Treatment Clinical Trials for Neuroblastoma

Clinical trials are research studies that involve people. The clinical trials on this list are for neuroblastoma treatment. All trials on the list are supported by NCI.

NCI’s basic information about clinical trials explains the types and phases of trials and how they are carried out. Clinical trials look at new ways to prevent, detect, or treat disease. You may want to think about taking part in a clinical trial. Talk to your doctor for help in deciding if one is right for you.

Trials 26-50 of 67

  • Endoscopic Surgery Followed by Cisplatin and Proton Beam Radiation Therapy in Treating Patients with Nasal Tumors That Cannot Be Removed by Surgery

    This phase II trial studies how well endoscopic surgery followed by cisplatin and proton beam radiation therapy works in treating patients with nasal tumors that cannot be removed by surgery. Endoscopic surgery is a type of operation that uses small tubes containing a camera and small tools so that a large incision does not need to be made. Proton beam radiation therapy uses radioactive material placed directly into or near a tumor to kill tumor cells. Drugs used in chemotherapy, such as cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Endoscopic surgery followed by cisplatin and proton beam radiation therapy may work better in treating patients with nasal tumors.
    Location: 5 locations

  • Fimepinostat in Treating Younger Patients with Relapsed or Refractory Solid Tumors, Central Nervous System Tumors, or Lymphoma

    This phase I trial studies the side effects of fimepinostat in treating younger patients with solid tumors, central nervous system tumors, or lymphoma that has come back or does not respond to treatment. Fimepinostat may stop the growth of cancer or tumor cells by blocking some of the enzymes needed for cell growth.
    Location: 5 locations

  • Safety and Pharmacokinetics of Cobimetinib in Pediatric and Young Adult Participants With Previously Treated Solid Tumors

    This open-label, dose-escalation study is designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of cobimetinib in pediatric and young adult participants with solid tumors with known or potential kinase pathway activation for which standard therapy has proven to be ineffective or intolerable or for which no curative standard-of-care treatment options exist. The study will be conducted in two stages: a dose-escalation stage and an expansion stage at the recommended dose.
    Location: 5 locations

  • Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and / or Bone Marrow

    Children and adults diagnosed with high-risk neuroblastoma patients with primary refractory disease or incomplete response to salvage treatment in bone and / or bone marrow will be treated for up to 93 weeks with naxitamab and granulocyte-macrophage colony stimulating factor (GM-CSF). Patients will be followed for up to five years after first dose. Naxitamab, also known as hu3F8 is a humanised monoclonal antibody targeting GD2
    Location: 4 locations

  • 131I-omburtamab Radioimmunotherapy for Neuroblastoma Central Nervous System / Leptomeningeal Metastases

    Children with a neuroblastoma diagnose and central nervous system (CNS) / leptomeningeal metastases will be given up to 2 rounds of intracerebroventricular treatment with a radiolabelled monoclonal antibody, 131I-omburtamab to evaluate efficacy and safety
    Location: 4 locations

  • Trial of Afatinib in Pediatric Tumours

    Open-label, dose escalation, monotherapy, basket trial with biomarker specific MTD expansion cohort / Phase II part. The trial will consist of 2 parts: 1. Dose finding part to determine the MTD 2. Biomarker specific MTD expansion cohort / Phase II part to assess clinical anti-tumour activity in included tumour types
    Location: 4 locations

  • OKT3 / Humanized 3F8 Bispecific Antibody-Activated T Lymphocytes, Aldesleukin, and Sargramostim in Treating Younger Patients with GD2-Positive Metastatic, Recurrent or Refractory Solid Tumors

    This phase I / II trial studies the side effects and best dose of OKT3 / humanized 3F8 bispecific antibody-activated T lymphocytes with given together with aldesleukin and sargramostim and to see how well they work in treating younger patients with disialoganglioside GD2 (GD2)-positive solid tumors that have spread to other parts of the body (metastatic), have come back (recurrent), or do not respond to treatment (refractory). Biological therapies, such as OKT3 / humanized 3F8 bispecific antibody-activated T lymphocytes, use substances made from living organisms that may attack specific tumor cells and stop them from growing or kill them. Aldesleukin and sargramostim may stimulate white blood cells to kill tumor cells. Giving white blood cells that have been activated by OKT3 / humanized 3F8 bispecific antibody-activated T lymphocytes with aldesleukin and sargramostim may kill more tumor cells.
    Location: 4 locations

