Neuroblastoma Clinical Trials

This laboratory study is collecting and storing tissue, blood, and bone marrow samples from young patients with cancer. Collecting and storing samples of tissue, blood, and bone marrow from patients with cancer to study in the laboratory may help doctors learn more about changes that may occur in DNA and identify biomarkers related to cancer. Read Less

The aim of this study is to evaluate the safety and efficacy of 67Cu-SARTATE in pediatric patients with high-risk neuroblastoma. Read Less

The goal of the Molecular Characterization Trial (MCT) is to obtain biological specimens and data resources from patients enrolled on prospective trials, to ensure that the Harvard/UCSF ROBIN Center accomplishes its key objective of advancing our understanding of the biological mechanisms that underlie how radiation treats tumors but also can cause unwanted side effects. The MCT focuses on collection of research biospecimens before, during, and after radiation. Also critical to the MCT is the deep annotation of these research biospecimens with elements that complement each other to provide a holistic, detailed view of each patient. Annotated elements include those used in the past such as clinical and biological features but extend to factors we have so far neglected but must incorporate in the future such as dosimetry (precise anatomical measurement of radiation dose), artificial intelligence, computational biology, and natural language processing. Read Less

This trial studies how well an investigational scan called 68Ga-DOTATATE PET/CT works in diagnosing pediatric patients with neuroendocrine tumors that have spread to other places in the body (metastatic). A neuroendocrine tumor is an abnormal growth of neuroendocrine cells, which are cells resembling nerve cells and hormone-producing cells. 68Ga-DOTATATE is a radioactive substance called a radiotracer that when used with PET/CT scans, may work better than standard of care MIBG scans in diagnosing pediatric metastatic neuroendocrine tumors and targeting them with radiation therapy. Read Less

High risk neuroblastoma is an aggressive and often lethal pediatric solid tumor. Survival remains less than 50% and those patients who do survive suffer many treatment-related acute and chronic toxicities. Chemoimmunotherapy using a combination of an anti-GD2 monoclonal antibody (dinutuximab) and different chemotherapy agents in the relapsed/refractory (r/r) neuroblastoma population, has demonstrated the most robust response rates to date, shifting the clinical practice to administer chemoimmunotherapy as a standard treatment for patients with r/r neuroblastoma. The goal of this study is to improve upon GD2 chemoimmunotherapy regimens for neuroblastoma by delivering standard drugs like temozolomide, irinotecan, and dinutuximab in combination with a novel cell-based immunotherapy called gamma delta (γδ) T cells in addition to zoledronate that enhances γδ T cell activation and potency. γδ T cells are an innovative approach to cell therapy for neuroblastoma as they are major histocompatibility complex (MHC) independent and directly cytotoxic to tumor cells without the need for engineering them to recognize the tumor. The study team has developed a Good Manufacturing Practice (GMP)-compliant manufacturing strategy to expand safe γδ T cells from healthy donors for this trial. This is a Phase 1 study to determine the safety, recommended phase 2 dose, and preliminary efficacy of allogeneic (third party), ex vivo expanded γδ T cells in combination with dinutuximab, temozolomide, irinotecan and zoledronate in children with refractory, relapsed, or progressive neuroblastoma. Read Less

This phase I trial studies the safety of transplantation with a haploidentical donor peripheral blood stem cell graft depleted of TCRαβ+ cells and CD19+ cells in conjunction with the immunomodulating drug, Zoledronate, given in the post-transplant period to treat pediatric patients with relapsed or refractory hematologic malignancies or high risk solid tumors. Read Less

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. The goals of this study are: To find the highest safe dose of vorinostat that can be given together with vincristine, irinotecan, and temozolomide without causing severe side effects; To learn what kind of side effects this four drug combination can cause; To learn about the effects of vorinostat and the combination of vorinostat, vincristine, irinotecan, and temozolomide on specific molecules in tumor cells; To determine whether the combination of vorinosat, vincristine, irinotecan, and temozolomide is a beneficial treatment. Read Less

The purpose of this study is to find out whether avutometinib is a safe treatment for advanced or recurrent solid tumor cancers in children and young adults. Researchers will look for the highest dose of avutometinib that is safe and cause few or mild side effects. Read Less

