[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"refractory-rhabdomyosarcoma\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:refractory-rhabdomyosarcoma":34},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,52],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":27,"conditions":28,"keywords":35,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":41,"startDateStruct":44,"completionDateStruct":46,"leadSponsor":48,"locationsCount":51},"100571548","phase-1-peel-224-vincristine-and-temozolomide-in-pediatric-solid-tumors-100571548",false,"NCT06721689","PEEL-224, Vincristine and Temozolomide in Pediatric Solid Tumors","A Phase 1\u002F2 Clinical Trial of the Novel Topoisomerase I Inhibitor PEEL-224 as a Single Agent and in Combination With Vincristine and Temozolomide in Children With Refractory, Progressive or Relapsed Solid Tumors","PEEL-224","Inclusion Criteria:\n\n1. Age:\n\n   * Phase 1: Age greater than or equal to 1 year and less than or equal to18 years\n   * Phase 2 Neuroblastoma (NBL) cohort: Age greater than or equal to 1 year and less than or equal to 30 years\n   * Phase 2 Rhabdomyosarcoma (RMS) cohort: Age greater than or equal to 1 year and less than or equal to18 years\n2. Diagnosis of:\n\n   * Phase 1: Refractory, progressive or relapsed non-central nervous system (CNS) solid tumors who have received at least 1 line of upfront therapy. Patients must have had histologic verification of their malignancy at original diagnosis or relapse\n   * Phase 2: Refractory, progressive or relapsed neuroblastoma (NBL) or rhabdomyosarcoma (RMS) who have received at least 1 line of upfront therapy. Patients must have had histologic verification of their malignancy at original diagnosis or relapse.\n3. Disease status:\n\n   * Phase 1: evaluable or measurable disease\n   * Phase 2, subjects with Neuroblastoma (NBL): evaluable or measurable disease by International Neuroblastoma Response Criteria (INRC); subjects with only bone marrow disease are not eligible\n   * Phase 2, subjects with rhabdomyosarcoma (RMS): measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST)1.1.\n4. Eastern Cooperative Oncology Group (ECOG) Performance Status of 0-2 (age greater than 16 years) or Lansky Performance Status of at least 60 (age less than 16 years).\n5. Females of childbearing potential must have a negative urine\u002Fserum pregnancy test.\n6. Adequate bone marrow function\n\n   Hematologic requirements for all subjects on phase 1 and subjects on phase 2 without malignant infiltration of the bone marrow:\n   * Absolute neutrophil count (ANC) greater than or equal to 750\u002Fmm3 (greater than or equal to 7 days since last dose of short acting myeloid growth factor medications (e.g. filgrastim) and greater than or equal to 14 days since last dose of long-acting myeloid medications (e.g. peg-filgrastim)\n   * Platelet count ≥ 75,000 mm3 (greater than or equal to 14 days since last dose of thrombopoietin receptor agonist such as romiplostim and without platelet transfusion within previous 7 days)\n   * Not refractory to packed red blood cell transfusions\n\n   Hematologic requirements for subjects on phase 2 with malignant infiltration of the bone marrow:\n   * Absolute neutrophil count (ANC) greater than or equal to 500\u002Fmm3 (greater than or equal to 7 days since last dose of short acting myeloid growth factor medications (e.g. filgrastim) and greater than or equal to 14 days since last dose of long-acting myeloid medications (e.g. peg-filgrastim))\n   * Platelet count greater than or equal to 50,000\u002Fmm3 (greater than or equal to 14 days since last dose of thrombopoietin receptor agonist such as romiplostim and without platelet transfusion within previous 7 days)\n   * Not refractory to packed red blood cell transfusions\n   * Patients on phase 2 with malignant infiltration of the bone marrow will not be evaluable for hematologic toxicity.