[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Giselle Sholler\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":153},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,44,66,86,107,128],{"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":25,"conditions":26,"keywords":28,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"100476895","phase-2-naxitamab-added-to-induction-for-newly-diagnosed-high-risk-neuroblastoma-100476895",false,"NCT05489887","Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma","A Phase II Study of Naxitamab Added to Induction Therapy for Subjects With Newly Diagnosed High-Risk Neuroblastoma","Inclusion Criteria:\n\n1. Diagnosis: Subjects must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular or intermixed) verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites. Subjects with the following disease stages at diagnosis are eligible, if they meet the other specified criteria:\n2. Subjects with newly diagnosed neuroblastoma with INRGSS Stage M disease with either of the following features:\n\n   1. MYCN amplification (\\> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR\n   2. 365 days to ≥ 547 days of age without MYCN amplification, but unfavorable biologic features such as unfavorable histology (INPC) or diploid tumor (DNA index=1) or the presence of any segmental chromosome aberration (SCA) (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number gain at 1q, 2p, or 17q); OR\n   3. Age \\> 547 days of age regardless of biologic features\n\n   Subjects with newly diagnosed neuroblastoma with INRGSS Stage MS disease with either of the following:\n   1. MYCN amplification (\\> 4-fold increase in MYCN signals as compared to reference signals); OR\n   2. 365 days to ≥ 547 days (18 months) of age without MYCN amplification, but unfavorable biologic features such as unfavorable histology (INPC) or diploid tumor (DNA index=1) or SCA as above\n\n   Subjects with newly diagnosed neuroblastoma INRGSS Stage L2 disease with either of the following:\n   1. MYCN amplification (\\> 4-fold increase in MYCN signals as compared to reference signals); OR\n   2. 18 months to \\\u003C5 years of age without MYCN amplification, but with unfavorable histology (INPC); OR\n   3. ≥5 years of age without MYCN amplification, but with undifferentiated or poorly differentiated INPC Subjects with newly diagnosed neuroblastoma INRGSS Stage L1 disease that is incompletely resected with MYCN amplification.\n\n   Subjects \\> 547 days of age initially diagnosed with INRGSS Stage L1, L2 or MS disease who progressed to Stage M without prior chemotherapy may enroll within 4 weeks of progression to Stage M.\n\n   Subjects ≥ 365 days of age initially diagnosed with MYCN amplified INRGSS Stage L1 disease who progress to Stage M without systemic therapy may enroll within 4 weeks of progression to Stage M.\n3. Subjects must be age ≤ 21 years at initial diagnosis.\n4. Subjects must be \\>12 months of age at enrollment.\n5. Adequate cardiac function defined as:\n\n   1. Shortening fraction of ≥ 27% by echocardiogram, or\n   2. Ejection fraction of ≥ 50% by radionuclide evaluation or echocardiogram.\n6. Adequate liver function must be demonstrated, defined as:\n\n   1. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND\n   2. ALT (SGPT) \\\u003C 5 x upper limit of normal (ULN) for age\n7. 1\\. Subjects must have adequate renal function defined as:\n\n   * For subjects \\\u003C 17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL\u002Fmin\u002F1.73 m2) or via radioisotope GFR of ≥ 70 mL\u002Fmin\u002F1.73 m2. The Bedside Schwartz equation is: \\[(0.413) X (Height in cm)\\] \u002F SCr\n   * For subjects ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL\u002Fmin\u002F1.73 m2) or via radioisotope GFR of ≥ 70 mL\u002Fmin\u002F1.73 m2. The Cockcroft and Gault formula is: \\[(140-age) x (Wt in kg) x (0.85 if female)\\] \u002F (72 x SCr)\n   * OR a 24 hour urine Creatinine clearance ≥ 70 mL\u002Fmin\u002F1.73 m2\n8. A negative serum pregnancy test is required for subjects of childbearing potential (≥13 years of age or after onset of menses)\n9. Both male and female post-pubertal study subjects must be willing to use a highly effective contraceptive method (i.e., achieves a failure rate of \\\u003C1% per year when used consistently and correctly) from the time of informed consent (and assent, as applicable) until 6 months after study treatment discontinuation. Such methods include: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomized partner, sexual abstinence.