic alterations or palliated lesions with residual hemodynamic alterations (ductal-dependent or shunt-dependent physiology, large unrestrictive ventricular septal defect, transposition of the great arteries, greater than moderate atrioventricular valve insufficiency, moderate or greater aortic valve stenosis, moderate or greater aortic valve insufficiency, large atrial septal defects with significant right ventricular volume overload, large patent ductus arteriosus)\n* Patients who have a pleural effusion, ascites or pericardial effusion that requires drainage, peritoneal shunt, or cell-free and concentrated ascites reinfusion therapy (CART) are not eligible. (Drainage and concentrated ascites reinfusion therapy are not allowed within 2 weeks prior to enrollment)\n* Patients who have spinal cord compression or clinically active central nervous system metastases, defined as untreated and symptomatic, or requiring therapy with corticosteroids or anticonvulsants to control associated symptoms are not eligible\n* Patients with a known history of severe hypersensitivity to DS-8201a, any excipient contained in the DS-8201a drug formulation, or HER2-targeted monoclonal antibodies (trastuzumab, pertuzumab, margetuximab) are not eligible\n* Patients who have an uncontrolled infection or non-healing surgical site are not eligible\n* Patients with a known history of substance abuse or any other clinically significant medical conditions (i.e. psychological conditions) that may, in the opinion of the investigator, interfere with the patient's participation in the clinical study or evaluation of the clinical study results are not eligible\n* Patients who have pulmonary compromise, ex hypoxia, resulting from intercurrent pulmonary illnesses including, but not limited to, any underlying pulmonary disorder (i.e. pulmonary emboli within three months of the study enrollment, severe asthma, severe chronic obstructive pulmonary disease (COPD), restrictive lung disease, pleural effusion etc.), or prior pneumonectomy are not eligible\n* Patients who have a history of (non-infectious) ILD (interstitial lung disease)\u002Fpneumonitis that required steroids, has current ILD\u002Fpneumonitis, or for whom suspected ILD\u002Fpneumonitis cannot be ruled out by imaging at screening are not eligible. Patients who have history of genetic disorders of the lung (i.e. cystic fibrosis 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\n* Patients with known hepatitis B or C with detectable viral load are not eligible\n* Patients with any autoimmune, connective tissue or inflammatory disorders (e.g., rheumatoid arthritis, Sjogren's, sarcoidosis etc.) where there is documented, or a suspicion of pulmonary involvement at the time of enrollment or genetic diseases involving the lung are not eligible\n* Patients with an active primary immunodeficiency are not eligible","ALL","12 Years","39 Years",{"count":20,"type":21},55,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","This phase I\u002FII trial studies the effects of trastuzumab deruxtecan (DS-8201a) in treating patients with osteosarcoma, Wilms tumor (WT) or desmoplastic small round cell tumor (DSRCT) that is newly diagnosed or has come back after a period of improvement (recurrent) or that has not responded to previous treatment (refractory). Trastuzumab deruxtecan is in a class of medications called antibody-drug conjugates. It is composed of a monoclonal antibody, called trastuzumab, linked to a chemotherapy drug, called deruxtecan. Trastuzumab attaches to HER2 positive tumor cells in a targeted way and delivers deruxtecan to kill them.",[28,29,30,31,32,33,34,35],"Desmoplastic Small Round Cell Tumor","Osteosarcoma","Recurrent Desmoplastic Small Round Cell Tumor","Recurrent Kidney Wilms Tumor","Recurrent Osteosarcoma","Refractory Desmoplastic Small Round Cell Tumor","Refractory Wilms Tumor","Wilms Tumor","RECRUITING","2026-07-10",{"date":39,"type":40},"2026-07-13","ACTUAL",{"date":42,"type":40},"2021-03-08",{"date":44,"type":21},"2027-12-31",{"name":46,"class":47},"National Cancer Institute (NCI)","NIH",28,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":4,"eligibilityCriteria":55,"healthyVolunteers":11,"sex":16,"minAge":56,"maxAge":57,"enrollmentInfo":58,"targetDuration":4,"studyType":22,"phases":60,"briefSummary":61,"conditions":62,"keywords":74,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":78,"lastUpdatePostDateStruct":79,"startDateStruct":81,"completionDateStruct":83,"leadSponsor":85,"locationsCount":88},"100564132","phase-1-alphabeta-t-and-b-cell-depletion-with-zoledronic-acid-for-solid-tumors-100564132","NCT06625190","Alpha\u002FBeta T and B Cell Depletion With Zoledronic Acid for Solid Tumors","Allogeneic Stem Cell Transplantation Utilizing Alpha\u002FBeta T Cell and CD19+ B Cell Depletion With Zoledronic Acid in Combination to Treat Pediatric, Adolescent, and Young Adult Patients With Relapsed\u002FRefractory Solid Tumors","Inclusion Criteria:\n\n* Patients 6 months to ≤ 25 years old\n* Relapsed\u002FRefractory Solid Tumor whom failed or deemed ineligible to receive autologous transplant or if autologous transplant did not offer \\>20% chance of cure with the following diseases:\n\n  1. neuroblastoma (high risk with relapsed or refractory disease),\n  2. relapsed\u002Frefractory rhabdomyosarcoma,\n  3. relapsed\u002Frefractory non-rhabdomyosarcoma soft tissue sarcoma (NRSTS): synovial sarcoma, malignant peripheral nerve sheath tumors (MPNST),\n  4. High risk adult type NRSTS: clear cell sarcoma, alveolar soft part sarcoma,\n  5. Other high-risk extracranial solid tumors: desmoplastic small round cell tumors, chordoma, malignant rhabdoid tumor, epithelioid sarcoma, myoepithelial tumor\n  6. relapsed\u002Frefractory bone tumors: osteosarcoma and Ewing sarcoma\u002FPNET, or\n  7. Wilm's tumor or other high-risk solid tumors with \\\u003C10% expected survival with conventional treatment.\n* Subjects must not have more than one active malignancy at the time of enrollment. (Subjects with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen \\[as determined by the treating physician and approved by the PI\\] may be included.)\n* Haplo-identical related donor (at least one full haplotype must be matched).\n* Karnofsky or Lansky score ≥60% at the time of enrollment. Karnofsky scores must be used for patients \\>16 years of age and Lansky scores for patients ≤16 years of age\n* Adequate organ function (within 4 weeks of initiation of preparative regimen), defined as:\n\n  1. Pulmonary: FEV1, FVC, and corrected DLCO must all be ≥ 50% of predicted by pulmonary function tests (PFTs). For children who are unable to perform for PFTs due to age, the criteria are: no evidence of dyspnea at rest and no need for supplemental oxygen.\n  2. Renal: Creatinine clearance or radioisotope GFR ≥60 mL\u002Fmin\u002F1.73 m2 or a serum creatinine based on age\u002Fgender\n  3. Cardiac: Ejection fraction of ≥ 40% by echocardiogram or radionuclide scan (MUGA).\n* Written informed consent obtained from the subject and the subject agrees to comply with all the study-related procedures\n* Individuals of childbearing potential (IOCBP) must be using an adequate method of contraception to avoid pregnancy throughout the study and for one year following transplantation to minimize the risk of pregnancy. Prior to study enrollment, individuals of childbearing potential must be advised of the importance of avoiding pregnancy during trial participation and the potential risk factor for an unintentional pregnancy.\n* Subjects with female partners of child-bearing potential must agree to use physician-approved contraceptive methods (e.g., abstinence, condoms, vasectomy) throughout the study and should avoid conceiving children for one year following stem cell transplantation.\n\nExclusion Criteria:\n\n* Patients with documented uncontrolled infection at the time of study entry are not eligible.\n\n  a. Uncontrolled infection is patient without treatment antimicrobials and\u002For demonstrating progression despite antimicrobials\n* Patients with progressive solid tumor disease after relapsed\u002Frefractory treatment.\n* Demonstrated lack of compliance with medical care, as determined by the treating physician.\n* Patients who have received an allogeneic HSCT within 6 months.\n* Patients who do not have an eligible allogeneic donor available.\n* Patients with a life expectancy \\\u003C3 months\n* Patients not meeting inclusion criteria for organ function.\n* Females or males of childbearing potential who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period and for at least one year after transplantation.\n* Females who are known to be pregnant or breastfeeding.\n* History of any other disease, metabolic dysfunction, clinical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of protocol therapy or that might affect the interpretation of the results of the study or that puts the subject at high risk for treatment complications, in the opinion of the treating physician.\n* Prisoners or subjects who are involuntarily incarcerated, or subjects who are compulsorily detained for treatment of either a psychiatric or physical illness.","6 Months","25 Years",{"count":59,"type":21},27,[24,25],"Hematopoietic stem cell transplantation can cure patients with blood cancer and other underlying diseases. αβ-T cell and B cell depletion has been introduced to decrease GVHD and PTLD and has demonstrated effectiveness for hematologic malignancies and non-malignant diseases additionally increasing the donor pool as to allow for haploidentical transplant to safely occur.\n\nWhile solid tumors can be highly chemotherapy sensitive, many remain resistant and require multimodalities of treatment. Immunotherapy has been developed to harness the immune system in fighting solid tumors, though not all have targeted effects. Some solid tumors are treated with autologous transplants; however, they do not always demonstrate an improved event free survival or overall survival. There has been evidence of the use of allogeneic stem cell transplants to provide a graft versus tumor effect, though studies remain limited.\n\nBy utilizing αβ-T cell and B cell depletion for stem cell transplants and combining with zoledronic acid, the immune system may potentially be harnessed and enhanced to provide an improved graft versus tumor effect in relapsed\u002Frefractory solid tumors and promote an improved event-free survival and overall survival.\n\nThis study will investigate the safety of treatment with a stem cell graft depleted of αβ-T cell and CD19+ B cells in combination with zoledronic acid in pediatric and young adult patients with select solid tumors, as well as whether this treatment improves survival rates in these patients.",[63,64,65,66,67,68,28,69,70,71,72,29,73,35],"Neuroblastoma","Rhabdomyosarcoma","Synovial Sarcoma","Peripheral Nerve Sheath Tumors","Clear Cell Sarcoma","Alveolar Soft Part Sarcoma","Chordoma","Rhabdoid Tumor","Epithelioid Sarcoma","Myoepithelial Tumor","Ewing Sarcoma",[75,76,77],"pediatric solid tumors","stem cell transplantation","graft manipulation","2026-06-25",{"date":80,"type":40},"2026-06-30",{"date":82,"type":40},"2026-02-11",{"date":84,"type":21},"2030-02",{"name":86,"class":87},"University of Florida","OTHER",1,{"id":90,"slug":91,"hasResults":11,"nctId":92,"briefTitle":93,"officialTitle":94,"acronym":95,"eligibilityCriteria":96,"healthyVolunteers":11,"sex":16,"minAge":97,"maxAge":98,"enrollmentInfo":99,"targetDuration":4,"studyType":22,"phases":101,"briefSummary":102,"conditions":103,"keywords":104,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":107,"lastUpdatePostDateStruct":108,"startDateStruct":110,"completionDateStruct":112,"leadSponsor":114,"locationsCount":116},"100606238","phase-1-selective-antigen-specific-t-cells-and-car-t-cells-in-subjects-with-relapsedrefractory-embryonal-tumors-sabre-100606238","NCT07172958","Selective Antigen Specific T Cells and CAR T Cells in Subjects With Relapsed\u002FRefractory Embryonal Tumors (SABRE)","Selective Antigen Specific dTβRII-expressing T Cells and B7-H3 CAR T Cells in Subjects With Relapsed\u002FRefractory Embryonal Tumors (SABRE)","SABRE","Inclusion Criteria:\n\nRecipient Inclusion Criteria for Procurement:\n\n* Diagnosis of relapsed\u002Frefractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, or Wilms tumor\n* Refractory disease, residual detectable disease or relapsed disease following available standard of care therapies with known clinical benefit for their specific tumor type, or unable to receive such therapies due to unacceptable toxicity or contraindication\n* Measurable or evaluable disease by imaging, as determined following most recent therapy\n* Age ≥ 1 year and \\\u003C 24 years\n* Weight \\> 10 kg\n* No systemic steroid exposure within 1 week of procurement\n* Karnofsky\u002FLansky score of ≥ 60 (See Appendix 3)\n* Participants of childbearing potential or capable of fathering a child must agree to use effective contraceptive measure\u002Fs (as described in Appendix 5) during study protocol participation through 6 months following the administration of the CAR-TA T cells\n* ANC \\> 500\u002FµL\n* ALC \\> 1000\u002FµL\n* Platelet count \\> 50,000\u002FuL (level can be achieved with transfusion)\n* Bilirubin ≤ 2.5 mg\u002FdL\n* Aspartate aminotransferase (AST)\u002FAlanine transaminase (ALT) ≤ 5x the upper limit of normal for age\n* Serum creatinine Maximum serum creatinine (mg\u002FdL) Age Male Female\n\n  1. to \\\u003C 2 years 0.6 0.6\n  2. to \\\u003C 6 years 0.8 0.8\n\n  6 to \\\u003C 10 years 1 1 10 to \\\u003C 13 years 1.2 1.2 13 to \\\u003C 16 years 1.5 1.2\n\n  ≥ 16 years 1.7 1.4 OR Creatinine clearance or glomerular filtration rate (GFR) ≥ 70 mL\u002Fmin\u002F1.73 m for patients with levels above\n* For FOCBP: Negative pregnancy test\n* Pulse oximetry of \\> 90% on room air\n* Adequate cardiac function defined as: o Shortening fraction of ≥ 27% by echocardiogram, or o Ejection fraction of \\> 50% by echocardiogram or radionuclide angiogram (i.e., MUGA).