\n7. Adequate renal function as evidenced by creatinine clearance as calculated by the Schwartz equation (see below), radioisotope glomerular filtration rate (GFR) greater than or equal to 70 mL\u002Fmin\u002F1.73 m2, or maximum serum creatinine as below:\n\n   Age Maximum Serum Creatinine (mg\u002FdL)\n\n   Male Female 1 to less than 2 years 0.6 0.6 2 to less than 6 years 0.8 0.8 6 to less than 10 years 1 1 10 to less than 13 years 1.2 1.2 13 to less than 16 years 1.5 1.4 greater than 16 years 1.7 1.4 Threshold derived from the Schwartz formula for estimating glomerular filtration rate (GFR) (Schwartz et al., J.Peds, 106:522,1985) utilizing child length and stature data published by the CDC The Schwartz equation for subjects less than 18 years of age: eGFR (mL\u002Fmin\u002F1.73 m2) = 0.413 x \\[height (cm)\u002Fserum creatinine (mg\u002FdL)\\]\n8. Adequate liver function\n\n   * Aspartate Aminotransferase (AST\u002FSGOT): less than or equal to 3 times the upper limit of normal (ULN) or less than or equal to 5 the upper limit of normal if attributable to disease involvement. For the purpose of this study, the upper limit of normal (ULN) for Aspartate Aminotransferase (AST) is 50 U\u002FL.\n   * Alanine Aminotransferase (ALT\u002FSGPT): less than or equal to 3 times the ULN or less than or equal to 5 the upper limit of normal if attributable to disease involvement. For the purpose of this study, the upper limit of normal (ULN) for Alanine Aminotransferase (ALT) is 45 U\u002FL.\n   * Total bilirubin: less than or equal to 1.5 times the upper limit of normal with the exception of patients with Gilbert's syndrome who must have bilirubin less than 3X institutional upper limit of normal (ULN).\n9. Prior Therapy: Patients must have had resolution of acute toxic effects of prior therapy to grade less than or equal to 1 according to the National Cancer Institute (NCI) common terminology criteria for adverse events (CTCAE) v 5.0 except organ function as noted above, adverse events (AE) that are considered clinically non-significant (i.e. alopecia), or controlled on supportive care (i.e. nausea\u002Fvomiting, hypothyroidism). Patients must meet the following minimum washout periods prior to enrollment:\n\n   * Myelosuppressive chemotherapy: At least 14 days after the last dose of myelosuppressive chemotherapy\n   * Small molecule targeted therapy: At least 7 days following the last dose of a small molecule targeted agent.\n   * Antibody therapy: At least 21 days following the last dose of antibody including anti-GD2 monoclonal antibody.\n   * Cellular therapy: At least 42 days following completion of a cellular therapy agent (e.g. modified T cells, NK cells, dendritic cells)\n   * Autologous hematopoietic stem cell transplant and stem cell boost: Subjects must be at least 60 days from day 0 of an autologous stem cell transplant or stem cell boost.\n   * Myeloid growth factors: At least 7 days following short-acting myeloid growth factor (e.g. filgrastim) and at least 14 days following the last dose of long-acting myeloid growth factor (e.g. peg-filgrastim)\n   * Thrombopoietin receptor agonists: At least 14 days following last dose of thrombopoietin receptor agonist such as romiplostim\n   * Interleukins, interferons, and cytokines (other than hematopoietic growth factors): At least 21 days following the completion of interleukins, interferon, or cytokines, including IL-2\n   * Radiotherapy:\n\n     * At least 14 days after limited field radiation therapy;\n     * At least 90 days after total body irradiation, craniospinal radiotherapy; or radiation to greater than 50% of pelvis;\n     * At least 42 days must have elapsed if other substantial BM radiation.\n   * Radiopharmaceutical therapy (e.g. radiolabeled antibody, 131I- MIBG): At least 42 days after radiopharmaceutical therapy\n   * Major Surgery: At least 2 weeks from prior major surgical procedure. Note: Biopsy, CNS shunt placement\u002Frevision, and central line placement\u002Fremoval are not considered major.\n   * Strong CYP1A2 and\u002For CYP3A4 inhibitors and\u002For inducers: At least 14 days following use of a strong CYP1A2 and\u002For CYP3A4 inhibitor and\u002For inducer. See Appendix 1 for examples. (Note that levofloxacin is permitted when clinically indicated)\n10. Prior treatment with irinotecan and\u002For temozolomide is permitted.\n11. Female patients of reproductive potential must agree to use a highly effective contraceptive method for the duration of study therapy and for at least six months after the final dose of PEEL-224. Males of reproductive potential with a female partner of child-bearing potential must use a highly effective for the duration of the study and for at least six months after the final dose of PEEL-224.