\n10. Informed Consent: All subjects and\u002For legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines and applicable local regulations.\n\nExclusion Criteria:\n\n1. Subjects who are less than 1 year of age\n2. Subjects who are 12-18 months of age with INRGSS Stage M and all stage L2 subjects with favorable biologic features (i.e., nonamplified MYCN, favorable pathology, and DNA index \\> 1) are not eligible.\n3. Subjects who have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and\u002For no more than 1 cycle of chemotherapy.\n4. Treatment with immunosuppressive treatment (topical, inhaled and short-term emergency steroids excluded) within 4 weeks prior to enrollment\n5. Inadequate pulmonary function defined as evidence of dyspnea at rest, exercise intolerance, and\u002For chronic oxygen requirement. In addition, room air pulse oximetry \\\u003C 94% and\u002For abnormal pulmonary function tests if these assessments are clinically indicated.\n6. Pregnant or breastfeeding (NOTE: breast milk cannot be stored for future use while the mother is being treated on study.)\n7. Subjects receiving any investigational drug concurrently.\n8. Subjects with any other medical condition, including but not limited to malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a subject's ability to sign or the legal guardian's ability to sign the informed consent, and subject's ability to cooperate and participate in the study\n9. Subjects with a significant intercurrent illness (any ongoing serious medical problem unrelated to cancer or its treatment) that is not covered by the detailed exclusion criteria and that is expected to interfere with the action of investigational medicinal products (IMPs) or to significantly increase the severity of the toxicities experienced from trial treatment.","ALL","12 Months","21 Years",{"count":20,"type":21},93,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","This is a prospective, multicenter clinical trial in subjects with newly diagnosed high-risk neuroblastoma to evaluate the efficacy and safety of administering naxitamab with standard induction therapy. The initial chemotherapy will include 5 cycles of multi-agent chemotherapy. Naxitamab will be added to all 5 Induction cycles. We hypothesize that the addition of anti-GD2 therapy to induction chemotherapy will result in improved end of induction responses and improved survival.",[27],"Neuroblastoma",[29,30],"naxitimab","induction","RECRUITING","2026-06-30",{"date":34,"type":35},"2026-07-01","ACTUAL",{"date":37,"type":35},"2022-09-14",{"date":39,"type":21},"2036-09",{"name":41,"class":42},"Giselle Sholler","OTHER",25,{"id":45,"slug":46,"hasResults":11,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":4,"eligibilityCriteria":50,"healthyVolunteers":11,"sex":16,"minAge":51,"maxAge":18,"enrollmentInfo":52,"targetDuration":4,"studyType":22,"phases":54,"briefSummary":55,"conditions":56,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":57,"lastUpdatePostDateStruct":58,"startDateStruct":60,"completionDateStruct":62,"leadSponsor":64,"locationsCount":65},"100557657","phase-2-tipifarnib-and-naxitamab-for-relapsedrefractory-neuroblastoma-100557657","NCT06540963","Tipifarnib and Naxitamab for Relapsed\u002FRefractory Neuroblastoma","Phase II Trial of Tipifarnib and Naxitamab for Relapsed\u002FRefractory Neuroblastoma","Inclusion Criteria:\n\n1. Age:\n\n   Subjects must be age ≤ 21 years at initial diagnosis. Subjects must be \\>12 months of age at enrollment. Safety Run-In (first 6 subjects) must be age 6 years or older.\n2. Pathology: All subjects must have a pathologically confirmed diagnosis of neuroblastoma at any point in their treatment.\n3. Tumor assessment: Disease staging must be performed. This disease assessment is required for eligibility and must be done within a maximum of 4 weeks before first dose of study drug.