\n* No acute neurological toxicity \\> grade 1 (with the exception of peripheral sensory neuropathy or controlled seizure disorder on anti-epileptics).\n* The following time frames must have elapsed between prior therapy completion and apheresis cell collection:\n\n  * Myelosuppressive chemotherapy\u002Fimmunomodulatory medications: At least 3 weeks, or 6 weeks if prior nitrosourea.\n  * Hematopoietic growth factors: At least 7 days since the completion of therapy with a growth factor. At least 14 days after receiving pegfilgrastim.\n  * Biological agent, tyrosine kinase inhibitor, targeted agent, metronomic chemotherapy: At least 7 days since the completion of therapy with a biologic agent, tyrosine kinase inhibitor, targeted agent, or metronomic non-myelosuppressive regimen.\n  * Monoclonal antibodies and checkpoint inhibitors: At least 3 weeks or 5 half-lives (whichever is shorter) since the last dose of a monoclonal antibody or checkpoint inhibitor.\n  * Radiotherapy (XRT): At least 3 weeks since XRT, and at least 6 weeks if radiation involved the CNS or lung fields. Exception: There is no time restriction for palliative radiation with minimal bone marrow involvement and the patient has measurable\u002Fevaluable disease outside the radiation port or the site of radiation has documented progression.\n  * Autologous stem cell transplant\u002Finfusion: At least 6 weeks from their infusion after an autologous stem cell infusion following myeloablative therapy. Patients who received an autologous stem cell infusion following non-myeloablative therapy do not have a wash-out period; they are eligible once they meet all other eligibility requirements, including recovery from acute side effects.\n  * Investigational agent: at least 28 days since receiving an investigational agent.\n* Patient or parent\u002Fguardian capable of providing informed consent.\n\nRecipient Inclusion Criteria for CAR-TA T cell product Infusion:\n\n* No systemic steroid exposure within 1 week prior to protocol therapy initiation\n* Karnofsky\u002FLansky score of ≥ 60 (See Appendix 3)\n* ANC \\> 750\u002FuL\n* Platelet count \\> 75,000\u002FuL\n* Bilirubin ≤ 2.5 mg\u002FdL\n* AST\u002FALT ≤ 5x the upper limit of normal for age\n* Serum creatinine Maximum serum creatinine (mg\u002FdL) Age Male Female\n\n  1 to \\\u003C 2 years 0.6 0.6 2 to \\\u003C 6 years 0.8 0.8 6 to \\\u003C 10 years 1 1 10 to \\\u003C 13 years 1.2 1.2 13 to \\\u003C 16 years 1.5 1.2\n\n  ≥ 16 years 1.7 1.4 OR Creatinine clearance or glomerular filtration rate (GFR) ≥ 70 mL\u002Fmin\u002F1.73 m for patients with levels above\n* For FOCBP: Negative pregnancy test\n* Participants of childbearing potential or capable of fathering a child must agree to use effective contraceptive measure\u002Fs (as described in Appendix 5) through 6 months following the administration of the CAR-TA T cells\n* Adequate respiratory function defined as oxygen saturation 90% or higher on room air\n* No acute neurological toxicity \\> grade 1 (with the exception of peripheral sensory neuropathy or controlled seizure disorder on anti-epileptics).\n* Adequate cardiac function defined as:\n\n  * Shortening fraction of ≥ 27% by echocardiogram, or\n  * Ejection fraction of \\> 50% by echocardiogram or radionuclide angiogram\n* The following time frames must have elapsed between completion of prior therapy and the initiation of SABRE protocol therapy:\n\n  * Myelosuppressive chemotherapy: At least 2 weeks from last dose of chemotherapy.\n  * Hematopoietic growth factors: At least 7 days since the completion of therapy with a growth factor. At least 14 days after receiving pegfilgrastim.\n  * Biological agent, tyrosine kinase inhibitor, targeted agent, metronomic chemotherapy: At least 7 days since the completion of therapy with a biologic agent, tyrosine kinase inhibitor, targeted agent, or metronomic non-myelosuppressive regimen.\n  * Monoclonal antibodies and checkpoint inhibitors: At least 3 weeks or 5 half-lives (whichever is shorter) since the last dose of a monoclonal antibody or checkpoint inhibitor.\n  * Radiotherapy (XRT): At least 3 weeks since XRT, and at least 6 weeks if radiation involved CNS or lung fields. Exception: There is no time restriction for palliative radiation with minimal bone marrow involvement and the patient has measurable\u002Fevaluable disease outside the radiation port or the site of radiation has documented progression.\n  * Investigational agent: At least 28 days since receiving an investigational agent.\n* Patient or parent\u002Fguardian capable of providing informed consent.\n\nExclusion Criteria:\n\nRecipient Procurement Exclusion Criteria:\n\n* Patients with known CNS disease.\n* Patients with uncontrolled infection\u002Fs or known HIV infection\n* Pregnant or lactating females.\n* Patients who have undergone previous allogeneic stem cell transplant.\n\nRecipient Exclusion Criteria for CAR-TA T cell product Infusions:\n\n* Patients with uncontrolled infections or known HIV infection.\n* Pregnant or lactating females\n* Whole lung\u002Fmediastinal radiation within 12 weeks\n* Clinically significant systemic illness or medical condition likely to interfere with assessment of safety or efficacy","1 Year","23 Years",{"count":100,"type":21},18,[24],"This is a phase I dose-escalation study to determine the safety and feasibility of autologous CAR-TA T cells (B7-H3 CAR+ T cells administered with DNR-PRAME Tumor Antigen-specific T cells) following lymphodepleting chemotherapy in participants with relapsed\u002Frefractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor.\n\nPatients will be enrolled to one of three planned dose levels with B7-H3 CAR T cell dose determined based on the percentage of B7-H3 transduced cells (B7-H3+ population of cells), and dTBRII-transduced PRAME TA-specific T cell dose based on the total cell population. Both doses will be based on the recipient's body weight.\n\nThe safety of the CAR-TA T cell product will be evaluated and the maximum tolerated dose (MTD) will be determined. The safety endpoint will be assessed by monitoring for dose limiting toxicities for 28 days following CAR-TA T cell administration.",[64,73,63,35],[105,106],"CAR T Therapy for Embryonal tumors","T cell Therapy for Embryonal tumors","2026-05-20",{"date":109,"type":40},"2026-05-22",{"date":111,"type":40},"2026-01-27",{"date":113,"type":21},"2038-12",{"name":115,"class":87},"Children's National Research Institute",2,{"id":118,"slug":119,"hasResults":11,"nctId":120,"briefTitle":121,"officialTitle":121,"acronym":4,"eligibilityCriteria":122,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":123,"enrollmentInfo":124,"targetDuration":4,"studyType":22,"phases":126,"briefSummary":127,"conditions":128,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":137,"lastUpdatePostDateStruct":138,"startDateStruct":140,"completionDateStruct":142,"leadSponsor":144,"locationsCount":88},"100431379","phase-1-b7-h3-specific-chimeric-antigen-receptor-autologous-t-cell-therapy-for-pediatric-patients-with-solid-tumors-3car-100431379","NCT04897321","B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cell Therapy for Pediatric Patients With Solid Tumors (3CAR)","Inclusion Criteria:\n\nProcurement and T-cell production eligibility\\*\n\n\\*a previously collected, autologous leukapheresis product can be used for T-cell production\n\n* Age ≤21 years old\n* B7-H3+ solid tumor with measurable disease; B7-H3 expression will be evaluated by standard immunohistochemistry (IHC) using a previously obtained biopsy; a tumor is considered B7-H3 positive with an H-score ≥100\n* Estimated life expectancy of \\>12 weeks\n* Karnofsky or Lansky (age-dependent) performance score ≥50\n* For females of child bearing age:\n* Not pregnant with negative serum pregnancy test within 7 days prior to enrollment\n* Not lactating with intent to breastfeed\n* Meets eligibility criteria to undergo autologous apheresis, or have previously undergone autologous apheresis\n\nExclusion Criteria:\n\n* Known primary immunodeficiency\n* Known HIV positivity\n* Severe intercurrent bacterial, viral or fungal infection (e.g. active hepatitis B or C infection or adenovirus infection)\n* History of hypersensitivity reactions to murine protein-containing products\n* Rapidly progressive disease (in the opinion of the study PIs)\n\nInclusion criteria\n\nTreatment eligibility\n\n* Age ≤21 years old\n* B7-H3+ solid tumor with measurable disease\n* Evidence of relapsed or refractory disease after standard first-line therapy\n* Estimated life expectancy of \\>8 weeks\n* Karnofsky or Lansky (age-dependent) performance score≥50\n* Echocardiogram with a ventricular ejection fraction\n* \\>40%; or shortening fraction ≥25%\n* Adequate renal function defined as creatinine clearance or radioisotope GFR 50 ml\u002Fmin\u002F1.73m2 (GFR 40 ml\u002Fmin\u002F1.73m2 if \\\u003C 2 years of age)\n* Adequate pulmonary function defined as pulse oximetry ≥92% on room air or forced vital capacity (FVC) ≥50% of predicted value\n* Total Bilirubin ≤3 times the upper limit of normal for age, except in subjects with Gilbert's syndrome\n* Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤5 times the upper limit of normal for age\n* Hemoglobin≥ 7g\u002FdL (can be transfused)\n* Platelet count \\>50,000\u002FuL (can be transfused)\n* Absolute neutrophil count (ANC) ≥ 1000\u002FuL\n* Has recovered from all NCI CTAE grade III-IV, non-hematologic acute toxicities from prior therapy\n* For females of child bearing age:\n* Not pregnant with negative serum pregnancy test within 7 days prior to enrollment\n* Not lactating with intent to breastfeed\n* If sexually active, agreement to use birth control until 3 months after T-cell infusion. Male partners should use a condom.\n* Available autologous transduced T-cell product that has met GMP release criteria\n* Agreement to participate in long-term follow-up protocol for patients, who have received genetically modified cell products\n\nExclusion criteria\n\n* Known primary immunodeficiency\n* History of HIV infection\n* Severe, uncontrolled intercurrent bacterial, viral or fungal infection\n* History of hypersensitivity reactions to murine protein-containing products\n* Receiving systemic steroid therapy exceeding the equivalent of 0.5 mg\u002Fkg\u002Fday of methylprednisolone, in the 7 days prior to B7-H3-CAR T-cell infusion\n* Receiving systemic therapy in the 14 days prior to CAR T-cell infusion, which will interfere with the activity of the B7-H3-CAR product (in the opinion of the study PIs).\n* Rapidly progressing disease (in the opinion of the study PIs)","21 Years",{"count":125,"type":21},48,[24],"3CAR is being done to investigate an immunotherapy for patients with solid tumors. It is a Phase I clinical trial evaluating the use of autologous T cells genetically engineered to express B7-H3-CARs for patients ≤ 21 years old, with relapsed\u002Frefractory B7-H3+ solid tumors. This study will evaluate the safety and maximum tolerated dose of B7-H3-CAR T cells.The purpose of this study is to find the maximum (highest) dose of B7-H3-CAR T cells that are safe to give to patients with B7-H3-positive solid tumors.