Use of Racotumomab in Patients With Pediatric Tumors Expressing N-glycolylated Gangliosides
NeuroblastomaEwing's Sarcoma3 moreThis study will be carried out in children with diagnosis of cancer with tumors known to express N-glycolylated gangliosides. The disease must be resistant to conventional therapy. The acute toxicity and immune response will be evaluated. The expression of N-glycolylated gangliosides in tumors has previously been investigated in the tumor sample bank at this Hospital. The expression of N-glycolyl GM3 was shown in neuroblastoma, Ewing's sarcoma, Wilm's tumor and retinoblastoma. Gliomas and the aforementioned tumor types have a very bad prognosis when conventional treatment is ineffective. New therapeutic strategies have thus been examined, and several immunotherapeutic approaches, including dendritic cell vaccines, peptide vaccines and anti-idiotype vaccines are currently being assessed. Racotumomab is an anti-idiotype antibody capable of inducing anti-N-glycolyl GM3 antibodies in patients with melanoma, breast cancer and lung cancer. Dose escalation studies have shown the safety of racotumomab in the 0.5 to 2 mg dose range. The 1 mg dose level was selected for the ensuing clinical studies. This clinical trial in children involves three dose levels: 0.15 mg, 0.25 mg and 0.4 mg, owing to the difference in body surface between an adult (1.73 sq. m in average) and the candidate population for this study (0.55 to 0.7 sq. m).
Safety Study of Recombinant Vaccinia Virus to Treat Refractory Solid Tumors in Pediatric Patients...
NeuroblastomaRhabdomyosarcoma3 moreThis is a Phase I, open-label, dose-escalation trial of JX-594 (Pexa-Vec) in pediatric patients with advanced/metastatic, unresectable solid tumors refractory to standard therapy and/or the patient does not tolerate standard therapies. Tumors are likely to include neuroblastoma, lymphoma, Wilms' tumor, rhabdomyosarcoma, Ewing's sarcoma, osteosarcoma, non-rhabdomyosarcoma soft tissue sarcomas, and malignant peripheral nerve sheath tumors. Benign tumors are excluded. These tumor types were selected because evidence of biological activity was observed in cancer cells lines and ex vivo infected primary human tissue samples, specifically pediatric cancer types such as sarcomas and neuroblastomas.
To Evaluate the Safety, Activity and Pharmacokinetics of Marqibo in Children and Adolescents With...
SarcomaNeuroblastoma4 moreBackground: Marqibo(Registered Trademark) is a new anticancer drug. It combines Vincristine sulfate, which is a widely used anticancer drug, and packages it into a tiny fat bubble known as a liposome. The goal of this is to improve the drug's ability to destroy cancer cells and help reduce the potential side effects of treatment. Vincristine sulfate was originally developed from chemicals found in the periwinkle plant and acts against multiple types of malignant cancer. It is approved for multiple cancer types including solid tumors and blood cancers. Research has shown that Marqibo(Registered Trademark) is able to slow or stop the growth of cancer cells in some adults, both alone and in combination with other chemotherapy drugs, but more research is needed to determine its use in children. There has been one previous small study of Marqibo(Registered Trademark) in children. Although some anti-cancer activity was seen, side effects and optimal dosing were not fully determined. As is seen with standard Vincristine suflate, the most common side effect of Marqibo(Registered Trademark) involves the nervous system. It can cause numbness and tingling in the hands and feet. Symptoms commonly improve when the drug is discontinued or the dose is lowered. Objectives: - To determine the safety and efficacy of Marqibo as a treatment for children who have been diagnosed with certain types of malignant cancer that has not responded to standard treatment. Eligibility: Children and adolescents between 2 and 21 years of age who have been diagnosed with certain types of malignant cancer that has not responded to standard treatment. These cancer types include solids tumors, primary brain tumors, leukemias, and lymphomas.
Phase I Study of Ipilimumab (Anti-CTLA-4) in Children and Adolescents With Treatment-Resistant Cancer...
SarcomaWilm's Tumor2 moreThis study will examine the safety and efficacy of ipilimumab-an experimental cancer treatment drug used to boost immune response-in children, adolescents, and young adults. Ipilimumab may allow immune cells to react to and destroy abnormal cells in the body, and has been tested in adults for a variety of cancers and has shown responses in some research studies. Because ipilimumab has not been tested in children, adolescents, or young adults, it is considered an experimental drug. The purposes of this research study are to determine the highest safe dose of ipilimumab for children, adolescents, and young adults with solid tumor cancers; examine its effectiveness and possible side effects; and better understand how the body and the immune system process it over time. Candidates must be between 2 and 21 years of age and must have solid malignant tumors that have been resistant to standard therapy. Volunteers will be screened with a medical history, a clinical examination, and computerized scans such as magnetic resonance imaging (MRI). Participants must have completed their last dose of chemotherapy, radiation, chemotherapy, or antibody or investigational therapy at least four weeks prior to enrollment. During the study, participants will receive an intravenous dose of ipilimumab once every three weeks. The infusion of ipilimumab will last 90 minutes, and the participant s vital signs will be monitored while the medicine is infusing and several times in the first 24 hours after the first dose (requiring a hospital stay during that time). If the participant is able to tolerate the first dose of ipilimumab, further doses (called cycles ) may be received on an outpatient basis. Blood and urine tests will be given on a regular basis during these cycles. After four cycles, participants whose tumors do not grow and who do not have unacceptable side effects will continue to receive ipilimumab every three months to maintain the current condition, until researchers conclude the study.
A Pilot Study of Tumor Cell Vaccine for High-risk Solid Tumor Patients Following Stem Cell Transplantation...