\n4. Disease Status: Relapsed\u002FRefractory Neuroblastoma Relapsed disease defined as neuroblastoma that was previously in remission after standard therapy (at least 4 cycles of aggressive multi-drug induction chemotherapy, with or without radiation and surgery, followed by immunotherapy, or according to a standard high-risk treatment\u002Fneuroblastoma protocol) and has now relapsed and is in any number of relapses.\n\n   Refractory disease defined as High-risk neuroblastoma as defined by the International Neuroblastoma Risk Group Staging System (INRG) that failed to achieve complete response (CR) after at least 4 cycles of aggressive multi-drug induction chemotherapy, progression during upfront therapy, or with disease remaining after standard immunotherapy.\n\n   INRG High Risk NB defined as one of the following:\n   1. Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M with MYCN amplification\n   2. Age ≥ 547 days and INRG Stage M regardless of biologic features\n   3. Any age initially diagnosed with INRG Stage L1 MYCN amplified neuroblastoma (NBL) who have progressed to Stage M without systemic chemotherapy\n   4. Age ≥ 547 days of age initially diagnosed with INRG Stage L1, L2, or MS who have progressed to Stage M without systemic chemotherapy\n5. Measurable Disease: Subjects must be relapsed or refractory with active disease. Subjects must have measurable or evaluable disease, including at least one of the following: Measurable tumor \\>10mm by computed tomography scan (CT) or magnetic resonance imaging (MRI); a positive metaiodobenzylguanidine (MIBG) scan or positron emission tomography (PET) scan or Positive bone marrow biopsy\u002Faspirate.\n6. Subjects with central nervous system (CNS) disease currently taking steroids must have been on a stable dose of steroids for at least one week prior to their biopsy and must not have progressive hydrocephalus at enrollment.\n7. Timing from prior therapy:\n\n   Subjects must have fully recovered from the acute toxic effects of all prior anti- cancer chemotherapy and be within the following timelines:\n   1. Myelosuppressive chemotherapy: Must not have received within 2 weeks of enrollment onto this study (6 weeks if prior nitrosourea).\n   2. Hematopoietic growth factors: At least 5 days since the completion of therapy with a growth factor.\n   3. Small Molecule Inhibitors (anti-neoplastic agent): At least 7 days since the completion of therapy with a small molecule inhibitor. 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. The duration of this interval must be discussed with the Study Chair.\n   4. Immunotherapy: At least 4 weeks since the completion of any type of immunotherapy, e.g. tumor vaccines, CAR-T cells, anti-GD2 Monoclonal antibodies (ex. naxitamab, dinutuximab, etc.).\n   5. XRT (Radiotherapy): At least 30 days since the last treatment except for radiation delivered with palliative intent to a non-target site.\n   6. Stem Cell Transplant:\n\n      * Allogeneic: No evidence of active graft vs. host disease\n      * Allo\u002FAuto: ≥ 2 months must have elapsed since transplant.\n   7. MIBG Therapy: At least 6 weeks since treatment with MIBG therapy.\n8. Subjects must have a Lansky or Karnofsky Performance Scale score of ≥ 50\n9. Subjects must have adequate organ function at the time of enrollment:\n\n   1. Hematological: Hematological recovery as defined by absolute neutrophil count (ANC) ≥750\u002FμL, platelets ≥30\u002FμL (may be transfused).\n   2. Liver: Normal liver function as defined by Aspartate transferase (AST), Alanine transaminase (ALT), and total bilirubin (TBL) all within upper limit of normal\n   3. Renal: Subjects must have adequate renal function defined as Creatinine clearance (in units ml\u002Fmin) or radioisotope GFR ≥ 70. The formula to be used: Adjusted Glomerular Filtration Rate (GFR)=(Estimated GFR×BSA\u002F1.73) mL\u002Fmin.\n   4. Cardiac: Subjects must have a QTcF ≤ 470 msc.\n10. Subjects of childbearing potential must have a negative pregnancy test. Subjects of childbearing potential must agree to use an effective birth control method.\n11. Subjects who are lactating must agree to stop breast-feeding. (NOTE: breast milk cannot be stored for future use while the mother is being treated on study.)\n12. Written informed consent in accordance with institutional and FDA guidelines must be obtained from all subjects (or subjects' legal representative).\n\nExclusion Criteria:\n\n1. Subjects who are less than 1 year of age\n2. BSA of \\\u003C0.25 m2\n3. Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.\n4. Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.\n5. Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.\n6. Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.\n7. Previous Gr.4 allergic or anaphylactic reaction to naxitamab, leading to the discontinuation of naxitamab during prior therapy.","1 Year",{"count":53,"type":21},98,[24],"The purpose of this study is to evaluate the investigational drug, tipifarnib (a pill taken by mouth), in combination with the Food and Drug Administration (FDA) approved drug, naxitimab, administered intravenously (IV; a liquid that continuously goes into your body through a tube that has been placed during a surgery into one of your veins). Naxitamab is FDA approved for pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy, it may not be approved in the type of disease used in this study.\n\nThe goals of this part of the study are:\n\n* Test the safety and tolerability of tipifarnib in combination with naxitimab in patients with cancer\n* To determine the activity of study treatments chosen based on:\n* How each subject responds to the study treatment\n* How long a subject lives without their disease returning\u002Fprogressing",[27],"2026-04-24",{"date":59,"type":35},"2026-04-28",{"date":61,"type":35},"2024-12-06",{"date":63,"type":21},"2035-12-01",{"name":41,"class":42},12,{"id":67,"slug":68,"hasResults":11,"nctId":69,"briefTitle":70,"officialTitle":71,"acronym":4,"eligibilityCriteria":72,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":18,"enrollmentInfo":73,"targetDuration":4,"studyType":22,"phases":75,"briefSummary":76,"conditions":77,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":57,"lastUpdatePostDateStruct":79,"startDateStruct":80,"completionDateStruct":82,"leadSponsor":84,"locationsCount":85},"100415929","phase-2-dfmo-as-maintenance-therapy-for-molecular-highvery-high-risk-and-relapsed-medulloblastoma-100415929","NCT04696029","DFMO as Maintenance Therapy for Molecular High\u002FVery High Risk and Relapsed Medulloblastoma","Phase II Trial of Eflornithine\u002FDFMO as Maintenance Therapy for Molecular High Risk\u002FVery High Risk and Relapsed\u002FRefractory Medulloblastoma","Inclusion Criteria:\n\n1. Age: 0-21 years of age at diagnosis\n2. Pathology All patients must either have a pathologically confirmed diagnosis of medulloblastoma with molecular grouping identified by either Nanostring or methylation profiling.\n\n   Cohort 1- Molecular High Risk:\n   * Metastatic non-MYC amplified Group 3\n   * Metastatic Group 4\n   * Metastatic non-WNT\u002Fnon-SHH (Must be non-MYC amplified)\n\n   Cohort 2- Molecular Very High Risk\n   * Metastatic OR MYCN amplified OR TP53 mutant non-infant (\\>3 yrs) SHH\n   * MYC amplified Group 3\n   * Non-WNT, non-SHH infant (\\\u003C 3 yrs)\n\n   Cohort 3: Relapsed\u002FRefractory Medulloblastoma\n3. Pre-enrollment tumor survey:\n\n   Prior to enrollment on this study, a determination of mandatory disease staging must be performed:\n   * Tumor imaging studies including: Brain and spine MRI\n   * Lumbar Puncture only if previously positive\n   * Bone Marrow aspiration\u002Fbiopsy only if previously positive\n   * This disease assessment is required for eligibility and preferably should be done within 2 weeks prior to first dose of study drug, but must be done within a maximum of 4 weeks before first dose of study drug.\n4. Disease Status: Subjects must have no evidence of disease, or stable\\* residual nonbulky\\*\\* disease.\n\n   \\*Stable residual disease defined as non-progression over 2 separate imaging studies at least 6 weeks apart\n\n   \\*\\*Non-bulky disease defined as maximal cross-sectional area \\\u003C 3cm\\^2 at enrollment. Patients with leptomeningeal disease are allowed to participate on study.\n5. Timing from prior therapy:\n\n   Enrollment (first dose of DFMO) no later than 60 days after last dose of conventional chemotherapy. Patients who have undergone high dose chemotherapy (HDCT) with autologous stem cell transplantation (SCT) are eligible if more than 45 days have elapsed since date of last SCT.\n6. Patients must have a Lansky or Karnofsky Performance Scale score of ≥ 50% (see Appendix II) and patients must have a life expectancy of ≥ 2 months.