This research study combines two different ways of fighting cancer: antibodies and Natural Killer T cells (NKT). Antibodies are types of proteins that protect the body from infectious diseases and possibly cancer. T cells, also called T lymphocytes, are special white 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. Investigators have found from previous research that they can put a new gene into T cells that will make them recognize cancer cells and kill them. In a previous clinical trial, investigators made artificial genes called a chimeric antigen receptors (CAR), from an antibody called 14g2a that recognizes GD2, a molecule found on almost all neuroblastoma cells (GD2-CAR). Investigators put these genes into the patients' own T cells and gave them back to patients that had neuroblastoma. NKT cells are another special subgroup of white blood cells that can specifically go into tumor tissue of neuroblastoma. Inside the tumor, there are other white blood cells called macrophages which help the cancer cells to grow and recover from injury. NKT cells can specifically kill these macrophages and slow the tumor growth. We will expand NKT cells and add GD2-specific chimeric antigen receptors to the cells. We think these cells might be better able to attack NB since they also work by destroying the macrophages that allows the tumor to grow. The chimeric antigen receptor will also contain a gene segment to make the NKT cells last longer. This gene segment is called CD28. In addition, to further improve the antitumor activity of the GINAKIT cells we added another gene expressing a molecule called Interleukin -15 (IL-15). The combination of these 3 components showed the most antitumor activity by CAR expressing NKT cells and improved these cells' survival in animal models. GD2-CAR expressing NKTs have not been tested in patients so far. The purpose of this study is to find the largest effective and safe dose of GD2-CAR NKT cells (GINAKIT cells), to evaluate their effect on the tumor and how long they can be detected in the patient's blood and what affect they have on the patient's neuroblastoma. Read Less

This is a phase II single center study to administer two courses of myeloablative consolidation chemotherapy each followed by an autologous peripheral blood stem cell (PBSC) rescue in patients with high-risk neuroblastoma who have completed induction chemotherapy (independent of this study). Ideally, patients should begin consolidation chemotherapy no later than 8 weeks after the start of Induction Cycle #5; however it is strongly recommended to begin consolidation within 4-6 weeks after the start of Induction Cycle #5. Read Less

Studies have shown that the anti-GD2 human-mouse chimeric monoclonal antibody dinutuximab has contributed significantly to the improvement of treatment for children with high-risk neuroblastoma and has become a mainstay in treating high risk neuroblastoma in children as part of up-front therapy and relapsed/refractory therapy. The administration of dinutuximab requires a significant amount of time and resources to complete the 10-20 hour standard infusion time for 4 days in the inpatient setting. During its early development, a phase I study profiling the clinical efficacy and tolerability of dinutuximab infusions in children successfully infused dinutuximab at various rates including over 1 hour at different dose levels. In the adult setting, dinutuximab has been tolerated over substantially shorter infusion times (less than 2 hours). Additionally, another anti-GD2 murine monoclonal antibody naxitamab, which has a similar toxicity profile to dinutuximab, is FDA approved for administration over 90 minutes and is successfully administered in outpatient setting. Given this reassuring data the investigators aim to evaluate the feasibility of the rapid administration of dinutuximab over four hours or less in our patient population of children with high-risk neuroblastoma. The pharmacokinetics, toxicity profile and supportive care requirements will be analyzed and described in order to determine if rapid infusion of dinutuximab can be successfully tolerated over four hours or less which would allow for administration of this agent in the outpatient setting. Should this trial prove to be successful, it would serve to decrease the hospital burden in a positive way by allowing for administration of this immunotherapy agent in the outpatient setting and patients may prefer shorter infusion duration. Furthermore, it could lessen overall costs and inpatient admissions for patients. Read Less

Medical scientists want to find better ways to treat neuroblastoma and to find ways to prevent the tumor from growing back. To do this, they need more information about the characteristics of neuroblastoma cells. Therefore, they want to study samples of neuroblastoma tissues and neuroblastoma and normal cells in the blood and bone marrow that may be related to the growth of neuroblastoma cells. Doctors and other medical scientists also want to find better ways to detect and measure neuroblastoma to improve the ability to follow the response of tumor cells to therapy. Read Less