  • Induction Chemotherapy without G-CSF in Treating Participants with High-Risk Neuroblastoma

    This pilot early phase I trial studies the side effects of induction chemotherapy without granulocyte colony-stimulating factor (G-CSF) when treating participants with high-risk neuroblastoma. Drugs used in chemotherapy, such as topotecan, cyclophosphamide, cisplatin, etoposide, vincristine, and doxorubicin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Induction chemotherapy is usually followed by a drug called G-CSF (filgrastim or pegfilgrastim) to prevent side effects from the chemotherapy. Removing G-CSF following induction chemotherapy may help kill more tumor cells in participants with high-risk neuroblastoma.
    Location: 3 locations

  • A Two-Part Study of TB-403 in Pediatric Subjects With Relapsed or Refractory Medulloblastoma

    The purpose of this study is to assess the safety and tolerability profile of TB-403 (humanized monoclonal antibody against placental growth factor (PlGF)) in pediatric subjects with relapsed or refractory Medulloblastoma.
    Location: 3 locations

  • Abemaciclib and Radiation Therapy in Treating Younger Patients with Newly Diagnosed Diffuse Intrinsic Pontine Glioma, Recurrent or Refractory Solid Tumors, or Malignant Brain Tumors

    This phase I trial studies the side effects and best dose of abemaciclib when given together with radiation therapy in treating younger patients with newly diagnosed diffuse intrinsic pontine glioma (a type of central nervous system tumor that forms from glial [supportive] tissue of the brain and spinal cord), or solid tumors that have come back (recurrent) or does not respond to treatment (refractory), or malignant brain tumors. Abemaciclib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Radiation therapy uses high energy x-rays to kill tumor cells and shrink tumors. Giving abemaciclib together with radiation therapy may be a better treatment in patients with diffuse intrinsic pontine glioma, solid tumors, or malignant brain tumors.
    Location: 3 locations

  • Pediatric Precision Laboratory Advanced Neuroblastoma Therapy

    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.
    Location: 4 locations

  • Fenretinide Lym-X-Sorb + Ketoconazole + Vincristine for Recurrent or Resistant Neuroblastoma

    Currently there is no known effective treatment for recurrent or resistant neuroblastoma. Fenretinide is an anticancer agent that may work differently than standard chemotherapy. It may cause the buildup of wax-like substances in cancer cells called ceramides. In laboratory studies, it was found that if too much ceramide builds up in the neuroblastoma cells, they die. Fenretinide has been given by mouth as a capsule to many people, including children. When Fenretinide is given in capsules, very little of the drug is absorbed through the intestines into the body. This means patients have to take many capsules of fenretinide by mouth several times a day. In this study, a new oral preparation of fenretinide (called 4-HPR / LXS oral powder) is being tested to see if more fenretinide can be absorbed into the body. 4-HPR / LXS oral powder has been tested previously in a limited number of both children and adult cancer patients. Ketoconazole, commonly used to treat fungus infections, can increase fenretinide levels in the body by interfering with the body's ability to break down fenretinide. Ketoconazole will be given at the same time as the fenretinide powder. There is preclinical data that shows that combining fenretinide and vincristine prolonged survival in animal models, therefore, it is hoped that giving the vincristine with fenretinide will work better against the neuroblastoma that either drug given alone. About 70 children with neuroblastoma have been treated with various versions of the fenretinide powder to date, including about a dozen children that also took the fenretinide powder with ketoconazole, and no toxicities have occurred that limited the dosage and no serious or unexpected side effects occurred. However, vincristine has never been given with fenretinide or fenretinide plus ketoconazole before. Vincristine has been been given before with ketoconazole to both children and adults with neuroblastomas and other cancers.
    Location: 3 locations

  • A Study of Therapeutic Iobenguane (131-I) for Relapsed, High-Risk Neuroblastoma Subjects

    The purpose of this study is to evaluate the efficacy and safety of 131I-MIBG in patients with neuroblastoma, who relapsed.
    Location: 4 locations