\n7. All clinical and laboratory studies for organ functions to determine eligibility must be performed within 7 days prior to first dose of study drug unless otherwise indicated below.\n8. Patients must have adequate organ functions at the time of registration:\n\n   * Hematological: Hematological recovery as defined by ANC ≥750\u002FμL, platelets ≥30 (non-transfused x 7 days)\n   * Liver: Adequate liver function as defined by AST and ALT \\\u003C10x upper limit of normal\n   * Renal: Adequate renal function defined as (perform one of the following): Creatinine clearance or radioisotope GFR ≥ 70 mL\u002Fmin\u002F1.73 m2 or a serum creatinine based on age\u002Fgender\n9. Females of childbearing potential must have a negative pregnancy test. Patients of childbearing potential must agree to use an effective birth control method. Female patients who are lactating must agree to stop breast-feeding.\n10. Written informed consent in accordance with institutional and FDA guidelines must be obtained from all subjects (or patients' legal representative).\n\nExclusion Criteria:\n\n1. BSA of \\\u003C0.25 m2\n2. Metastatic disease outside of CNS\n3. Relapsed\u002Frefractory patients who are radiation-naïve and age 5 years or older at time of enrollment\n4. Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.\n5. Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.\n6. Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.\n7. Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.",{"count":74,"type":21},118,[24],"Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study as Maintenance Therapy for Molecular High Risk\u002FVery High Risk and Relapsed\u002FRefractory Medulloblastoma.",[78],"Medulloblastoma",{"date":59,"type":35},{"date":81,"type":35},"2021-03-29",{"date":83,"type":21},"2029-03",{"name":41,"class":42},24,{"id":87,"slug":88,"hasResults":11,"nctId":89,"briefTitle":90,"officialTitle":91,"acronym":92,"eligibilityCriteria":93,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":94,"enrollmentInfo":95,"targetDuration":4,"studyType":22,"phases":97,"briefSummary":98,"conditions":99,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":57,"lastUpdatePostDateStruct":100,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":105,"locationsCount":106},"100252047","phase-2-pediatric-precision-laboratory-advanced-neuroblastoma-therapy-100252047","NCT02559778","Pediatric Precision Laboratory Advanced Neuroblastoma Therapy","A Study Using Molecular Guided Therapy With Induction Chemotherapy Followed by a Randomized Controlled Trial of Standard Immunotherapy With or Without DFMO Followed by DFMO Maintenance for Subjects With Newly Diagnosed High-Risk Neuroblastoma","PEDS-PLAN","Part A- CLOSED:\n\n1. Diagnosis: Subjects must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular or intermixed) verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites. Subjects with the following disease stages at diagnosis are eligible, if they meet the other specified criteria:\n\n   a) Subjects with newly diagnosed neuroblastoma with INSS Stage 4 are eligible with the following: i. Age \\> 18 months (\\> 547 days) regardless of biologic features or ii. Age 12-18 months (365-547 days) with any of the following 3 unfavorable biologic features (MYCN amplification, unfavorable pathology and\u002For DNA index = 1) or iii. MYCN amplification (\\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features.\n\n   b) Subjects with newly diagnosed neuroblastoma with INSS Stage 3 are eligible with the following: i. MYCN amplification (\\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features or ii. Age \\> 18 months (\\> 547 days) with unfavorable pathology, regardless of MYCN status.\n\n   c) Subjects with newly diagnosed neuroblastoma with INSS Stage 2A\u002F2B with MYCN amplification (\\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features.\n2. Subjects must be age ≤ 21 years at initial diagnosis\n3. Subjects must not have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and\u002For no more than 1 cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (as per P9641, A3961, ANBL0531, or similar) prior to determination of MYCN amplification status and histology.\n4. Specimens will be obtained only in a non-significant risk manner and not solely for the purpose of investigational testing.