NEUROBLASTOMA

This study tests whether adding the drug dinutuximab to standard chemotherapy and other treatments helps children with high-risk neuroblastoma live longer without their cancer returning. About 478 children with newly diagnosed high-risk neuroblastoma will be randomly assigned to …

This study tests a two-step treatment for children and young adults with high-risk neuroblastoma. After initial chemotherapy, patients receive two rounds of very strong chemo, each followed by an infusion of their own stem cells to help the body recover. The goal is to keep the c…

This study tests a new treatment for children with neuroblastoma that has come back or not responded to standard therapy. Researchers take the child's own immune cells (called NKT cells), add a special receptor to help them recognize and attack the cancer, and also add a molecule…

This study tests a drug called tegavivint in children, teens, and young adults (ages 1 to 30) whose solid tumors have returned or not responded to standard therapy. The drug works by blocking a key signal that tells cancer cells to grow. The trial aims to find the safest dose, un…

This early-phase study tests a new treatment for children whose neuroblastoma or osteosarcoma has not responded to standard therapy or has returned. The approach combines specially grown immune cells (gamma delta T cells) from a healthy donor with several chemotherapy drugs and a…

This study tests a radioactive drug called I-MIBG in people with neuroblastoma or related tumors that have not responded to standard treatments. The drug targets cancer cells directly to deliver radiation. Researchers want to see if it can shrink tumors and how safe it is. About …

This study tests whether adding the drug naxitamab to standard chemotherapy helps children with newly diagnosed high-risk neuroblastoma. About 93 children will receive naxitamab during their first 5 rounds of chemo. The goal is to see if this combination leads to more complete re…

This study tests a combination of a bivalent vaccine, a sugar called beta-glucan, and a protein (GM-CSF) to help the immune system fight high-risk neuroblastoma that is in complete remission. About 286 participants will receive the vaccine and beta-glucan, with some also getting …

This study tests a drug called alectinib, already approved for some lung cancers, in people with other rare cancers that have a specific gene change (ALK). About 30 adults, teens, and children will receive the drug to see if it shrinks tumors or stops cancer growth for at least 2…

This study tests an oral drug called DFMO in children and young adults with high-risk neuroblastoma who are currently in remission. The goal is to see if taking DFMO for two years can prevent the cancer from coming back. About 441 participants will take the drug twice daily and b…

This study is testing a precise type of radiation called proton beam therapy for children with neuroblastoma, a nerve cell cancer. The goal is to see if this treatment can control the disease while causing fewer long-term side effects than standard radiation. About 30 participant…

This study tests a mix of four drugs (vorinostat, vincristine, irinotecan, and temozolomide) in children, teens, and young adults up to age 30 whose solid tumors or brain cancers have returned or not responded to standard treatment. The main goal is to find the safest dose of vor…

This study tests a stem cell transplant that removes certain immune cells (alpha/beta T cells and CD19+ B cells) and adds a drug called zoledronic acid to help the donor cells fight solid tumors that have come back or not responded to treatment. It includes children and young adu…

This early-stage trial tests a new treatment using the body's own immune cells (T cells) that are specially trained to attack cancer. It is for children with certain cancers (rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, or Wilms tumor) that have come back or not responded to s…

This study tests whether the drug hetrombopag can help children with neuroblastoma recover their platelets more quickly after a stem cell transplant. About 30 children will take the drug daily for up to 6 weeks. The main goal is to see if it reduces the need for platelet transfus…

This early-phase trial tests a new drug (rhIL-15) combined with three standard cancer drugs for children and young adults (ages 3 to 35) with neuroblastoma that returned or didn't respond to prior treatment. The goal is to find a safe dose and see if the combination shrinks tumor…

This study tests a combination of two drugs (apatinib and an IT regimen) in children aged 5 to 18 with neuroblastoma that has come back or not responded to standard treatment. The goal is to see if the combination can shrink tumors and help children live longer. About 125 childre…

This study tests a drug called naxitamab, given with GM-CSF, in children and adults with high-risk neuroblastoma that hasn't fully responded to standard treatments. The goal is to see if this combination can shrink or eliminate tumors in the bone or bone marrow. Participants rece…

This expanded access program provides 131 I-MIBG therapy to children aged 1 year and older with neuroblastoma that has not responded to standard treatments or has returned. MIBG is a substance that carries radioactive iodine directly to cancer cells, killing them with minimal har…

This study tests a combination of two drugs, DFMO and etoposide, in children and young adults (up to age 30) whose neuroblastoma has returned or not responded to standard treatment. The goal is to see if the combo can help them live longer without the cancer getting worse. About …

This study tests a reduced chemotherapy plan called N10 for children with high-risk neuroblastoma. The goal is to see if it works well and is safe enough to shrink tumors early. About 45 children under 19 with newly diagnosed high-risk neuroblastoma will take part.