\n\nPrimary objective\n\nTo determine the safety of one intravenous infusion of autologous, B7-H3-CAR T cells in patients (≤ 21 years) with recurrent\u002Frefractory B7-H3+ solid tumors after lymphodepleting chemotherapy\n\nSecondary objective\n\nTo evaluate the antitumor activity of B7-H3-CAR T cells\n\nExploratory objectives\n\n* To evaluate the tumor environment after treatment with B7-H3-CAR T cells\n* To assess the immunophenotype, clonal structure and endogenous repertoire of B7-H3-CAR T cells and unmodified T cells\n* To characterize the cytokine profile in the peripheral blood after treatment with B7-H3-CAR T cells",[129,29,64,63,73,35,130,28,131,70,67,132,133,134,135,136],"Pediatric Solid Tumor","Adrenocortical Cancer","Germ Cell Cancer","Hepatoblastoma","Melanoma","Carcinoma","Malignant Peripheral Nerve Sheath Tumors","Soft Tissue Sarcoma","2026-05-18",{"date":139,"type":40},"2026-05-19",{"date":141,"type":40},"2022-07-06",{"date":143,"type":21},"2028-03-01",{"name":145,"class":87},"St. Jude Children's Research Hospital",{"id":147,"slug":148,"hasResults":11,"nctId":149,"briefTitle":150,"officialTitle":151,"acronym":152,"eligibilityCriteria":153,"healthyVolunteers":11,"sex":16,"minAge":154,"maxAge":155,"enrollmentInfo":156,"targetDuration":4,"studyType":22,"phases":158,"briefSummary":159,"conditions":160,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":168,"lastUpdatePostDateStruct":169,"startDateStruct":171,"completionDateStruct":173,"leadSponsor":175,"locationsCount":88},"100641039","phase-1-phase-i-study-of-becotatug-vedotin-for-safety-and-efficacy-in-egfr-positive-pediatric-relapsedrefractory-or-metastatic-solid-tumors-100641039","NCT07584499","Phase I Study of Becotatug Vedotin for Safety and Efficacy in EGFR-Positive Pediatric Relapsed\u002FRefractory or Metastatic Solid Tumors","A Phase I Clinical Study to Explore the Safety and Efficacy of Becotatug Vedotin in Pediatric Patients With EGFR-Positive Relapsed\u002FRefractory or Metastatic Solid Tumors","MRG003","Inclusion Criteria:\n\n* All participants must meet all of the following criteria to be eligible for enrollment:\n\nInformed Consent: The patient (and\u002For legal guardian, as age-appropriate) fully understands the study, voluntarily agrees to participate, and signs a written informed consent form (ICF). A separate biomarker consent form is required for EGFR testing prior to screening.\n\nAge: 2 to 18 years old at the time of consent. Life Expectancy: Estimated overall survival of at least 3 months.\n\nHistologically Confirmed Disease: Pathologically confirmed relapsed\u002Frefractory or metastatic EGFR-positive solid tumor, belonging to one of the following subtypes:\n\nHead and neck squamous cell carcinoma, nasopharyngeal carcinoma, or lymphoepithelial carcinoma that progressed during or after at least one line of platinum-based chemotherapy and PD-1\u002FPD-L1 inhibitor therapy Rhabdomyosarcoma Neuroblastoma Medulloblastoma Wilms tumor Atypical teratoid\u002Frhabdoid tumors (AT\u002FRTs) Diffuse intrinsic pontine gliomas (DIPGs) Other EGFR-positive solid tumor subtypes deemed eligible by the investigator Measurable Disease: At least one measurable tumor lesion by computed tomography (CT) or magnetic resonance imaging (MRI) per RECIST v1.1 criteria (longest diameter ≥10 mm; pathological lymph node short axis ≥15 mm).\n\nPerformance Status: Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2.\n\nAdequate Bone Marrow Function:\n\nAbsolute neutrophil count (ANC) ≥1.5 × 10⁹\u002FL Platelet count ≥75 × 10⁹\u002FL Hemoglobin ≥80 g\u002FL Exception for patients with bone marrow involvement: ANC ≥1.0 × 10⁹\u002FL, platelets ≥50 × 10⁹\u002FL, hemoglobin ≥75 g\u002FL\n\nAdequate Hepatic and Renal Function:\n\nSerum creatinine ≤1.5 × upper limit of normal (ULN) Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 × ULN Total bilirubin ≤1.5 × ULN Exception for patients with liver involvement: AST\u002FALT ≤5 × ULN, total bilirubin ≤3 × ULN\n\nExclusion Criteria:\n\n* Participants will be excluded from the study if they meet any of the following criteria:\n\nHypersensitivity: Known hypersensitivity to any component of Becotatug Vedotin (MRG003) or its excipients.\n\nSymptomatic CNS Metastases: Presence of symptomatic central nervous system (CNS) metastases.\n\nPrior Malignancies: History of other primary malignant tumors, except for:\n\nLocally excised basal cell or squamous cell carcinoma of the skin Cervical carcinoma in situ Any prior malignancy that has been in complete remission for ≥3 years without treatment Note: Melanoma (any stage) is explicitly excluded\n\nSignificant Liver Disease: Clinically significant liver disease, including:\n\nPositive hepatitis C virus (HCV) antibody Chronic active hepatitis B (HBV DNA \\>20,000 IU\u002FmL) HIV Infection: Known human immunodeficiency virus (HIV) infection. Severe Ocular Abnormalities: History of severe ophthalmologic conditions, such as severe dry eye syndrome or exposure keratitis.\n\nUncontrolled Systemic Diseases: Severe or uncontrolled medical conditions, including:\n\nInterstitial lung disease or pneumonitis Active autoimmune diseases requiring systemic immunosuppressive therapy\n\nCardiac Disease: Clinically significant cardiac dysfunction or cardiac disease, including:\n\nCongestive heart failure (New York Heart Association Class ≥II) Uncontrolled arrhythmias QTc interval prolongation \\>450 ms (males) or \\>470 ms (females) Recent Antitumor Therapy: Received any systemic antitumor therapy (chemotherapy, biological therapy, immunotherapy, targeted therapy) within 3 weeks prior to the first dose of study drug, and have not recovered to CTCAE v4.03 Grade ≤1 (except alopecia).\n\nRecent Major Surgery: Underwent major surgical procedure within 3 weeks prior to the first dose of study drug.\n\nPlanned Surgery: Planned surgical procedure during the study period, or any surgery deemed necessary by the investigator.\n\nPrior EGFR Therapy Toxicity: History of severe skin toxicity caused by prior EGFR-targeted therapy, or chronic skin disease requiring ongoing oral or intravenous treatment.\n\nOther Significant Risks: Any other concurrent medical condition that, in the investigator's judgment, would increase the risk of toxicity or compromise the patient's ability to complete the study.","2 Years","18 Years",{"count":157,"type":21},51,[24],"There is a significant unmet medical need for effective therapies for pediatric relapsed\u002Frefractory solid tumors. EGFR is highly and stably expressed in multiple pediatric solid tumor subtypes, and adult Phase I data of Becotatug Vedotin demonstrated a manageable safety profile and promising antitumor activity in EGFR-positive advanced solid tumors.This is a multicenter, non-randomized, single-arm, open-label Phase I clinical trial sponsored by Sun Yat-sen University Cancer Center (SYSUCC). The trial evaluates the safety, tolerability, pharmacokinetics (PK), immunogenicity, and preliminary efficacy of Becotatug Vedotin-an EGFR-targeted antibody-drug conjugate (ADC)-in pediatric patients with EGFR-positive relapsed\u002Frefractory or metastatic solid tumors.",[161,162,163,64,63,164,35,165,166,167],"Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent\u002FMetastatic (R\u002FM)","Nasopharyngeal Carcinoma","Lymphoepithelial Carcinoma","Medulloblastoma","Atypical Teratoid\u002FRhabdoid Tumors","Diffuse Intrinsic Pontine Gliomas","Other EGFR-positive Pediatric Solid Tumors Deemed Eligible by the Investigator","2026-05-07",{"date":170,"type":40},"2026-05-13",{"date":172,"type":21},"2026-05-30",{"date":174,"type":21},"2028-05-30",{"name":176,"class":87},"Sun Yat-sen University",{"id":178,"slug":179,"hasResults":11,"nctId":180,"briefTitle":181,"officialTitle":182,"acronym":4,"eligibilityCriteria":183,"healthyVolunteers":11,"sex":16,"minAge":184,"maxAge":4,"enrollmentInfo":185,"targetDuration":4,"studyType":22,"phases":187,"briefSummary":188,"conditions":189,"keywords":194,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":199,"lastUpdatePostDateStruct":200,"startDateStruct":202,"completionDateStruct":204,"leadSponsor":206,"locationsCount":5},"100514950","phase-2-a-study-of-selinexor-in-people-with-wilms-tumors-and-other-solid-tumors-100514950","NCT05985161","A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors","A Multi-Center Phase II Study of Selinexor in Treating Recurrent or Refractory Wilms Tumor and Other Pediatric Solid Tumors","Inclusion Criteria:\n\n* Age:\n\n  1. Age ≥ 6 at the time of informed consent\n  2. Age ≥ 2 years to \\\u003C 6 years at time of informed consent (Refer to Section 4.3): If PK cohort 1 is open, patients in this age range may enroll onto this cohort. If PK cohort 1 has been completed and deemed sufficient to proceed, then such patients may enroll onto the phase 2.\n  3. Age ≥ 12 months to \\\u003C 2 years at time of informed consent (Refer to Section 4.3):\n\nIf PK cohort 2 is open, patients in this age range may enroll onto this cohort. If PK cohort 2 has been completed and deemed sufficient to proceed, then such patients may enroll onto the phase 2.\n\n* Consent: All patients and\u002For their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.\n* Performance: Karnofsky ≥ 60% for patients \\> 16 years of age and Lansky ≥ 60 for patients ≤ 16 years of age.\n* Diagnosis: Patients must enroll into one of the following cohorts:\n\n  1. Cohort A: Any type of Wilms tumor or nephroblastoma is eligible for this study provided they meet at least one of these criteria: (1) in their second or greater relapse, (2) refractory or in their first relapse with high risk histology (i.e., any anaplastic or blastemal-type after neoadjuvant chemotherapy), or (3) refractory or in first relapse without high risk histology but after having received chemotherapies other than the initial 4 agents used as current standard of care in the up-front setting for non-high risk cases - specifically vincristine, dactinomycin, doxorubicin, and irinotecan (i.e., any patient who relapses following an initial regimen more intense than EE4A, DD4A, VAD, AVD, or VIVA; for example, those including cyclophosphamide\u002Fetoposide - such as Regimen I, M, or MVI - or those additionally including carboplatin - such as Regimens UH-1, UH-2, or UH-3).\n  2. Cohort B: Any Rhabdoid tumor is eligible for this cohort. This includes, but is not limited to, related subtypes of rhabdoid tumors such as atypical teratoid rhabdoid tumors (ATRT), malignant rhabdoid tumors of the kidney (MRTK), malignant rhabdoid tumors of the soft tissue and liver, small cell undifferentiated hepatoblastomas (SCUH), and small-cell carcinoma of the ovary of hypercalcemic type (SCCOHT). Patients must have failed to respond to at least 1 line of systemic therapy prior to enrollment.\n  3. Cohort C: Patients with progressive, relapsed, unresectable or metastatic MPNST, are eligible for this cohort. Patients must have failed to respond to at least 1 line of systemic therapy prior to enrollment.\n  4. Cohort D: Patients must not qualify for Cohorts A, B, or C but have a solid tumor (no hematologic malignancies including lymphoma) for which there is specific evidence that this particular patient's tumor may benefit from selinexor.\n\nPatients must have failed to respond to at least 1 line of systemic therapy prior to enrollment. Examples of evidence are listed below. All patients in this cohort require approval of study principal investigator and must provide documentation of specific supporting evidence. i. Tumor XPO1 Dependency: Defined as either Darwin OncoTarget demonstrating XPO1 as aberrantly activated or Darwin OncoTreat demonstrating context-specific tumor checkpoint inversion with Selinexor, both of which must be significant at a -log10 (Bonferroni corrected p-value) of 5 or greater. ii. Tumor XPO1 Activation: Defined as the detection of a gain of function mutation in XPO1, specifically E571K. Additionally, detection of elevated transcriptomic or proteomic expression of XPO1 in the tumor via RNAseq or IHC, respectively, would be considered sufficient for treatment.\n\niii. Preclinical Tumor Testing: Defined as testing of Selinexor on patient derived cell line, organoid, or xenograft models of the patient's tumor (or other related tumors) performed in a laboratory context and for which, in the investigator's opinion, demonstrates promising activity. Testing may include commercial testing as well as academic laboratory testing.\n\n* Cohort E: Patients must have a solid tumor with an activating genomic alteration (e.g. fusion or internal tandem duplication) involving BCOR. Specific examples of qualifying alterations including BCOR-ITD, BCOR-CCNB3, BCOR-MAML3 and ZC3H7B-BCOR; Other potentially qualifying BCOR alterations require approval of study principal investigator; note that loss of function alterations of BCOR would not qualify.