\n5. Ability to tolerate PBSC collection: No known contraindication to PBSC collection. Examples of contraindications would include a weight or size less than that determined to be feasible at the collecting institution, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and\u002For the apheresis procedure.\n\n   Part A and B both- Part A CLOSED, Part B- OPEN:\n6. Adequate Cardiac Function Defined As:\n\n   1. Shortening fraction of ≥ 27% by echocardiogram, or\n   2. Ejection fraction of ≥ 50% by radionuclide evaluation or echocardiogram.\n7. Adequate liver function must be demonstrated, defined as:\n\n   c. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND d. ALT (SGPT) \\\u003C 10 x upper limit of normal (ULN) for age\n8. Subjects must have adequate renal function defined as a serum creatinine based on age\u002Fgender as follows:\n\n   Age Maximum Serum Creatinine (mg\u002FdL) Male Female 1 month to \\\u003C 6 months 0.4 0.4 6 months to \\\u003C 1 year 0.5 0.5 1 to \\\u003C 2 years 0.6 0.6 2 to \\\u003C 6 year 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 1.4\n\n   ≥ 16 years 1.7 1.4\n9. A negative serum pregnancy test is required for female participants of child bearing potential (≥13 years of age or after onset of menses)\n10. Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives (\"the pill\"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended.\n11. Informed Consent: All subjects and\u002For legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines.\n\n    Part B- OPEN:\n12. All patients must have a pathologically confirmed diagnosis of neuroblastoma, be age ≤ 21 years at initial diagnosis, and classified as high risk by the criteria used by COG or SIOPEN at the time of diagnosis. Exception: patients who are initially diagnosed as non-high-risk neuroblastoma, but later converted (and\u002For relapsed) to high risk neuroblastoma are also eligible.\n13. Previous Therapy- subjects must fit into one of the strata categories listed in section 10.5 to be eligible to enroll on Part B of this study.\n14. Pre-enrollment tumor survey:\n\n    Prior to enrollment on Part B, a determination of mandatory disease staging must be performed. Tumor imaging studies including CT or MRI, MIBG or PET, and VMA\u002FHVA (PET scan should be done for patients with prior disease that was MIBG non-avid). Bone marrow aspirates and biopsies are required.\n\n    This disease assessment is required for eligibility and should be done preferably within 2 weeks, but must be done within a maximum of 4 weeks before first dose of study drug.\n15. Timing- Enrollment to occur prior to Day + 120 post-transplant, preferably when the subject is within 28 days after completing local radiation therapy (if given).\n\nExclusion Criteria (Part A and B)\n\n1. Subjects who are 12-18 months of age with INSS Stage 4 and all stage 3 subjects with favorable biologic features (ie, nonamplified MYCN, favorable pathology, and DNA index \\> 1) are not eligible.\n2. Lactating females are not eligible unless they have agreed not to breastfeed their infants.\n3. Subjects receiving any investigational drug concurrently.\n4. Subjects with any other medical condition, including but not limited to malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a subject's ability to sign or the legal guardian's ability to sign the informed consent, and subject's ability to cooperate and participate in the study","22 Years",{"count":96,"type":21},500,[24],"A prospective open label, multicenter study to evaluate the feasibility and acute toxicity of using molecularly guided therapy in combination with standard therapy followed by a Randomized Controlled Trial of standard immunotherapy with or without DFMO followed by DFMO maintenance for Subjects with Newly Diagnosed High-Risk Neuroblastoma.",[27],{"date":59,"type":35},{"date":102,"type":35},"2015-09",{"date":104,"type":21},"2035-09",{"name":41,"class":42},28,{"id":108,"slug":109,"hasResults":11,"nctId":110,"briefTitle":111,"officialTitle":111,"acronym":4,"eligibilityCriteria":112,"healthyVolunteers":11,"sex":16,"minAge":113,"maxAge":114,"enrollmentInfo":4,"targetDuration":4,"studyType":115,"phases":4,"briefSummary":116,"conditions":117,"keywords":4,"overallStatus":122,"whyStopped":4,"lastUpdateSubmitDate":123,"lastUpdatePostDateStruct":124,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":126,"locationsCount":127},"100330361","an-intermediate-expanded-use-trial-of-dfmo-100330361","NCT03581240","An Intermediate Expanded Use Trial of DFMO","Inclusion Criteria:\n\n* Age: 0-30 years at the time of initial diagnosis.