\n12. Subjects must agree to use sun protective measures while receiving treatment and for 4 weeks after the last dose of PEEL-224\n13. Parental\u002Fguardian permission (informed consent) and if appropriate, child assent.\n\nExclusion Criteria:\n\n1. Prior treatment with PEEL-224.\n2. Subjects receiving any other anti-cancer agents.\n3. Subjects with primary central nervous system (CNS) solid tumors or central nervous system (CNS) metastatic disease.\n4. Subjects with prior allogeneic stem cell or solid organ transplantation.\n5. Pregnant or lactating females.\n6. Subjects with a known history of human immunodeficiency virus (HIV), hepatitis B, and\u002For hepatitis C (testing not required as part of screening).\n7. Subjects with symptomatic congestive heart failure.","ALL","1 Year","30 Years",{"count":21,"type":22},59,"ESTIMATED","INTERVENTIONAL",[25,26],"PHASE1","PHASE2","The phase 1 primary objective is to determine the pediatric recommended phase 2 dose (RP2D) of PEEL-224 as a single agent (phase 1A) and in combination with vincristine and temozolomide (phase 1B). The phase 2 primary objective is to estimate the objective response rate (ORR) in children with refractory, progressive and relapsed NBL and rhabdomyosarcoma (RMS) treated with the RP2D of PEEL-224 in combination with vincristine and temozolomide.",[29,30,31,32,33,34],"Refractory Solid Tumors","Relapsed Solid Tumors","Relapsed Neuroblastoma","Refractory Neuroblastoma","Relapsed Rhabdomyosarcoma","Refractory Rhabdomyosarcoma",[36,37,38],"Relapsed","Refractory","Solid Tumor","RECRUITING","2026-06-12",{"date":42,"type":43},"2026-06-15","ACTUAL",{"date":45,"type":43},"2025-03-23",{"date":47,"type":22},"2031-04",{"name":49,"class":50},"Theodore Laetsch","OTHER",7,{"id":53,"slug":54,"hasResults":11,"nctId":55,"briefTitle":56,"officialTitle":57,"acronym":4,"eligibilityCriteria":58,"healthyVolunteers":11,"sex":17,"minAge":59,"maxAge":19,"enrollmentInfo":60,"targetDuration":4,"studyType":23,"phases":62,"briefSummary":63,"conditions":64,"keywords":85,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":94,"lastUpdatePostDateStruct":95,"startDateStruct":97,"completionDateStruct":99,"leadSponsor":101,"locationsCount":103},"100431714","phase-1-study-of-onivyde-with-talazoparib-or-temozolomide-in-children-with-recurrent-solid-tumors-and-ewing-sarcoma-100431714","NCT04901702","Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma","A Randomized Phase I\u002FII Study of Talazoparib or Temozolomide in Combination With Onivyde in Children With Recurrent Solid Malignancies and Ewing Sarcoma","Inclusion Criteria\n\nPatients must be \\> 12 months and \\\u003C 30 years at the time of enrollment on study.\n\nPhase I\n\n* Patients with refractory or recurrent non-central nervous system (CNS) solid tumors not amenable to curative treatment are eligible. Patients must have had histologic verification of malignancy at original diagnosis or at the time of relapse. Patients eligible for the expansion cohort, A2, will include non-ES patients with refractory or recurrent non-CNS solid tumors with a deleterious alteration in germline or somatic genes involved in HR repair and DSBs signaling, germline or somatic assessed by prior comprehensive sequencing performed in a CLIA-approved (or equivalent) facility.\n\nPhase II\n\n* Patients with refractory or recurrent Ewing sarcoma (during or after completion of first-line therapy). Refractory disease is defined as progression during first line treatment or within 12 weeks of completion of first line treatment. Recurrent disease includes patients who received first line treatment and experienced disease progression at any time point \\>12 weeks from the completion of first line therapy.\n* Patients must have a histologic diagnosis of Ewing sarcoma with EWSR1- FLI1 translocation or other EWS rearrangement at the time of initial diagnosis. Repeat biopsy at the time of disease recurrence is strongly encouraged but it is not required\u002Fmandated for enrollment.\n\nDisease status\n\n* Patients must have either measurable or evaluable disease (see Section 7.0 for definitions). Measurable disease includes soft tissue disease evaluable by cross-sectional imaging (RECIST). Patients with bone disease without a measurable soft tissue component or bone marrow disease only are eligible for the phase 1 and phase 2 study but will not be included in the OR endpoint.