\n* Disease Status: Patients on the phase II portion of the study must have measurable disease whereas patients on the PK cohorts can have either evaluable or measurable disease as measured by the revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (Version 1.1).\n\n  a. Primary Brain Tumors: Patients with primary brain tumors are eligible and must also have measurable disease for the phase II (as well as evaluable or measurable for the PK cohorts), but this can be defined as at least equal or greater than twice the slice thickness in two perpendicular diameters on MRI OR diffuse leptomeningeal disease OR clear MRI evidence of disease that may not be measurable in two perpendicular diameters OR positive CSF cytology alone.\n* Prior Therapy: Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and meet minimum washout durations (shown below) from prior therapy.\n\n  1. Anti-cancer agents not known to be myelosuppressive: ≥ 7 days\n  2. Anti-cancer and cytotoxic agents known to be myelosuppressive: ≥ 21 days\n  3. Immunotherapies (including antibodies, interleukins, interferons, etc.): ≥ 21 days\n  4. Adoptive cellular therapies (including modified T cells, vaccines, etc.): ≥ 42 days\n  5. Autologous stem cell infusion (boost, no conditioning): ≥ 21 days\n  6. Autologous stem cell transplantation (with conditioning): ≥ 42 days\n  7. Allogeneic bone marrow transplantation: ≥ 84 days\n  8. Focal external beam radiation (e.g., limited sites of disease): ≥ 14 days\n  9. Substantial external beam radiation (e.g. whole lung or abdomen): ≥ 42 days\n  10. Radiopharmaceutical therapy (e.g., radiolabeled antibody or MIBG): ≥ 42 days\n* Hepatic Function: Adequate function (within 14 days prior to C1D1), defined as:\n\n  1. Total bilirubin \\\u003C 1.5 × upper limit of normal (ULN) (except patients with Gilbert's syndrome, who must have a total bilirubin of \\\u003C3 × ULN)\n  2. Alanine aminotransferase (ALT) \\\u003C 3 × ULN\n  3. Serum albumin ≥ 2 g\u002FdL\n* Renal Function: Adequate function (within 14 days prior to C1D1) defined as a GFR\n\n  ≥ 50 ml\u002Fmin\u002F1.73 m2 determined via any of these methods:\n  1. Nuclear radioisotope\n  2. 24 hr urine creatinine clearance\n  3. Serum cystatin c\n  4. Serum creatinine using the Schwartz formula for estimating creatinine clearance (Schwartz et al. J Peds, 106:522, 1985)\n* Hematologic Function: Adequate function (within 14 days prior to C1D1), defined as:\n\n  1. Absolute neutrophil count (ANC) ≥ 1000\u002Fmm3\n  2. Platelet count ≥ 100,000\u002Fmm3\n  3. Note: patients may not receive platelet transfusions nor hematopoietic growth factor support, including granulocyte-colony stimulating factor (e.g. filgrastim) and platelet stimulators (e.g. romiplostim) for at least 7 days prior to demonstrating adequate hematologic function.\n\nExclusion Criteria:\n\n* Prior Therapy: Has received selinexor or another XPO1 inhibitor previously.\n* Infection: Patients who have an uncontrolled infection are not eligible. Patients on prophylactic antibiotics or with a controlled infection within 1 week prior to C1D1 are acceptable\n* Transplants: Patients who have received allogeneic bone marrow transplant are potentially eligible unless they are being actively treated for GvHD. Patients who have had a prior solid organ transplantation are not eligible.\n* Compliance: Patients who as a result of serious medical, psychiatric, and\u002For social situation(s), in the opinion of the investigator, may not be able to comply with supportive care, safety monitoring, or any other key requirements of the study protocols are not eligible.\n* Pregnancy and Breast-feeding: Pregnant or breast-feeding women will not be entered on this study because there is yet no available information regarding human fetal or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal.\n* Contraception: 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) for the duration of the study. Abstinence is an acceptable method of birth control.","12 Months",{"count":186,"type":21},45,[25],"The purpose of this study is to find out whether selinexor is an effective treatment for people who have a relapsed\u002Frefractory Wilms tumor, rhabdoid tumor, MPNST, BCOR-driven sarcoma, or another solid tumor that makes a higher than normal amount of XPO1 or has genetic changes that increase the activity of XP01.",[35,70,135,190,191,192,193],"MPNST","Nephroblastoma","XPO1 Gene Mutation","Solid Tumor",[35,70,135,190,191,195,196,197,198,193],"XPO1","Selinexor","Memorial Sloan Kettering Cancer Center","22-393","2026-05-04",{"date":201,"type":40},"2026-05-06",{"date":203,"type":40},"2023-08-01",{"date":205,"type":21},"2029-08-01",{"name":197,"class":87},{"id":208,"slug":209,"hasResults":11,"nctId":210,"briefTitle":211,"officialTitle":212,"acronym":4,"eligibilityCriteria":213,"healthyVolunteers":11,"sex":16,"minAge":184,"maxAge":214,"enrollmentInfo":215,"targetDuration":4,"studyType":22,"phases":217,"briefSummary":218,"conditions":219,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":238,"lastUpdatePostDateStruct":239,"startDateStruct":241,"completionDateStruct":243,"leadSponsor":245,"locationsCount":248},"100427831","phase-1-tegavivint-for-the-treatment-of-recurrent-or-refractory-solid-tumors-including-lymphomas-and-desmoid-tumors-100427831","NCT04851119","Tegavivint for the Treatment of Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors","A Phase 1\u002F2 Study of Tegavivint (NSC#826393) in Children, Adolescents, and Young Adults With Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors","Inclusion Criteria:\n\n* PART A: Patients must be \\>= 12 months and =\\\u003C 21 years of age at the time of study enrollment\n* PART B: Patients must be \\>= 12 months and =\\\u003C 30 years of age at the time of study enrollment\n* Patients with recurrent or refractory solid tumors including non-Hodgkin lymphoma and desmoid tumors are eligible. Patients must have had histologic verification of malignancy at original diagnosis or relapse\n* PART A: Patients with relapsed or refractory solid tumors, including patients with non-Hodgkin lymphoma and desmoid tumors\n* PART B: Patients with recurrent or refractory Ewing sarcoma, desmoid tumors, osteosarcoma, liver tumors (HCC and hepatoblastoma), Wilms tumor, and tumors with Wnt pathway aberrations. For the Wnt pathway aberrations cohort we will include the most common CTNNB1 mutations (S37F, S45F, T41A, S45P, S33C, S37C, D32Y, S33F, T41I, G34R, G34V, D32N, S33P, G34E, D32G) as well as any loss of function mutations in the APC, Axin2FBXW7, TCF7L2, and RNF43 genes or any gain-of-function mutations in the GSK3B, LRP6, and LGR5 genes. For patients without prior sequencing, immunohistochemistry (IHC), is required. IHC showing strong nuclear beta-catenin staining will be accepted for the following tumor types: colorectal carcinoma, melanoma, endometrial cancer, ovarian cancer, neuroblastoma, non-Hodgkin lymphoma, pancreatic ductal adenocarcinoma, and solid pseudopapillary tumor of the pancreas\n* PART A: Patients must have either measurable or evaluable disease. For desmoid tumors, the patient must have disease that the investigator deems unresectable or sufficiently morbid or potentially life-threatening that there is favorable risk\u002Fbenefit to the patient to participate in the trial\n* PART B: Patients must have measurable disease. For desmoid tumors, the patient must have measurable disease that the investigator deems unresectable or sufficiently morbid or potentially life-threatening that there is favorable risk\u002Fbenefit to the patient to participate in the trial\n* Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients \\> 16 years of age and Lansky for patients =\\\u003C 16 years of age\n* Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g., blood count criteria, the patient is considered to have recovered adequately.\n\n  * Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive\n\n    * Solid tumor patients: \\>= 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea)\n    * Non-Hodgkin lymphoma patients\n\n      * A waiting period prior to enrollment is not required for patients receiving standard maintenance chemotherapy (i.e., corticosteroid, vincristine, thioguanine \\[6MP\\], and\u002For methotrexate)\n      * \\>= 14 days must have elapsed after the completion of other cytotoxic therapy, with the exception of hydroxyurea, for patients not receiving standard maintenance therapy\n      * NOTE: Cytoreduction with hydroxyurea must be discontinued \\>= 24 hours prior to the start of protocol therapy\n  * Anti-cancer agents not known to be myelosuppressive (e.g., not associated with reduced platelet or absolute neutrophil counts \\[ANC\\]): \\>= 7 days after the last dose of agent\n  * Antibodies: \\>= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =\\\u003C 1\n  * Corticosteroids: If used to modify immune adverse events related to prior therapy, \\>= 14 days must have elapsed since last dose of corticosteroid\n  * Hematopoietic growth factors: \\>= 14 days after the last dose of a long-acting growth factor (e.g., pegfilgrastim) or 7 days for short acting growth factor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur\n  * Interleukins, interferons and cytokines (other than hematopoietic growth factors): \\>= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)\n  * Stem cell Infusions (with or without total-body irradiation \\[TBI\\]):\n\n    * Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: \\>= 84 days after infusion and no evidence of graft versus host disease (GVHD)\n    * Autologous stem cell infusion including boost infusion: \\>= 42 days.\n  * Cellular therapy: \\>= 42 days after the completion of any type of cellular therapy (e.g., modified T cells, natural killer \\[NK\\] cells, dendritic cells, etc.).\n  * External beam radiation therapy (XRT)\u002Fexternal beam irradiation including protons: \\>= 14 days after local XRT; \\>= 150 days after TBI, craniospinal XRT or if radiation to \\>= 50% of the pelvis; \\>= 42 days if other substantial bone marrow (BM) radiation\n  * Radiopharmaceutical therapy (e.g., radiolabeled antibody, iobenguane I-131 \\[131I MIBG\\]): \\>= 42 days after systemically administered radiopharmaceutical therapy\n  * Patients must not have received prior exposure to tegavivint\n* PATIENTS WITH SOLID TUMORS WITHOUT KNOWN BONE MARROW INVOLVEMENT: Peripheral absolute neutrophil count (ANC) \\>= 1000\u002FuL (within 7 days prior to enrollment)\n* PATIENTS WITH SOLID TUMORS WITHOUT KNOWN BONE MARROW INVOLVEMENT: Platelet count \\>= 100,000\u002FuL (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) (within 7 days prior to enrollment)\n* PATIENTS WITH SOLID TUMORS WITHOUT KNOWN BONE MARROW INVOLVEMENT: Hemoglobin \\>= 8.0 g\u002FdL at baseline (may receive red blood cell \\[RBC\\] transfusions) (within 7 days prior to enrollment)\n* Patients with known bone marrow metastatic disease will be eligible for study provided they meet blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions). These patients will not be evaluable for hematologic toxicity. At least 5 of every cohort of 6 patients must be evaluable for hematologic toxicity for the dose-escalation part of the study. If dose-limiting hematologic toxicity is observed, all subsequent patients enrolled on Part A must be evaluable for hematologic toxicity\n* Creatinine clearance or radioisotope glomerular filtration rate (GFR) \\>= 70 mL\u002Fmin\u002F1.73 m\\^2 or a creatinine based on age\u002Fgender as follows (within 7 days prior to enrollment):\n\n  * Age; maximum serum creatinine\n  * Age 1 to \\\u003C 2 years; 0.6 mg\u002FdL (male); 0.6 mg\u002FdL (female)\n  * Age 2 to \\\u003C 6 years; 0.8 mg\u002FdL (male); 0.8 mg\u002FdL (female)\n  * Age 6 to \\\u003C 10 years; 1 mg\u002FdL (male); 1 mg\u002FdL (female)\n  * Age 10 to \\\u003C 13 years; 1.2 mg\u002FdL (male); 1.2 mg\u002FdL (female)\n  * Age 13 to \\\u003C 16 years; 1.5 mg\u002FdL (male); 1.4 mg\u002FdL (female)\n  * Age \\>= 16 years; 1.7 mg\u002FdL (male); 1.4 mg\u002FdL (female)\n* PATIENTS WITH SOLID TUMORS: Bilirubin (sum of conjugated + unconjugated or total) =\\\u003C 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment)\n* PATIENTS WITH SOLID TUMORS: Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase \\[ALT\\]) =\\\u003C 135 U\u002FL. For the purpose of this study, the ULN for SGPT is 45 U\u002FL (within 7 days prior to enrollment)\n* PATIENTS WITH SOLID TUMORS: Albumin \\>= 2 g\u002FdL (within 7 days prior to enrollment)\n\nExclusion Criteria:\n\n* Pregnant or breast-feeding women will not be entered on this study because there is yet no available information regarding human fetal or teratogenic toxicities. 