This study tests a new pill (tipifarnib) combined with an approved IV drug (naxitamab) for people with relapsed or hard-to-treat neuroblastoma. The goal is to see if the combination is safe and shrinks tumors or delays cancer growth. About 98 participants aged 1 to 21 at diagnosi…

This study tests whether adding a radioactive drug (131I-MIBG) or a targeted pill (lorlatinib) to standard therapy helps children with high-risk neuroblastoma live longer without the cancer coming back. About 750 children and young adults (ages 1 to 30) with newly diagnosed high-…

This early-phase study tests a new treatment for children whose neuroblastoma or osteosarcoma has returned or not responded to standard therapy. The treatment uses the patient's own T-cells, which are modified in a lab to better recognize and attack cancer cells. The main goals a…

This study tests a new oral drug, AMXT 1501, combined with another drug called DFMO for children and young adults (up to age 21) with certain cancers like neuroblastoma, brain tumors, and sarcomas. The first part finds the safest dose, and the second part checks if the combo help…

This early-stage study tests a new type of stem cell transplant for children whose blood cancer or solid tumor has returned or is hard to treat. Doctors use a special method to remove certain immune cells from the donor's stem cells, then give a drug called zoledronate after tran…

This early-stage study is testing a combination of two drugs, avutometinib and defactinib, in children and young adults (ages 3 to 30) with solid tumors that have returned or not responded to treatment. The main goal is to find a safe dose and check for side effects. The study is…

This study is for children with high-risk neuroblastoma whose cancer is in complete remission. It tests a bivalent vaccine (with an immune booster called OPT-821) plus an oral supplement called β-glucan to see which schedule of β-glucan works best to strengthen the immune system …

This study tests a new treatment for children and young adults with neuroblastoma that has come back or not responded to standard therapy. It combines chemotherapy drugs (temozolomide, irinotecan) with an antibody (dinutuximab) and special immune cells called NK cells from a heal…

This study tests an experimental pill called silmitasertib combined with standard chemotherapy in children and young adults (under 30) whose solid tumors (like neuroblastoma or sarcoma) have come back or not responded to treatment. The goal is to find a safe dose and see if the c…

This study is for children newly diagnosed with high-risk neuroblastoma, a serious nerve tissue cancer. It first uses genetic tests to pick targeted drugs for each child's tumor during initial chemotherapy. Then, in a second phase, children are randomly assigned to receive standa…

This study offers expanded access to the drug DFMO for children and young adults (ages 0-30) with rare relapsed tumors like neuroblastoma or medulloblastoma that have certain genetic markers. The goal is to provide treatment when no other options are available, aiming to control …

This early-stage trial tests a personalized cell therapy for children and young adults (up to age 21) with solid tumors that have come back or not responded to standard treatment. Doctors take the patient's own immune cells, modify them to recognize a protein called B7-H3 on tumo…

This study tests the drug nivolumab in people with advanced rare cancers that have a specific marker (PD-L1). The goal is to see if the drug can shrink or control tumors for at least 12 months. About 28 adults who have not responded to standard treatments will take part.

This study tests a new drug called zilovertamab vedotin in children and young adults (up to age 30) whose blood cancers or solid tumors have come back or not responded to treatment. The goal is to see if the drug is safe and can shrink or control the cancer. About 90 participants…

This early-phase study tests whether adding a chemotherapy drug (topotecan) given into the spinal fluid, followed by maintenance chemo, is safe and feasible for children under 6 with high-risk embryonal brain tumors. The goal is to improve cancer control after standard high-dose …

This study tests capmatinib, a drug already approved for certain lung cancers, in adults with other rare cancers that have specific MET gene changes. About 30 participants will receive the drug to see if their tumors shrink or stop growing for at least 24 weeks. The goal is to fi…

This study tests whether special PET scans using targeted tracers can more accurately find and stage neuroblastoma and pheochromocytoma/paraganglioma tumors than current methods. About 30 people aged 1 to 70 will receive an injection of a radioactive tracer and then have a PET sc…

This study is testing a special PET scan (18F-DOPA) to see if it can better detect and measure several conditions, including congenital hyperinsulinism in children, neuroblastoma, neuroendocrine tumors, Parkinson's disease, Lewy body dementia, and brain tumors. About 800 children…

This study tests a special dye that makes neuroblastoma tumors glow during surgery, helping surgeons remove them more completely. About 22 children and teens aged 1 to 18 will receive the dye before their planned operation. The goal is to find the safest and most effective dose f…

This study looks at whether adding a special support program (called RISE) to usual care can reduce money-related stress for low-income families whose child has high-risk neuroblastoma. About 84 children and their parents will take part, with some getting the extra support and ot…

This study aims to create immune organoids—miniature lab-grown models of the immune system—using cells from children and young adults with brain tumors, kidney tumors, neuroblastoma, or sarcoma. Researchers will take blood or tissue samples and turn them into stem cells, then gro…

This study aims to collect tumor and healthy tissue samples from up to 2,415 people scheduled for surgery or biopsy for endocrine tumors (thyroid, parathyroid, adrenal, pancreas, or neuroendocrine). Researchers will analyze the samples to find genetic and molecular differences th…

This study collects extra blood, tissue, and urine samples from children and adults with childhood cancers like leukemia and brain tumors. The goal is to create a large repository for researchers to study the biology of these cancers. No extra procedures are needed—samples are ta…

This study watches how children and their caregivers recover after major cancer surgery using smartwatches and questionnaires. It also looks at blood proteins and metabolites linked to stress. The goal is to find patterns that show how well a child is bouncing back, so doctors ca…

This study collects samples of tumor tissue, blood, and bone marrow from children with high-risk neuroblastoma. Researchers will analyze these samples to learn more about how the cancer grows and how to better detect and treat it. The goal is to find ways to prevent the tumor fro…

This study aims to understand the genetic and biochemical makeup of neuroblastic tumors, which are a type of childhood cancer. By analyzing tumor samples and blood from over 2000 participants, researchers hope to link these molecular features with how the disease behaves and resp…

This research study aims to discover new genes that may increase the risk of childhood cancer. Researchers will collect blood samples and health information from 1,500 people in families where cancer clusters. The goal is to build a registry and sample bank to help future diagnos…

This study looks at children with neuroblastoma or Ewing sarcoma who had ovarian or testicular tissue frozen to preserve their fertility before cancer treatment. The problem is that this tissue might contain hidden cancer cells that could cause the cancer to return if the tissue …