\n* Performance level: Karnofsky \\> 50% for patients \\> 16 years of age and Lansky \\> 50% for patients \\\u003C 16 years of age. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n\nPrior therapy\n\nPhase I Patients who have received prior therapy with an irinotecan-based or temozolomide-based regimen are eligible. Patients who have received prior therapy with a PARP inhibitor other than talazoparib are eligible.\n\nPhase II\n\n* Patients should have received first line therapy and developed either refractory or recurrent disease (first relapse).\n* Organ function: Must have adequate organ and bone marrow function as defined by the following parameters:\n* Patients with solid tumors not metastatic to bone marrow:\n* Peripheral absolute neutrophil count (ANC) \\>1,000\u002Fmm3 (1x109\u002FL)\n* Platelet count \\> 75,000\u002Fmm3 (75x109\u002FL) (no transfusion within 7 days of enrollment)\n* Hemoglobin \\> 9 g\u002FdL (with or without support)\n\nIn the phase I study, patients with solid tumors metastatic to bone marrow or with bone marrow hypocellularity defined as \\\u003C30% cellularity in at least one bone marrow site will be eligible for study, but they will not be evaluable for hematologic toxicity. These patients must not be refractory to red cell or platelet transfusions. At least 2 of every cohort of 3 patients (in the phase I study) must be evaluable for hematologic toxicity. If dose limiting hematologic toxicity is observed at any dose level, all subsequent patients enrolled at that dose level must be evaluable for hematologic toxicity.\n\n* Adequate renal function defined as: Creatinine clearance or radioisotope GFR \\> 60ml\u002Fmin\u002F1.73m2 or a serum creatinine maximum based on age\u002Fsex: age 6months to \\\u003C1 year, creatinine 0.4; 1 to \\\u003C 2 years, creatinine 0.6; 2 \\\u003C 6 years, creatinine 0.8; 6 \\\u003C 10 years, creatinine 1; 10 to \\\u003C13 years, creatinine 1.2; 13 to \\\u003C 16 years creatinine 1.5 (males) or 1.4 (females); \\> 16 years, creatinine 1.7 (males) 1.4 (females)\n* Adequate liver function defined as: normal liver function as defined by SGPT (ALT) concentration \\\u003C5x the institutional ULN, a total bilirubin concentration \\\u003C2x the institutional ULN for age, and serum albumin \\> 2g\u002FdL.\n* Adequate pulmonary function defined as no evidence of dyspnea at rest and a pulse oximetry \\> 94% if there is a clinical indication for determination. Pulmonary function tests are not required.\n* Patients must have fully recovered from the acute toxic effects of chemotherapy, immunotherapy, surgery, or radiotherapy prior to entering this study:\n* Myelosuppressive chemotherapy: Patient has not received myelosuppressive chemotherapy within 3 weeks of enrollment onto this study (8 weeks if received prior myeloablative therapy).\n* Hematopoietic growth factors: At least 7 days must have elapsed since the completion of therapy with a growth factor. At least 14 days must have elapsed after receiving pegfilgrastim.\n* Biologic (anti-neoplastic agent): At least 7 days must have elapsed since completion of therapy with a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period prior to enrollment must be extended beyond the time during which adverse events are known to occur.\n* Monoclonal antibodies: At least 3 half-lives must have elapsed since prior therapy that included a monoclonal antibody or 28 days have elapsed since last dose of the monoclonal antibody with complete resolution of symptoms related to treatment.\n* Radiotherapy: At least 2 weeks must have elapsed since any irradiation; at least 6 weeks must have elapsed since craniospinal RT, 131I-mIBG therapy or substantial bone marrow irradiation (e.g., \\>50% pelvis irradiation).\n* Female participant who is post-menarchal must have a negative urine or serum pregnancy test and must be willing to have additional serum and urine pregnancy tests during the study.\n* Female or male participant of reproductive potential must agree to use effective contraceptive methods at screening and throughout duration of study treatment.