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) for the duration of the study. Abstinence is an acceptable method of birth control\n* Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. If used to modify immune adverse events related to prior therapy, \\>= 14 days must have elapsed since last dose of corticosteroid\n* Patients who are currently receiving another investigational drug are not eligible\n* Patients who are currently receiving other anti-cancer agents are not eligible\n* Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial\n* Patients who are currently receiving drugs that are strong inducers or inhibitors of CYP3A4 are not eligible. Strong inducers or inhibitors of CYP3A4 should be avoided from 14 days prior to the 1st dose of tegavivint to the end of the study\n* Patients who have received bisphosphonates within 4 weeks prior to study enrollment will be excluded\n* Patients who have received denosumab within 180 days prior to study enrollment will be excluded\n* Patients with primary brain tumors are ineligible\n* Patients with known central nervous system (CNS) metastasis, except for craniopharyngeal tumors, will be excluded\n* Patients with a known metabolic bone disease (ex: hyperparathyroidism, Paget's disease, osteomalacia) are not eligible\n* Patients with a disorder associated with abnormal bone metabolism will be excluded\n* Patients with grade \\>= 2 hypocalcemia that is not corrected with oral calcium supplementation will be excluded\n* Patients with vitamin D \\\u003C 20 ng\u002FmL will require supplementation, or will otherwise be excluded. Patients must agree to take vitamin D +\u002F- calcium supplements (if necessary) according to institutional or published guidelines. Additional calcium supplementation is not required if adequate dietary intake can be ascertained\n* Patients with pre-existing grade 3 osteoporosis are excluded\n* Patients who have an uncontrolled infection are not eligible\n* Patients who have received a prior solid organ transplantation 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","30 Years",{"count":216,"type":21},147,[24,25],"This phase I\u002FII trial evaluates the highest safe dose, side effects, and possible benefits of tegavivint in treating patients with solid tumors that has come back (recurrent) or does not respond to treatment (refractory). Tegavivint interferes with the binding of beta-catenin to TBL1, which may help stop the growth of tumor cells by blocking the signals passed from one molecule to another inside a cell that tell a cell to grow.",[220,221,133,63,222,223,224,225,226,227,228,229,32,230,231,232,233,234,235,236,237,35],"Colorectal Carcinoma","Endometrial Carcinoma","Ovarian Carcinoma","Pancreatic Ductal Adenocarcinoma","Recurrent Desmoid Fibromatosis","Recurrent Ewing Sarcoma","Recurrent Hepatoblastoma","Recurrent Hepatocellular Carcinoma","Recurrent Malignant Solid Neoplasm","Recurrent Non-Hodgkin Lymphoma","Refractory Desmoid Fibromatosis","Refractory Ewing Sarcoma","Refractory Hepatoblastoma","Refractory Hepatocellular Carcinoma","Refractory Malignant Solid Neoplasm","Refractory Non-Hodgkin Lymphoma","Refractory Osteosarcoma","Solid Pseudopapillary Neoplasm of the Pancreas","2026-05-01",{"date":240,"type":40},"2026-05-05",{"date":242,"type":40},"2021-11-08",{"date":244,"type":21},"2028-06-30",{"name":246,"class":247},"Children's Oncology Group","NETWORK",21,{"id":250,"slug":251,"hasResults":11,"nctId":252,"briefTitle":253,"officialTitle":254,"acronym":4,"eligibilityCriteria":255,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":256,"targetDuration":4,"studyType":22,"phases":258,"briefSummary":259,"conditions":260,"keywords":261,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":267,"lastUpdatePostDateStruct":268,"startDateStruct":270,"completionDateStruct":272,"leadSponsor":274,"locationsCount":88},"100436878","phase-2-treatment-of-newly-diagnosed-patients-with-wilms-tumor-requiring-abdominal-radiation-delivered-with-proton-beam-irradiation-100436878","NCT04968990","Treatment of Newly Diagnosed Patient's With Wilm's Tumor Requiring Abdominal Radiation Delivered With Proton Beam Irradiation","Multi-Center Trial Sponsored by St. Jude Children's Research Hospital for the Treatment of Newly Diagnosed Patient's With Wilm's Tumor Requiring Abdominal Radiation Delivered With Proton Beam Irradiation","Inclusion Criteria:\n\n* Patients are eligible to be enrolled on this trial at the time of initial (or presumptive) diagnosis of Wilms tumor, at the time of surgery or at the time of radiation treatment.\n* Patients that are determined to be stage I or II will be eligible for the surgical and biologic aspects of this trial but will otherwise be followed per institutional standards and be \"off therapy\" at the time of stage determination (followed only for survival).\n* Patients identified to have an anaplastic or other unfavorable tissue component (non-Wilms histology) to their tumor may have tissue assessed for the biology objective but will be removed from the therapeutic portion of the study at the time of identification of this pathologic finding.\n\nPerformance Level • The Karnofsky performance status must be ≥50 for patients \\>16 years of age and the Lansky performance status must be ≥ 50 for patients ≤ 16 years of age.\n\nPrior Therapy\n\n* Only prior therapy with the initial chemotherapy regimen defined in section 5.1 and Appendix III are allowed for patients that are treated with neoadjuvant chemotherapy. These patients may change to an alternate regime based on response or biologic features and noted in section 5.1. Prior biopsy or surgery is allowed.\n* Prior radiation therapy is not allowed if directed at the abdomen. Emergent RT to other sites of disease is acceptable.\n\nOrgan Function Requirements:\n\n* None - As per institutional standard of care. Diagnosis \u002F Stage\n* Presumed diagnosis of Wilms tumor (continued management on trial will depend on stage and histology)\n* Resected upfront stage III, IV or V favorable histology Wilms tumor. Stage III includes any one or multiples of the following:\n\n  * Lymph nodes within the abdomen or pelvis are involved by tumor. (Lymph node involvement in the thorax, or other extra-abdominal sites is a criterion for Stage IV)\n  * The tumor has penetrated through the peritoneal surface\n  * Tumor implants are found on the peritoneal surface\n  * Gross or microscopic tumor remains post-operatively (e.g., tumor cells are found at the margin of surgical resection on microscopic examination - in the post chemotherapy setting only specific types of viable tumor at the surgical margin are considered stage III AND require adjuvant radiation - see surgery \u002F pathology \u002F radiation section for additional details)\n  * The tumor is not completely resectable because of local infiltration into vital structures\n  * Tumor spillage occurring either before or during surgery\n  * A trans abdominal biopsy (regardless of type- tru-cut, open or fine needle aspiration) was performed prior to resection or chemotherapy. Retroperitoneal biopsies DO NOT require radiotherapeutic management like stage III\n  * Tumor is removed in greater than one piece (e.g. tumor cells are found in a separately excised adrenal gland; a tumor thrombus within the renal vein is removed separately from the nephrectomy specimen). Extension of the primary tumor within vena cava into thoracic vena cava and heart is considered Stage III, rather than Stage IV even though outside the abdomen.\n* Stage IV - Hematogenous metastases (lung, liver, bone, brain, etc.), or lymph node metastases outside the abdomino-pelvic region are present. (The presence of tumor within the adrenal gland is not interpreted as metastasis and staging depends on all other staging parameters present). Patients with lung involvement and local abdominal stage I or II are eligible to remain on trial for whole lung irradiation objective.\n* Stage V - Bilateral renal involvement by tumor is present at diagnosis. An attempt should be made to stage each side according to the above criteria on the basis of the extent of disease\n* Patients that have or will receive neoadjuvant chemotherapy for unresected unilateral or bilateral Wilms tumor without a biopsy are not assumed to be require adjuvant radiation on this study. That determination will be based on surgical findings and the approach to treatment detailed in the protocol.\n\nTiming\n\n* Patients undergoing upfront surgery must start RT, if indicated, within 28 days of definitive surgery with a goal of starting RT by day 21.\n* Patients receiving neoadjuvant chemotherapy will be enrolled either at diagnosis (if at St. Jude) or at the time of RT.\n\nOther Criteria\n\n• Female patients of childbearing potential (age ≥10 years old or post-menarche) must have a negative pregnancy test prior to enrollment.\n\nExclusion Criteria:\n\n* Inability or unwillingness to provide written informed consent\n* Prior radiation therapy to a site to be treated with proton \u002F photon radiation as part of this trial. Emergent radiation is allowed.",{"count":257,"type":21},260,[25],"Participants are being asked to take part in this clinical trial, a type of research study, because the participants have a Wilms Tumor cancer.\n\nPrimary Objectives\n\nTo determine whether delivery of proton beam radiation to a conformal reduced target volume in the flank allows normal flank growth compared to the contralateral untreated side and non-irradiated patients.\n\nSecondary Objectives\n\nTo deliver proton beam radiation to a conformal reduced target volume (partial kidney proton beam radiation therapy) in the affected kidney(s) for patients with Stage V (bilateral Wilms tumor) and specific involved surgical margins yielding no reduction in the high control rates seen with more traditional flank \u002F whole kidney fields.\n\nExploratory Objectives\n\n* Study the feasibility of sparing the residual kidney, spine and liver in patients requiring whole abdomen radiation therapy using either a proton beam treatment technique or intensity-modulated radiation therapy ( IMRT) photon based technique.\n* Study the feasibility of delivering whole lung radiation therapy with proton beams with the goals of sparing the developing breast tissue, heart structures, thyroid and liver.\n* Develop simultaneous xenografts and organoid models from the same starting material to study Wilms tumor biology and compare responses to chemotherapeutic agents.\n* Define the evolution of organ specific (kidney, liver, pancreas, etc.) abnormalities (laboratory studies) as an early marker of possible late end organ damage and their relationship to radiation.\n* Study and evaluate impact of proton therapy on the musculoskeletal system and physical performance and compare with photon therapy cases treated with classical treatment fields.\n* Assess CTC-AE and Pediatric Patient Reported Outcomes during radiation and in follow-up, correlating with disease, treatment and patient variables.\n* Correlate quantitative MRI values, including apparent diffusion coefficient (ADC) values, with histopathology findings post-surgery in children with (bilateral) Wilms.