  • Modified Immune Cells (GD2 Specific Chimeric Antigen Receptor and IL-15 Expressing Autologous Natural Killer T-Cells) in Treating Children with Relapsed or Refractory Neuroblastoma

    This phase I trial studies the best dose and side effects of GD2 specific chimeric antigen receptor (CAR) and interleukin-15 (IL-15) expressing autologous natural killer T-cells (G28z.15 NKTs) in treating children with neuroblastoma that has come back or does not respond to treatment. This trial combines two different ways of fighting cancer: antibodies and natural killer 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. GD2-CAR natural killer T cells are modified immune cells that have been engineered in the laboratory to specifically target GD2 proteins found on neuroblastoma tumor cells and kill them. IL-15 is critical for the development and maintenance of T cells. These new cells may be able to slow the growth of tumor cells in patients with neuroblastoma.
    Location: 2 locations

  • Immunotherapy of Relapsed Refractory Neuroblastoma With Expanded NK Cells

    This NANT trial will determine the maximum tolerated dose (MTD) of autologous expanded natural killer (NK) cells when combined with standard dosing of ch14.18 and will assess the feasibility of adding lenalidomide at the recommended Phase II dose of the expanded NK cells with ch14.18, for treatment of children with refractory or recurrent neuroblastoma.
    Location: 5 locations

  • Phase 1 Study of MM-398 Plus Cyclophosphamide in Pediatric Solid Tumors

    This is a Phase 1 study of the combination of two drugs: MM-398 and Cyclophosphamide. The goal is to find the highest dose of MM-398 that can be given safely when it is used together with the chemotherapy drug Cyclophosphamide.
    Location: 3 locations

  • Hu3F8-BsAb in Treating Patients with Relapsed or Refractory Neuroblastoma, Osteosarcoma, or other GD2 Positive Solid Tumors

    This phase I / II trial studies the side effects and best dose of Hu3F8-BsAb, and how well it works in treating patients with neuroblastoma, osteosarcoma, or other GD2 positive solid tumors that have come back or does not respond to treatment. Immunotherapy with Hu3F8-BsAb, may induce changes in body’s immune system and may interfere with the ability of tumor cells to grow and spread.
    Location: Memorial Sloan Kettering Cancer Center, New York, New York

  • Modified Immune Cells (Autologous iC9.GD2.CAR.IL-15 T cells) for Treating Patients with Relapsed or Refractory High Risk Neuroblastoma or Ganglioneuroblastoma

    This phase I trial studies the side effects and best dose of modified immune cells (autologous iC9.GD2.CAR.IL-15 T cells) for treating patients with high risk neuroblastoma that has come back (recurrent) or does not respond to treatment (refractory), or ganglioneuroblastoma. Antibodies and T cells are two different ways the body fights disease. When T cells have part of an antibody attached to them they are better at recognizing and killing tumor cells. The treatment that is being research on this trial, autologous iC9.GD2.CAR.IL-15 T cells, combines T cells and antibodies in order to create a more effective treatment. An antibody called anti-GD2 joins to T cells in the blood and can detect and stick to neuroblastoma cells because they have a substance on the outside of them called GD2. The IL-15 gene, which cells use to communicate with one another, is added so that the T cells can attack tumor cells more effectively. The iC9 gene is added as an "off-switch" so that it can stop the T cells from working if serious side effects are experienced. This study is being done to see if giving autologous iC9.GD2.CAR.IL-15 T cells is safe, tolerable, and helpful in treating neuroblastoma or ganglioneuroblastoma.
    Location: UNC Lineberger Comprehensive Cancer Center, Chapel Hill, North Carolina

  • Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent / Refractory Solid Tumors

    This study will evaluate palbociclib in combination with chemotherapy (temozolomide and irinotecan) in children, adolescents and young adults with recurrent or refractory solid tumors. The main purpose of this study is to evaluate the safety of palbociclib in combination with chemotherapy in order to estimate the maximum tolerated dose. Pharmacokinetics and efficacy of palbociclib in combination with chemotherapy will be evaluated.
    Location: 5 locations

  • A Study of EGFR806 CAR T-Cell Immunotherapy for Treating Children and Young Adults with Recurrent / Refractory Solid Tumors