SarcomaNeuroblastoma1 moreLocalized solid tumors such as, sarcoma, neuroblastoma, and Wilms' tumor, can generally be effectively treated with a combination of surgery, radiation and chemotherapy. However, patients with metastatic or relapsed disease have a very poor prognosis. New approaches to the management of these difficult groups of patients are needed. There is evidence to suggest that solid tumors may be good candidates for immunotherapy approaches. In fact, recent experimental evidence indicates that the period of lymphopenia that occurs after stem cell transplant may be an opportune time to use an immunotherapy treatment approach. In light of the very poor prognosis of young patients with advanced solid tumors, this treatment approach warrants further investigation.
Wilm's Tumor 1 (WT1) Peptide Vaccine for High Risk Hematologic Malignancy
Myelodysplastic SyndromeAcute Myeloid Leukemia (AML)1 moreThis study will determine the safety and effectiveness of an experimental vaccine in controlling the abnormal growth of cells in patients with myelodysplastic syndrome (MDS, also known as myelodysplasia), acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and chronic myeloid leukemia (CML). It will test whether the vaccine can increase the number of immune cells responding to the cancer and thereby slow progression of the illness, improve blood counts, reduce the need for transfusions of blood and platelets, or even achieve a disease remission. The vaccine contains part of a protein that is produced in large amounts by cells of patients with these cancers and an added substance called Montanide that helps the immune system respond to the vaccine. Sargramostim, another substances that boosts the immune response, is also given. Patients 18 to 85 years of age with MDS, AML, ALL or CML may be eligible for this study. Candidates are screened with a medical history, physical examination, blood tests, chest x-ray and bone marrow biopsy. Women of childbearing age also have a pregnancy test. Participants undergo the following: Chemotherapy entering the study. Leukapheresis to collect large amounts of white blood cells for infusion before vaccine administration. Participants may need placement of a central line (plastic tube, or catheter) in the upper part of the chest to be used for giving chemotherapy, blood or platelet transfusions, antibiotics and white blood cells, and for collecting blood samples. Weekly vaccine injections for nine weeks, given in the upper arm, upper leg or abdomen. Sargramostim injections following each vaccination. Standard of care treatment for MDS, AML, ALL or CML, which may include blood or platelet transfusions, growth factors, and drugs to control underlying disease and potential side effects of the vaccine. Weekly safety monitoring, including vital signs check, brief health assessment, blood tests and observation after the vaccination, on the day of each vaccination. Follow-up evaluations with blood tests and chest x-ray 3 weeks after the last vaccine dose and with blood tests and bone marrow biopsy 7 weeks after the last vaccine dose.
Continuous Hyperthermic Peritoneal Perfusion (CHPP) With Cisplatin for Children With Peritoneal...
Peritoneal NeoplasmsRetroperitoneal Neoplasms9 moreThere has been no successful treatment of diffuse peritoneal metastasis or carcinomatosis, in childhood tumors. Once this advanced stage of disease is evident, survival is measured in weeks. The selective lethal effect of supranormal temperatures on neoplastic cells and the additive or synergistic effect of combining chemotherapy has been well established in adult clinical trials using continuous hyperthermic peritoneal perfusion (CHPP) for advanced peritoneal adenocarcinoma of gastrointestinal origin, ovarian carcinoma and mesothelioma. This phase I study will evaluate the safety of continuous hyperthermic peritoneal perfusion with escalating doses of intraperitoneal cisplatin in the treatment of children with refractory tumors limited to the abdominal cavity. If tumors are outside the abdominal cavity, the tumors must be able to be controlled. Since CHPP has potential to improve outcome of children with peritoneal and retroperitoneal metastases, this study will evaluate the safety of elevated temperature (40oC) with intraperitoneal cisplatin chemotherapy. Primary Objectives: To determine the MTD and dose-limiting toxicity of intraperitoneal cisplatin given in combination with CHPP as a 90 minute perfusion in children with advanced peritoneal and retroperitoneal solid tumors To determine the safe and tolerable dose of CHPP with cisplatin to be used in Phase II trials To determine the pharmacokinetics of intraperitoneal cisplatin platinum given with CHPP as a 90 minute abdominal perfusion (Optional)
Simvastatin With Topotecan and Cyclophosphamide in Relapsed and/or Refractory Pediatric Solid and...
RetinoblastomaClear Cell Sarcoma10 moreThis is a Phase I trial with new experimental drugs such as simvastatin in combination with topotecan and cyclophosphamide in the hopes of finding a drug that may work against tumors that have come back or that have not responded to standard therapy. This study will define toxicity of high dose simvastatin in combination with topotecan and cyclophosphamide and evaluate for cholesterol levels and IL6/STAT3 pathway changes as biomarkers of patient response.
Intensity-Modulated Radiation Therapy in Treating Younger Patients With Lung Metastases
Adult RhabdomyosarcomaLung Metastases13 moreThis pilot clinical trial studies intensity-modulated radiation therapy (IMRT) in treating younger patients with lung metastases. Specialized radiation therapy that delivers a high dose of radiation directly to the tumor may kill more tumor cells and cause less damage to normal tissue.
Etanercept in Treating Young Patients With Idiopathic Pneumonia Syndrome After Undergoing a Donor...
Accelerated Phase Chronic Myelogenous LeukemiaBlastic Phase Chronic Myelogenous Leukemia23 moreThis phase II trial is studying how well etanercept works in treating young patients with idiopathic pneumonia syndrome after undergoing a donor stem cell transplant. Etanercept may be effective in treating patients with idiopathic pneumonia syndrome after undergoing a donor stem cell transplant.