\n* Assess daily variations in proton range along each treatment beam using standard pre-treatment cone beam CT or on-treatment MR.",[35],[262,263,264,265,266],"Retroperitoneum","Retroperitoneal subsites","Kidney","Lymph node involvement","Abdomen","2026-04-22",{"date":269,"type":40},"2026-04-23",{"date":271,"type":40},"2021-08-19",{"date":273,"type":21},"2036-12",{"name":145,"class":87},{"id":276,"slug":277,"hasResults":11,"nctId":278,"briefTitle":279,"officialTitle":280,"acronym":4,"eligibilityCriteria":281,"healthyVolunteers":11,"sex":16,"minAge":282,"maxAge":214,"enrollmentInfo":283,"targetDuration":4,"studyType":22,"phases":284,"briefSummary":286,"conditions":287,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":289,"lastUpdatePostDateStruct":290,"startDateStruct":292,"completionDateStruct":294,"leadSponsor":296,"locationsCount":88},"100347967","early-phase-1-pencil-beam-scanning-in-patients-with-renal-tumors-100347967","NCT03810651","Pencil Beam Scanning in Patients With Renal Tumors","Pilot Study of Pencil Beam Scanning Proton Beam Radiation Therapy in Patients With Renal Tumors","Inclusion Criteria:\n\n* Less than 30 years of age\n* Diagnosis\n\n  a. Any patient with Wilms tumor or clear cell sarcoma of the kidney who would require radiation therapy as standard of care including\n  1. Any patient with favorable histology (FH), stage III disease\n  2. Any patient with focal or diffuse anaplasia\n  3. Any patient with CCSK\n* The patient is a candidate for external beam radiotherapy based on standard of care for treatment of Wilms tumor or CCSK\n\nExclusion Criteria:\n\n* Prior radiotherapy to the region of the study cancer\n* Chemotherapy administered for diagnosis of Wilms tumor or CCSK\n* Pregnant or breastfeeding","0 Years",{"count":48,"type":21},[285],"EARLY_PHASE1","This is a pilot study to assess acute toxicity in patients receiving flank irradiation using proton therapy for renal tumors.",[288,35],"Renal Tumor","2026-04-13",{"date":291,"type":40},"2026-04-15",{"date":293,"type":40},"2017-12-26",{"date":295,"type":21},"2028-10-30",{"name":297,"class":87},"Children's Hospital of Philadelphia",{"id":299,"slug":300,"hasResults":11,"nctId":301,"briefTitle":302,"officialTitle":303,"acronym":304,"eligibilityCriteria":305,"healthyVolunteers":11,"sex":16,"minAge":97,"maxAge":214,"enrollmentInfo":306,"targetDuration":4,"studyType":22,"phases":308,"briefSummary":309,"conditions":310,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":312,"lastUpdatePostDateStruct":313,"startDateStruct":314,"completionDateStruct":316,"leadSponsor":317,"locationsCount":88},"100386177","phase-1-vorinostat-in-combination-with-chemotherapy-in-relapsedrefractory-solid-tumors-and-cns-malignancies-100386177","NCT04308330","Vorinostat in Combination With Chemotherapy in Relapsed\u002FRefractory Solid Tumors and CNS Malignancies","A Phase I Study of Vorinostat in Combination With Vincristine, Irinotecan, and Temozolomide in Children, Adolescents, and Young Adults With Relapsed or Refractory Solid Tumors and CNS Malignancies","NYMC195","Inclusion Criteria:\n\n* Age: Patients must be less than or equal to 1 year and less than or equal to 30 years of age at initiation of protocol therapy.\n* Diagnosis: Patients must have a confirmed histologic diagnosis of a relapsed or refractory solid tumor or CNS malignancy.\n* Performance status: Patients over 16 years of age must have a Karnofsky score greater than or equal to 50. Children under 16 years of age must have a Lansky score greater than or equal to 50.\n* Prior therapy: Patients may have received prior therapy with vincristine, irinotecan, or temozolomide. They may not however have received therapy that included a treatment cassette of irinotecan and temozolomide in combination.\n\n  * Prior myelosuppressive therapy: Patients must have not received myelosuppressive therapy in 3 weeks or nitrosourea chemotherapy within 6 weeks of initiation of protocol therapy.\n  * Hematologic growth factor support: Patients may not have received G-CSF within the previous 3 days or peg-filgrastim within the past 7 days.\n  * Biologic anti-neoplastic therapy: At least 21 days or 5 half-lives (whichever is of longer duration) must have elapsed since the last administration of biologic antineoplastic therapy.\n  * Radiation therapy: ≥ 14 days since the last dose of local XRT; ≥ 6 months must have elapsed if prior TBI, craniospinal XRT or ≥ 50% radiation of pelvis; ≥ 6 wks must have elapsed if other substantial BM radiation.\n  * Autologous or allogeneic stem cell transplant: No active graft vs. host disease or need for immunosuppressive therapy. At least 3 months must have passed since neutrophil engraftment.\n* Organ function:\n\nBone marrow function:\n\n* Peripheral absolute neutrophil count (ANC) greater than or equal to 1000 cells\u002FmcL.\n* Platelet count greater than or equal to100,000\u002FmcL and no platelet transfusion within prior 7 days.\n* Hemoglobin greater than or equal to 8 gm\u002FdL\n* Patients with known bone marrow metastatic disease may enroll on the study if they have a peripheral ANC greater than or equal to 750 cells\u002FmcL. They will not be evaluable for hematologic toxicity.\n\n  \\- Adequate liver function:\n* Total bilirubin less than or equal to 1.5x upper limit of normal (ULN) for age.\n* SGPT (ALT) less than or equal to 5x ULN\n* Serum albumin greater than or equal to 2 gm\u002FdL\n\n  \\- Adequate renal function:\n* Creatinine clearance or glomerular filtration rate \\>70 ml\u002Fmin\u002F1.73 m2 or a serum creatinine based on age and gender as follows:\n\nAge Maximum serum creatinine concentration (mg\u002FdL) Male Female 1-\\\u003C2 years 0.6 0.6 2-\\\u003C6 years 0.8 0.8 6-\\\u003C10 years 1 1 10-\\\u003C13 years 1.2 1.2 13-\\\u003C16 years 1.5 1.4 greater than or equal to 16 years 1.7 1.4 The threshold creatinine values in this table were derived from the Schwartz formula to estimate glomerular filtration rates (Schwartz et al. J. Peds. 106; 522. 1985) using child length and stature data from the CDC.\n\n\\- Informed consent: All patients less than 18 years of age must sign a written informed consent. For patients \\\u003C18 years of age, a parent or guardian must sign a written informed consent, unless the patient is an emancipated minor. Childhood assent, when appropriate, should be obtained as well per institutional guidelines.\n\nExclusion Criteria:\n\n* Pregnancy or breast feeding: Women who are pregnant or breast feeding will not be entered on the protocol due to the risks of fetal and teratogenic adverse events with the therapeutic agents used in the protocol therapy.\n* Corticosteroid use: Patients with CNS tumors who have not been on a stable or decreasing dose of corticosteroids for the 7 days prior to the initiation of protocol therapy.\n* Antineoplastic therapy: Patients receiving any other antineoplastic therapy.\n* Medication allergy:\n\nAllergy or intolerance to any of the protocol agents: vincristine, irinotecan, temozolomide, or vorinostat.\n\nAllergy or intolerance to cephalosporins.\n\n* Infection: Patients who have any uncontrolled infection, positive blood culture within 48 hours prior to protocol entry, or diagnosed or receiving therapy for Clostridium difficile infection.\n* Patients may not have taken valproic acid or any other histone deacetylase inhibitor for at least 2 weeks prior to study enrollment.\n* Children with neurofibromastosis Type 1, if being used for treatment of a low grade glioma.",{"count":307,"type":21},30,[24],"Investigators are testing new experimental drug combinations such as the combination of vorinostat, vincristine, irinotecan, and temozolomide in the hopes of finding a drug that may be effective against tumors that have come back or that have not responded to standard therapy.\n\nThe goals of this study are:\n\n* To find the highest safe dose of vorinostat that can be given together with vincristine, irinotecan, and temozolomide without causing severe side effects;\n* To learn what kind of side effects this four drug combination can cause;\n* To learn about the effects of vorinostat and the combination of vorinostat, vincristine, irinotecan, and temozolomide on specific molecules in tumor cells;\n* To determine whether the combination of vorinosat, vincristine, irinotecan, and temozolomide is a beneficial treatment.",[73,64,35,63,132,311],"Germ Cell Tumor","2026-04-10",{"date":291,"type":40},{"date":315,"type":40},"2017-03-17",{"date":44,"type":21},{"name":318,"class":87},"New York Medical College",{"id":320,"slug":321,"hasResults":11,"nctId":322,"briefTitle":323,"officialTitle":324,"acronym":4,"eligibilityCriteria":325,"healthyVolunteers":11,"sex":16,"minAge":97,"maxAge":123,"enrollmentInfo":326,"targetDuration":4,"studyType":22,"phases":328,"briefSummary":329,"conditions":330,"keywords":335,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":339,"lastUpdatePostDateStruct":340,"startDateStruct":342,"completionDateStruct":344,"leadSponsor":346,"locationsCount":88},"100417400","phase-1-interleukin-15-and--21-armored-glypican-3-specific-chimeric-antigen-receptor-expressed-in-t-cells-for-pediatric-solid-tumors-100417400","NCT04715191","Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor Expressed in T Cells for Pediatric Solid Tumors","Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor Expressing Autologous T Cells as an Immunotherapy for Children With Solid Tumors (CARE)","Procurement Eligibility\n\nInclusion Criteria:\n\n* Diagnosis of GPC3-positive\\* solid tumors (as determined by immunohistochemistry with an extent score of \\>=Grade 2 \\[\\>25% positive tumor cells\\] and an intensity score of \\>= 2 \\[scale 0-4\\]).\n* Age ≥1 year and ≤ 21 years\n* Lansky or Karnofsky score ≥60%\n* Life expectancy ≥16 weeks\n* Barcelona Clinic Liver Cancer Stage A, B or C (for patients with hepatocellular carcinoma only)\n* Child-Pugh-Turcotte score \\\u003C7 (for patients with hepatocellular carcinoma only)\n* Informed consent explained to, understood by and signed by patient\u002Fguardian. Patient\u002Fguardian given copy of informed consent\n\nExclusion Criteria:\n\n* History of hypersensitivity reactions to murine protein-containing products OR presence of human anti-mouse antibody (HAMA) prior to enrollment (only patients who have received prior therapy with murine antibodies).\n* History of organ transplantation\n* Known HIV positivity\n* Active bacterial, fungal or viral infection (except Hepatitis B or Hepatitis C virus infections)\n\nTreatment Eligibility\n\nInclusion Criteria:\n\n* Age ≥ 1 year and ≤ 21 years\n* Barcelona Clinic Liver Cancer Stage A, B or C (for patients with hepatocellular carcinoma only)\n* Lansky or Karnofsky score ≥ 60%\n* Child-Pugh-Turcotte score \\\u003C 7 (for patients with hepatocellular carcinoma only)\n* Adequate organ function:\n* Creatinine clearance as estimated by Cockcroft Gault or Schwartz ≥ 60 ml\u002Fmin\n* Total bilirubin \\\u003C 3 times ULN for age\n* INR ≤1.7 (for patients with hepatocellular carcinoma only)\n* Absolute neutrophil count \\> 750\u002Fµl\n* Platelet count \\> 75,000\u002Fµl (Needs to be confirmed prior to treatment whether with or without transfusion)\n* Hgb ≥ 8.0 g\u002Fdl (Needs to be confirmed prior to treatment whether with or without transfusion)\n* Pulse oximetry ≥ 92% on room air\n* Incurable disease after treatment with up- front therapy (Patients who have relapsed disease despite a standard of care salvage therapy)\n* Wash out period, such that patient has recovered from acute toxic effects of all prior chemotherapy and investigational agents before entering this study, and returned to their clinical baseline, as determined by history and physical exam.\n* Sexually active patients must be willing to utilize one of the more effective birth control methods for 6 months after the T-cell infusion.\n* Informed consent explained to, understood by and signed by patient\u002Fguardian. Patient\u002Fguardian given copy of informed consent\n\nExclusion Criteria:\n\n* Pregnancy or lactation\n* Uncontrolled infection\n* Systemic steroid treatment (greater than or equal to 0.5 mg prednisone equivalent\u002Fkg\u002Fday, dose adjustment or discontinuation of medication must occur at least 24 hours prior to CAR T cell infusion)\n* Known HIV positivity\n* Active bacterial, fungal or viral infection \\[except Hepatitis B (HBV patients with active disease who meet the criteria for anti-HBV therapy should be on a suppressive antiviral therapy prior to initiation of cancer therapy) or Hepatitis C virus infections (should have completed curative antiviral treatment with HCV viral load below the limit of quantification\\]\n* 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\n* Active autoimmune or inflammatory disorder\n* Live vaccines within 30 days prior to enrollment\n* History of organ transplantation\n* History of hypersensitivity reactions to murine protein-containing products OR presence of human anti-mouse antibody (HAMA) prior to enrollment (only patients who have received prior therapy with murine antibodies)",{"count":327,"type":21},24,[24],"Patients may be considered if the cancer has come back, has not gone away after standard treatment or the patient cannot receive standard treatment. This research study uses special immune system cells called CARE T cells, a new experimental treatment.\n\nThe body has different ways of fighting infection and disease. No single way seems perfect for fighting cancers. This research study combines two different ways of fighting cancer: antibodies and T cells. Antibodies are types of proteins that protect the body from infectious diseases and possibly cancer. T cells, also called T lymphocytes, are special infection-fighting blood cells that can kill other cells, including cells infected with viruses and tumor cells. Both antibodies and T cells have been used to treat patients with cancers. They have shown promise, but have not been strong enough to cure most patients.