    This phase I trial studies side effects and best dose of EGFR806-specific and EGFR806xCD19 CAR T cells and how well they work in treating patients with solid tumors that have come back or do not respond to treatment. T cells are a type of immune cell that can recognize proteins, such as EGFR806 and CD19, on the surface of other cells. Some solid tumor cells have the EGFR protein on their surface, and T cells can be modified with a receptor, called a chimeric antigen receptor (CAR), to help recognize this protein and kill these cells. Researchers have also modified T cells to recognize a protein called CD19, which is present on the surface of a group of normal white blood cells called B cells. Modified T cells directed against CD19 may act as an additional signal to help the T cells directed against EGFR to grow and last longer. Giving EGFR806-specific and CD19 CAR T cells may work better in treating patients with malignant solid tumors.
    Location: Fred Hutch / University of Washington Cancer Consortium, Seattle, Washington

  • Dose Escalation Study of CLR 131 in Children and Adolescents With Relapsed or Refractory Malignant Tumors Including But Not Limited to Neuroblastoma, Rhabdomyosarcoma, Ewings Sarcoma, and Osteosarcoma

    The study evaluates CLR 131 in children and adolescents with relapsed or refractory malignant solid tumors and lymphoma and recurrent or refractory malignant brain tumors for which there are no standard treatment options with curative potential.
    Location: University of Wisconsin Hospital and Clinics, Madison, Wisconsin

  • Ex-Vivo Expanded and Activated Donor NK Cells and Hu14.18-IL2 in Treating Patients with Relapsed or Refractory Neuroblastoma

    This phase I trial studies the side effects of ex-vivo expanded and activated donor NK cells and hu14.18-IL2 in treating patients with neuroblastoma that has come back or does not respond to treatment. Expanded and activated donor NK cells may be able to kill the cancer cells better. Hu14.18-IL2 binds to NK cells and may be able to activate them, improving their ability to stay alive, multiply, and kill cancer cells. Giving ex-vivo expanded and activated donor NK cells and hu14.18-IL2 may work better in treating patients with neuroblastoma.
    Location: University of Wisconsin Hospital and Clinics, Madison, Wisconsin

  • Naxitamab, Irinotecan Hydrochloride, Temozolomide, and Sargramostim in Treating Patients with High-Risk Neuroblastoma

    This phase II pilot clinical trial studies the side effects of naxitamab, irinotecan hydrochloride, temozolomide, and sargramostim in treating patients with high-risk neuroblastoma. Immunotherapy with naxitamab, may induce changes in body’s immune system and may interfere with the ability of tumor cells to grow and spread. Drugs used in chemotherapy, such as irinotecan hydrochloride, temozolomide, and sargramostim, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving naxitamab, irinotecan hydrochloride, temozolomide, and sargramostim may work better in treating patients with high-risk neuroblastoma.
    Location: Memorial Sloan Kettering Cancer Center, New York, New York

  • Akt / ERK Inhibitor ONC201 in Treating Patients with Neuroendocrine Tumors That Are Locally Advanced, Metastatic, Recurrent, Refractory, or Cannot Be Removed by Surgery

    This phase II trial studies how well akt / ERK inhibitor ONC201 works in treating patients with neuroendocrine tumors that have spread to other places in the body, have come back, do not respond to treatment, or cannot be removed by surgery. Akt / ERK inhibitor ONC201 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
    Location: Case Comprehensive Cancer Center, Cleveland, Ohio

  • Humanized Monoclonal Antibody 3F8, Sargramostim, and Isotretinoin in Treating Patients with High-Risk Neuroblastoma in First Remission

    This phase II trial studies how well humanized monoclonal antibody 3F8, sargramostim, and isotretinoin work in treating patients with high-risk neuroblastoma in first remission. Monoclonal antibodies, such as humanized monoclonal antibody 3F8, may interfere with the ability of tumor cells to grow and spread. Biological therapies, such as sargramostim, use substances made from living organisms that may stimulate or suppress the immune system in different ways and stop tumor cells from growing. Drugs used in chemotherapy, such as isotretinoin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving humanized monoclonal antibody 3F8, sargramostim, and isotretinoin may work better in treating patients with neuroblastoma.
    Location: Memorial Sloan Kettering Cancer Center, New York, New York