\n* Diagnosis: Histologic verification at either the time of original diagnosis or a previous relapse of High Risk neuroblastoma, medulloblastoma, atypical teratoid rhabdoid tumor, embryonal tumor with abundant neuropil and true rosettes, ependymoblastoma, medulloepithelioma and other rare pediatric MYC, ODC or LIN28\u002FLet7 driven tumors (each type will form a new subset).\n* Disease Status: Subjects must be in one of the following disease categories:\n\n  1. High risk neuroblastoma patients that have completed standard of care upfront therapy and are not eligible for NMTRC014.\n  2. Medulloblastoma patients who have completed standard of care therapies.\n  3. Relapsed\u002Frefractory neuroblastoma patients who have completed standard of care therapies.\n  4. Rare tumors with increased LIN28 expression or MYCN amplification or up regulation of ornithine decarboxylase who have completed standard of care therapies.\n* Subjects are not eligible to enroll on DFMO studies NMTRC002, NMTRC003, NMTRC010, or NMTRC014.\n* A negative serum or urine pregnancy test is required for female subjects of child bearing potential (onset of menses or ≥13 years of age).\n* Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives (\"the pill\"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended.\n* Informed Consent: All subjects and\u002For legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines\n\nExclusion Criteria:\n\n* BSA (m2) of \\\u003C0.25\n* Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.","0 Years","30 Years","EXPANDED_ACCESS","To provide DFMO in an expanded use setting to subjects with relapsed rare tumors with increased LIN28 expression or MYCN amplification or up regulation of ornithine decarboxylase.",[27,78,118,119,120,121],"Typical Teratoid Rhabdoid Tumor","Embryonal Tumor With Abundant Neuropil and True Rosettes","Ependymoblastoma","Medulloepithelioma","AVAILABLE","2026-02-18",{"date":125,"type":35},"2026-02-20",{"name":41,"class":42},1,{"id":129,"slug":130,"hasResults":11,"nctId":131,"briefTitle":132,"officialTitle":133,"acronym":4,"eligibilityCriteria":134,"healthyVolunteers":135,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":136,"targetDuration":138,"studyType":139,"phases":4,"briefSummary":140,"conditions":141,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":144,"lastUpdatePostDateStruct":145,"startDateStruct":147,"completionDateStruct":149,"leadSponsor":151,"locationsCount":152},"100417399","beat-childhood-cancer-specimen-banking-and-data-registry-100417399","NCT04715178","Beat Childhood Cancer Specimen Banking and Data Registry","Specimen Banking With Clinical and Genomic Data Registry With the Establishment of Tumor Models for Pediatric Cancers","Inclusion Criteria:\n\n* No age restrictions\n* Subjects that fit into one of the following categories:\n\n  1. Subjects with suspected or confirmed cancers of childhood, adolescence, or young adulthood.\n  2. Survivors of a cancer of childhood, adolescence, or young adulthood (\\> 6 months off last therapy for cancer)\n  3. Subjects with a disease-matched condition\n  4. Healthy volunteers- including any participants with medical conditions that are not disease-matched.\n* Written informed consent in accordance with institutional and FDA guidelines must be obtained from all subjects (or subjects' legal representative).\n\nExclusion Criteria:\n\n* Subject or his\u002Fher legally authorized representative (where applicable) did not consent for specimen banking and data collection for this study.",true,{"count":137,"type":21},1000,"10 Years","OBSERVATIONAL","This is an observational data registry study of pediatric cancer patients at participating Beat Childhood Cancer Consortium sites involving specimen banking and data collection.",[142,143],"Pediatric Cancer","Survivorship","2026-01-30",{"date":146,"type":35},"2026-02-03",{"date":148,"type":35},"2021-02-02",{"date":150,"type":21},"2032-01",{"name":41,"class":42},18,""]