\n\nInvestigators have found from previous research that they can put a new gene (a tiny part of what makes-up DNA and carries a person's traits) into T cells that will make them recognize cancer cells and kill them. In the lab, investigators made several genes called a chimeric antigen receptor (CAR), from an antibody called GPC3. The antibody GPC3 recognizes a protein found solid tumors including pediatric liver cancers. This CAR is called GPC3-CAR. To make this CAR more effective, investigators also added two genes that includes IL15 and IL21, which are protein that helps CAR T cells grow better and stay in the blood longer so that they may kill tumors better. The mixture of GPC3-CAR and IL15 plus IL21 killed tumor cells better in the laboratory when compared with CAR T cells that did not have IL15 plus IL21 .This study will test T cells that investigators made (called genetic engineering) with GPC3-CAR and the IL15 plus IL21 (CARE T cells) in patients with GPC3-positive solid tumors.\n\nT cells made to carry a gene called iCasp9 can be killed when they encounter a specific drug called AP1903. The investigators will insert the iCasp9 and IL15 plus IL21 together into the T cells using a virus that has been made for this study. The drug (AP1903) is an experimental drug that has been tested in humans with no bad side-effects. The investigators will use this drug to kill the T cells if necessary due to side effects.\n\nThis study will test T cells genetically engineered with a GPC3-CAR and IL15 plus IL21 (CARE T cells) in patients with GPC3-positive solid tumors.\n\nThe CARE T cells are an investigational product not approved by the Food and Drug Administration.\n\nThe purpose of this study is to find the biggest dose of CARE T cells that is safe, to see how long they last in the body, to learn what the side effects are and to see if the CARE T cells will help people with GPC3-positive solid tumors.",[331,64,332,333,35,334],"Liver Cancer","Malignant Rhabdoid Tumor","Liposarcoma","Yolk Sac Tumor",[336,337,338],"15.21.GPC3-CAR T cells","GPC3","Glypican","2026-04-01",{"date":341,"type":40},"2026-04-06",{"date":343,"type":40},"2024-05-24",{"date":345,"type":21},"2041-07-03",{"name":347,"class":87},"Baylor College of Medicine",{"id":349,"slug":350,"hasResults":11,"nctId":351,"briefTitle":352,"officialTitle":353,"acronym":354,"eligibilityCriteria":355,"healthyVolunteers":11,"sex":16,"minAge":356,"maxAge":357,"enrollmentInfo":358,"targetDuration":4,"studyType":22,"phases":359,"briefSummary":360,"conditions":361,"keywords":362,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":366,"lastUpdatePostDateStruct":367,"startDateStruct":369,"completionDateStruct":371,"leadSponsor":373,"locationsCount":100},"100468825","phase-1-metronomic-chemotherapy-in-wilms-tumor-metrowilms-1906-100468825","NCT05384821","Metronomic Chemotherapy in Wilms Tumor (MetroWilms-1906)","Phase 1-2 Trial Evaluating Metronomic Chemotherapy in Patients With a Relapsed or Refractory Wilms Tumor","MetroWilms","Inclusion Criteria:\n\n* Patient ≥18 months old and ≤ 17 years old\n* Relapsed or refractory Wilms tumor, histologically proven at diagnosis\n* After at least 2 lines of chemotherapy (conventional or high dose, which may include the trial molecules) or after 1 line for high risk relapse for which there would not be any curative therapy. If 1 line for high risk relapse, the enrolment should be confirmed by coordinators.\n* Radiologically measurable or evaluable disease (visible, target or non-target-lesion on MRI or CT-scan)\n* Performance status: Karnofsky performance status (for patients \\>16 years of age) or Lansky Play score (for patients ≤16 years of age) ≥ 70%.\n* Able to take oral medication or nasal gastric tube or authorized gastrostomy\n* Adequate biological criteria:\n\n  * Neutrophils \\> 1000\u002Fmm3 ; Platelets \\> 75 000\u002Fmm3\n  * Transaminases (ALT\u002F AST) ≤ 3 times ULN (or ≤ 6 times ULN if liver metastasis); total bilirubin ≤ 2 ULN (except in case of Gilbert's disease)\n* Creatinine ≤ 1,5 ULN or clearance ≥ 60 mL\u002F min\u002F 1,73m2 (In case of doubt, to be confirm by assessment of cystatin )\n* Females of childbearing potential must have a negative seric pregnancy test within 7 days prior to initiation of treatment.\n* Sexually active patients must agree to use adequate and appropriate contraception (at least one highly effective contraception or two complementary methods of contraception), 1 month before beginning of treatment while on trial drug and for 7 months after stopping the trial drug for female patients and after 6 months for male patients.\n* Written informed consent from parents\u002Flegal representative, patient, and age-appropriate assent before any trial-specific screening procedures according to national guidelines.\n* Patient covered by the French \"Social Security\" regime\n\nExclusion Criteria:\n\n* Prior history of other cancer within 5 years\n* Chemotherapy or radiotherapy of target lesion within 3 weeks prior to inclusion\n* Target therapy within less than 5 \\* half-life of the substance prior to inclusion\n* Major surgery within 15 days prior to inclusion\n* Presence of any NCI-CTCAE v5 grade ≥ 2 cardiac, hepatic, pulmonary or renal toxicity\n* Severe myelosuppression\n* Severe peripheral neuropathy (grade ≥ 2)\n* Fructose intolerance\n* Inflammatory bowel chronic disease and\u002For intestinal obstruction\n* Patients with demyelinating form of Charcot-Marie-Tooth disease\n* Known active viral hepatitis or known human immunodeficiency virus (HIV) infection or any other uncontrolled infection.\n* Known hypersensitivity to dacarbazine (DTIC), isotretinoin or to any of the trial drugs, trial drug classes, excipients in the formulation\n* Hyperlipidemia and hypervitaminosis A\n* Vaccination with a live attenuated vaccine within 1 month prior to inclusion\n* Pregnant or breastfeeding patients\n* Inability to comply with medical follow-up of the trial (geographical, social or psychological reasons)","18 Months","17 Years",{"count":48,"type":21},[24,25],"This is a multicenter, interventional, non-randomized study among patients with a relapsed or refractory Wilms tumor. The study will aim to assess efficacy of metronomic chemotherapy, in terms of disease control after two cycles of metronomic chemotherapy.",[35],[363,364,35,365],"Multidrug chemotherapy","metronomic chemotherapy","Renal cancer","2026-03-16",{"date":368,"type":40},"2026-03-18",{"date":370,"type":40},"2022-09-14",{"date":372,"type":21},"2028-10",{"name":374,"class":87},"Centre Oscar Lambret",{"id":376,"slug":377,"hasResults":11,"nctId":378,"briefTitle":379,"officialTitle":379,"acronym":380,"eligibilityCriteria":381,"healthyVolunteers":11,"sex":16,"minAge":97,"maxAge":382,"enrollmentInfo":383,"targetDuration":4,"studyType":22,"phases":384,"briefSummary":385,"conditions":386,"keywords":391,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":396,"lastUpdatePostDateStruct":397,"startDateStruct":399,"completionDateStruct":401,"leadSponsor":403,"locationsCount":88},"100604325","phase-1-immunotherapy-for-solid-tumor-malignancies-in-pediatrics-using-interleukin-15-and--21-armored-glypican-3-specific-chimeric-antigen-receptor-t-cells-100604325","NCT07148050","Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor T Cells","IMPACT","1. Procurement Eligibility\n\n   Inclusion Criteria:\n   * Diagnosis of a solid tumor expressing GPC3\n   * Lansky or Karnofsky score of \\>=60%\n   * Life expectancy of \\>16 weeks\n   * Informed consent explained to, understood by and signed by patient\u002Fguardian.\n\n   For patients with hepatocellular carcinoma only:\n   * Barcelona Liver Cancer Stage A, B or C\n   * Child-Pugh Turcotte Score \\\u003C7\n\n   Exclusion Criteria:\n   * History of hypersensitivity reactions to murine protein-containing products OR presence of human anti-mouse antibody (HAMA) prior to enrollment for patients who have received prior therapy with murine antibodies.\n\n     * History of organ transplantation\n     * Known HIV positivity\n     * Active bacterial, fungal, or viral infection (except Hepatitis B or Hepatitis C virus infections)\n2. Treatment eligibility\n\nInclusion Criteria:\n\n* Lansky or Karnofsky score of \\>=60%\n* Life expectancy of \\>16 weeks\n* Informed consent explained to, understood by and signed by patient\u002Fguardian.\n* Adequate organ function\n* Adequate laboratory values\n* Refractory or relapsed disease after treatment with up- front therapy and at least one salvage treatment cycle\n* Recovered from acute toxic effects of all prior chemotherapy and investigational agents before entering this study\n* Sexually active patients must be willing to utilize one of the more effective birth control methods for 12 months after the T-cell infusion.\n* Informed consent explained to, understood by and signed by patient\u002Fguardian.\n\nFor patients with hepatocellular carcinoma only:\n\n* Barcelona Liver Cancer Stage A, B or C\n* Child-Pugh Turcotte Score \\\u003C7\n\nExclusion Criteria:\n\n* History of hypersensitivity reactions to murine protein-containing products OR presence of human anti-mouse antibody (HAMA) prior to enrollment for patients who have received prior therapy with murine antibodies.\n\n  * History of organ transplantation\n  * Known HIV positivity\n* Active autoimmune or inflammatory disorder\n* Live vaccines within 30 days prior to enrollment\n\n  • Active bacterial, fungal, or viral infection (except Hepatitis B or Hepatitis C virus infections)\n* Pregnancy or lactation\n* Uncontrolled infection\n* Systemic steroid treatment (≥ 0.5 mg prednisone equivalent\u002Fkg\u002Fday, dose adjustment or discontinuation of medication must occur at least 24hrs prior to CAR T cell infusion)\n* 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.","26 Years",{"count":248,"type":21},[24],"This Phase 1, open-label, non-randomized study will enroll pediatric and young adult subjects with relapsed or refractory non-central nervous system (CNS) malignant solid tumors expressing glypican-3 (GPC3) to examine the safety, feasibility, and efficacy of administering T cell products derived from peripheral blood mononuclear cells (PBMC) that have been genetically modified to co-express a GPC3-specific chimeric antigen receptor (CAR), interleukin (IL)-15 and IL-21 as well as the inducible caspase 9 (iC9) suicide gene (SC-CAR.GPC3xIL15.21 T cells).\n\nA child or young adult meeting all eligibility criteria and meeting none of the exclusion criteria will have a blood sample collected, which will be used to bioengineer the CAR T cells targeting their tumor.",[387,388,332,334,333,64,389,35,390,132],"Solid Tumor (Excluding CNS)","Liver Cell Carcinoma","Embryonal Sarcoma of Liver","Hepatocellular Carcinoma",[392,393,394,395,331,193,337,338],"CAR T cell","Pediatric","Young Adult","Non-CNS Tumor","2026-02-13",{"date":398,"type":40},"2026-02-17",{"date":400,"type":40},"2025-12-22",{"date":402,"type":21},"2044-04-22",{"name":404,"class":87},"Seattle Children's Hospital",{"id":406,"slug":407,"hasResults":11,"nctId":408,"briefTitle":409,"officialTitle":410,"acronym":4,"eligibilityCriteria":411,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":123,"enrollmentInfo":412,"targetDuration":4,"studyType":22,"phases":413,"briefSummary":414,"conditions":415,"keywords":416,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":419,"lastUpdatePostDateStruct":420,"startDateStruct":422,"completionDateStruct":424,"leadSponsor":426,"locationsCount":88},"100531322","phase-1-immunotherapy-for-adults-with-gpc3-positive-solid-tumors-using-il-15-and-il-21-armored-gpc3-car-t-cells-100531322","NCT06198296","Immunotherapy For Adults With GPC3-Positive Solid Tumors Using IL-15 and IL-21 Armored GPC3-CAR T Cells","Immunotherapy For Adults With GPC3-Positive Solid Tumors Using Interleukin-15 And -21 Armored Glypican-3-Specific Chimeric Antigen Receptor Expressing Autologous T Cells","Procurement Inclusion Criteria:\n\n* Diagnosis of GPC3-positive\\* solid tumors (as determined by immunohistochemistry with an extent score of \\>=Grade 2 \\[\\>25% positive tumor cells\\] and an intensity score of \\>= 2 \\[scale 0-4\\]).\n* Age ≥21 years\n* Lansky or Karnofsky score ≥60%\n* Life expectancy ≥16 weeks\n* Barcelona Clinic Liver Cancer Stage A, B or C (- Child-Pugh-Turcotte score \\\u003C7 (for patients with hepatocellular carcinoma only)\n* Informed consent explained to, understood by and signed by patient\u002Fguardian. Patient\u002Fguardian given copy of informed consent \\* GPC3 expression will be evaluated by standard immunohistochemistry (IHC) at Texas Patients's Hospital\u002FBaylor College of Medicine, Department of Pathology for all patients to meet procurement eligibility. All patients will send at least 5 unstained slides.