\n\nExclusion Criteria\n\nPregnant or breastfeeding\n\n* 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 methods of birth control: a medically accepted barrier of contraceptive method (e.g., male or female condom) and a second method of birth control during protocol therapy. Two highly effective methods of contraception are required for female patients during treatment and for at least 7 months after completing therapy. Male patients with female partners of reproductive potential and\u002For pregnant partners are advised to use two highly effective methods of contraception during treatment and for at least 4 months after the final dose.\n* Male and female participants must agree not to donate sperm or eggs, respectively, after the first dose of study drug through 105 days and 45 days after the last dose of study drug. Females considered not of childbearing potential include those who are surgically sterile (bilateral salpingectomy, bilateral oophorectomy, or hysterectomy).","12 Months",{"count":61,"type":22},90,[25,26],"The phase I portion of this study is designed for children or adolescents and young adults (AYA) with a diagnosis of a solid tumor that has recurred (come back after treatment) or is refractory (never completely went away). The trial will test 2 combinations of therapy and participants will be randomly assigned to either Arm A or Arm B. The purpose of the phase I study is to determine the highest tolerable doses of the combinations of treatment given in each Arm.\n\nIn Arm A, children and AYAs with recurrent or refractory solid tumors will receive 2 medications called Onivyde and talazoparib. Onivyde works by damaging the DNA of the cancer cell and talazoparib works by blocking the repair of the DNA once the cancer cell is damaged. By damaging the tumor DNA and blocking the repair, the cancer cells may die. In Arm B, children and AYAs with recurrent or refractory solid tumors will receive 2 medications called Onivyde and temozolomide. Both of these medications work by damaging the DNA of the cancer call which may cause the tumor(s) to die.\n\nOnce the highest doses are reached in Arm A and Arm B, then \"expansion Arms\" will open. An expansion arm treats more children and AYAs with recurrent or refractory solid tumors at the highest doses achieved in the phase I study. The goal of the expansion arms is to see if the tumors go away in children and AYAs with recurrent or refractory solid tumors. There will be 3 \"expansion Arms\". In Arm A1, children and AYAs with recurrent or refractory solid tumors (excluding Ewing sarcoma) will receive Onivyde and talazoparib. In Arm A2, children and AYAs with recurrent or refractory solid tumors, whose tumors have a problem with repairing DNA (identified by their doctor), will receive Onivyde and talazoparib. In Arm B1, children and AYAs with recurrent or refractory solid tumors (excluding Ewing sarcoma) will receive Onivyde and temozolomide.\n\nOnce the highest doses of medications used in Arm A and Arm B are determined, then a phase II study will open for children or young adults with Ewing sarcoma that has recurred or is refractory following treatment received after the initial diagnosis. The trial will test the same 2 combinations of therapy in Arm A and Arm B. In the phase II, a participant with Ewing sarcoma will be randomly assigned to receive the treatment given on either Arm A or Arm B.",[65,66,67,68,69,70,71,72,73,74,75,76,77,78,79,80,32,81,82,83,34,84],"Recurrent Solid Tumor","Recurrent Ewing Sarcoma","Recurrent Hepatoblastoma","Recurrent Malignant Germ Cell Tumor","Recurrent Malignant Solid Neoplasm","Recurrent Neuroblastoma","Recurrent Osteosarcoma","Recurrent Peripheral Primitive Neuroectodermal Tumor","Recurrent Rhabdoid Tumor","Recurrent Rhabdomyosarcoma","Recurrent Soft Tissue Sarcoma","Recurrent Wilms Tumor","Refractory Ewing Sarcoma","Refractory Hepatoblastoma","Refractory Malignant Germ Cell Tumor","Refractory Malignant Solid Neoplasm","Refractory Osteosarcoma","Refractory Peripheral Primitive Neuroectodermal Tumor","Refractory Rhabdoid Tumor","Refractory Soft Tissue Sarcoma",[86,87,88,89,90,91,92,93],"Pediatric Cancer","Childhood Cancer","Ewing Sarcoma","PARP inhibitor","Solid tumor","Irinotecan","Onivyde","Talazoparib","2026-05-18",{"date":96,"type":43},"2026-05-19",{"date":98,"type":43},"2021-06-09",{"date":100,"type":22},"2026-12-31",{"name":102,"class":50},"St. Jude Children's Research Hospital",10]