\n\nProcurement Exclusion Criteria:\n\n* History of hypersensitivity reactions to murine protein-containing products OR presence of human anti-mouse antibody (HAMA) prior to enrollment (only patients who have received prior therapy with murine antibodies).\n* History of organ transplantation\n* Known HIV positivity\n* Active bacterial, fungal or viral infection (except Hepatitis B or Hepatitis C virus infections)\n\nTreatment Inclusion Criteria:\n\n* Diagnosis of GPC3-positive solid tumor\n* Age ≥ 21 years\n* Barcelona Clinic Liver Cancer Stage A, B or C\n* Life expectancy of ≥ 12 weeks\n* Lansky or Karnofsky score ≥ 60%\n* Child-Pugh-Turcotte score \\\u003C 7\n* Adequate organ function:\n* Creatinine clearance as estimated by Cockcroft Gault or Schwartz ≥ 60 ml\u002Fmin\n* total bilirubin \\\u003C 3 times ULN for age\n* INR ≤1.7 (for patients with hepatocellular carcinoma only)\n* absolute neutrophil count \\> 500\u002Fµl\n* platelet count \\> 25,000\u002Fµl (can be transfused)\n* Hgb ≥ 7.0 g\u002Fdl (can be transfused)\n* Pulse oximetry \\>90% on room air\n* Refractory or relapsed disease after treatment with up- front therapy and at least one salvage treatment cycle\n* Recovered from acute toxic effects of all prior chemotherapy and investigational agents before entering this study, as determined by history and physical exam\n* Sexually active patients must be willing to utilize one of the more effective birth control methods for 3 months after the T-cell infusion.\n* Informed consent explained to, understood by and signed by patient\u002Fguardian. Patient\u002Fguardian given copy of informed consent\n\nTreatment Exclusion Criteria:\n\n* Pregnancy or lactation\n* Uncontrolled infection\n* Systemic steroid treatment (greater than or equal to 0.5 mg prednisone equivalent\u002Fkg\u002Fday, dose adjustment or discontinuation of medication must occur at least 24hrs prior to CAR T cell infusion)\n* Known HIV positivity\n* Active bacterial, fungal or viral infection (except Hepatitis B or Hepatitis C virus infections)\n* History of organ transplantation\n* History of hypersensitivity reactions to murine protein-containing products OR presence of human anti-mouse antibody (HAMA) prior to enrollment (only patients who have received prior therapy with murine antibodies)",{"count":248,"type":21},[24],"The body has different ways of fighting infection and disease. No single way seems perfect for fighting cancers. This research study combines two different ways of fighting cancer: antibodies and T cells.\n\nAntibodies are types of proteins that protect the body from infectious diseases and possibly cancer. T cells, also called T lymphocytes, are special infection-fighting blood cells that can kill other cells, including cells infected with viruses and tumor cells. Both antibodies and T cells have been used to treat patients with cancers. They have shown promise but have not been strong enough to cure most patients.\n\nIn order to get them to kill cancers more effectively, in the laboratory, the study team inserted a new gene called a chimeric antigen receptor (CAR) into T cells that makes them recognize cancer cells and kill them. When inserted, this new CAR T cell can specifically recognize a protein found on solid tumors, called glypican-3 (GPC3). To make this GPC3-CAR more effective, the study team also added two genes called IL15 and IL21 that help CAR T cells grow better and stay in the blood longer so that they may kill tumors better. When the study team did this in the laboratory, they found that this mixture of GPC3-CAR,IL15 and IL21 killed tumor cells better when compared with CAR T cells that did not have IL15 plus IL21 in the laboratory. This study will use those cells, which are called 21.15.GPC3-CAR T cells, to treat patients with solid tumors that have GPC3 on their surface.\n\nThe study team also wanted to make sure that they could stop the 21.15.GPC3-CAR T cells from growing in the blood should there be any bad side effects. In order to do so, they inserted a gene called iCasp9 into the CO-EXIST T cells. This allows us the elimination of 21.15.GPC3-CAR T cells in the blood when the gene comes into contact with a medication called AP1903. The drug (AP1903) is an experimental drug that has been tested in humans with no bad side-effects. This drug will only be used to kill the T cells if necessary due to side effects .\n\nThe study team has treated patients with T cells that include GPC3. Patients have also been treated with IL-21 and with IL-15. Patients have not been treated with a combination of T cells that contain GPC3, IL-21 and IL-15. To summarize, this study will test the effect of 21.15.GPC3-CAR T cells in patients with solid tumors that express GPC3 on their surface.\n\nThe 21.15.GPC3-CAR T cells are an investigational product not yet approved by the Food and Drug Administration.",[132,390,35,332,334,64,333,389],[417,418],"GPC3-positive tumors","GPC3-CAR T cells","2025-10-30",{"date":421,"type":40},"2025-11-03",{"date":423,"type":40},"2025-06-10",{"date":425,"type":21},"2043-02-01",{"name":347,"class":87},{"id":428,"slug":429,"hasResults":11,"nctId":430,"briefTitle":431,"officialTitle":431,"acronym":4,"eligibilityCriteria":432,"healthyVolunteers":11,"sex":16,"minAge":282,"maxAge":155,"enrollmentInfo":433,"targetDuration":4,"studyType":435,"phases":4,"briefSummary":436,"conditions":437,"keywords":438,"overallStatus":442,"whyStopped":4,"lastUpdateSubmitDate":443,"lastUpdatePostDateStruct":444,"startDateStruct":446,"completionDateStruct":448,"leadSponsor":450,"locationsCount":4},"100579326","accurate-diagnosis-and-grading-of-pediatric-solid-tumors-based-on-pathological-large-models-100579326","NCT06822842","Accurate Diagnosis and Grading of Pediatric Solid Tumors Based on Pathological Large Models","Inclusion Criteria:\n\n1. Neuroblastoma (NB): For newly diagnosed patients with NB aged 0-18 years, the diagnosis criteria are one of the following two items: (1) the patient's tumor tissue has obtained a positive pathological diagnosis under the light microscope; (2) Bone marrow biopsy or aspiration revealed characteristic neuroblastoma cells, which were small round cells, arranged in a nested or chrysanthemum clump or positive staining for anti-GD2 antibodies, and accompanied by an increase in urinary vanillylmandelic acid (VMA) and an increase in blood neuron-specific enolase (NSE).\n2. Wilms tumor (nephroblastoma): patients aged 0-18 years old who have been diagnosed with Wilms tumor by histopathology.\n3. Hepatoblastoma (HB): Patients aged 0-18 years who have been diagnosed with hepatoblastoma by histopathology.\n4. Medulloblastoma (MB): Patients aged 0-18 years with a confirmed histopathological diagnosis of medulloblastoma.\n5. rhabdomyosarcoma (RMS): patients aged 0-18 years old who have been diagnosed with medulloblastoma by histopathology.\n\nExclusion Criteria:\n\n1. The patient's medical record and treatment follow-up information are incomplete; HE is not stained or faded\n2. Those who have 2 or more types of tumors at the same time;\n3. Those who do not meet the enrollment criteria.\n4. Tumor subtype with less than 3 WSI images",{"count":434,"type":21},2000,"OBSERVATIONAL","Pediatric malignancies are the second leading cause of death in the pediatric population, with solid tumors accounting for approximately 60% of all pediatric malignancies. The pathological diagnosis of pediatric solid tumors is highly complex and specialized, because of its diverse tissue morphology, rare tumor subtypes and lack of labeling data, the traditional pathological diagnosis relies on the experience of senior pathologists, but in actual clinical practice, due to the lack of expert resources and inconsistent diagnostic standards, more efficient and accurate auxiliary diagnostic tools are urgently needed. In this study, we aim to construct a multimodal dataset by collecting high-quality pathological images and pathological diagnosis results of pediatric solid tumors (neuroblastoma, medulloblastoma, Wilms tumor, hepatoblastoma, rhabdomyosarcoma, etc.), and introduce medical knowledge enhancement strategies on this basis, and improve the medical reasoning ability and adaptability to fine-grained pathological tasks by injecting domain knowledge (such as molecular characteristics of tumors, pathological grading standards, diagnostic rules, etc.) into the model. Through the model, the representation space of images and texts is unified, and diversified diagnostic tasks of pediatric solid tumors such as tumor region segmentation, cancer detection, and tumor subtype identification are realized, providing intelligent support for the accurate diagnosis and personalized treatment of pediatric solid tumors.",[63,164,35,132,64],[439,440,441],"Machine learning","Diagnosis","Pathological classification","NOT_YET_RECRUITING","2025-02-06",{"date":445,"type":40},"2025-02-12",{"date":447,"type":21},"2025-02-01",{"date":449,"type":21},"2025-04-30",{"name":451,"class":87},"Xinhua Hospital, Shanghai Jiao Tong University School of Medicine",{"id":453,"slug":454,"hasResults":11,"nctId":455,"briefTitle":456,"officialTitle":457,"acronym":4,"eligibilityCriteria":458,"healthyVolunteers":459,"sex":16,"minAge":460,"maxAge":461,"enrollmentInfo":462,"targetDuration":464,"studyType":435,"phases":4,"briefSummary":465,"conditions":466,"keywords":484,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":504,"lastUpdatePostDateStruct":505,"startDateStruct":507,"completionDateStruct":509,"leadSponsor":511,"locationsCount":88},"100315102","international-ppbdicer1-registry-100315102","NCT03382158","International PPB\u002FDICER1 Registry","International Pleuropulmonary Blastoma\u002FDICER1 Registry (for PPB, DICER1 and Associated Conditions)","Inclusion Criteria:\n\n1. Known or suspected PPB or related thoracic tumor\n2. Known or suspected sex-cord stromal tumor including Sertoli-Leydig cell tumor and gynandroblastoma (males or females)\n3. Other known or suspected DICER1-related condition including ovarian sarcoma, cystic nephroma, renal sarcoma, pineoblastoma, pituitary blastoma, nasal chondromesenchymal hamartoma, ciliary body medulloepithelioma and others\n4. Individuals with known or suspected DICER1 pathogenic variation regardless of whether they have an established DICER1-associated condition\n5. Informed consent by patient\u002F or parent\u002Fguardian (also, where appropriate: assent and HIPAA consent)\n\nExclusion criteria:\n\nAbsence of appropriate consent for Registry participation",true,"0 Minutes","100 Years",{"count":463,"type":21},3400,"10 Years","Pleuropulmonary blastoma (PPB) is a rare malignant neoplasm of the lung presenting in early childhood. Type I PPB is a purely cystic lesion, Type II is a partially cystic, partially solid tumor, Type III is a completely solid tumor. Treatment of children with PPB is at the discretion of the treating institution. This study builds off of the 2009 study and will also seek to enroll individuals with DICER1-associated conditions, some of whom may present only with the DICER1 gene mutation, which will help the Registry understand how these tumors and conditions develop, their clinical course and the most effective treatments.",[467,468,469,470,35,471,472,473,474,475,63,476,477,478,479,480,481,482,483],"Pleuropulmonary Blastoma","Sertoli-Leydig Cell Tumor","DICER1 Syndrome","Cystic Nephroma","Pineoblastoma","Renal Sarcoma","Nodular Hyperplasia of Thyroid","Nasal Chondromesenchymal Hamartoma","Ciliary Body Medulloepithelioma","Pituitary Cancer","Embryonal Rhabdomyosarcoma","Ovarian Sarcoma","Gynandroblastoma","Thyroid Carcinoma","Embryonal Rhabdomyosarcoma of Vagina (Diagnosis)","Embryonal Rhabdomyosarcoma of Uterus (Diagnosis)","Embryonal Rhabdomyosarcoma of Cervix",[485,486,487,488,468,470,489,490,491,35,471,472,492,473,493,480,474,494,475,495,63,476,477,496,478,479,497,498,499,500,501,502,503],"pleuropulmonary blastoma","PPB","DICER1","SLCT","CN","DICER1 mutation","DICER1 syndrome","ASK","Thyroid Nodules","NCMH","CBME","ERMS","Peritoneal PPB","pPPB","multinodular goiter","PPB Type I","PPB Type II","PPB Type III","PPB Type Ir","2025-01-28",{"date":506,"type":40},"2025-01-30",{"date":508,"type":40},"2016-12-06",{"date":510,"type":21},"2035-12-06",{"name":512,"class":87},"Children's Hospitals and Clinics of Minnesota","Wilms' Tumor"]