NEUROBLASTOMA

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

This early-phase trial tests a new drug (rhIL-15) combined with three standard chemotherapy drugs for children and young adults (ages 3–35) whose neuroblastoma has returned or not responded to prior treatment. The main goal is to find a safe dose and check for side effects. Up to…

This study tests whether combining two drugs, DFMO and etoposide, can help 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 combination improves how long participants live without the cancer …

This study tests a new pill (tipifarnib) plus an approved IV drug (naxitamab) for children and adults with neuroblastoma that has come back or not responded to treatment. The goal is to see if the combination is safe and shrinks tumors or delays the cancer's return. About 98 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 study tests an experimental oral drug called AMXT 1501 combined with another drug, eflornithine (DFMO), in 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 ch…

This early-stage trial tests a treatment where a child's own immune cells are collected, modified to better attack cancer, and given back as a one-time infusion. It is for children and young adults up to age 21 with solid tumors that have come back or not responded to standard th…

This study tests a bivalent vaccine (with an immune booster) plus an oral supplement called β-glucan in 94 children with high-risk neuroblastoma that is in complete remission. The goal is to see which schedule of β-glucan helps the vaccine produce stronger antibodies to keep the …

This study tests whether adding special immune cells (NK cells) from a healthy donor to a standard chemo-immunotherapy regimen can help children and young adults with neuroblastoma that has come back or not responded to treatment. About 62 participants aged 1 to 31 will receive t…

This early-phase study tests a combination of two types of immune cells (B7-H3 CAR T cells and PRAME-specific T cells) in children with relapsed or treatment-resistant cancers like rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, and Wilms tumor. The main goal is to check safety a…

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

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. The goal is to help the donor cells attack the tumor without causing severe side effects. It is for children and young adults a…

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 a two-part approach for children newly diagnosed with high-risk neuroblastoma, a serious nerve cancer. First, doctors use genetic tests to pick personalized drugs during initial chemotherapy. Then, patients receive standard immunotherapy either alone or with an e…

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 offers expanded access to the drug DFMO for children and young adults (ages 0-30) with rare, relapsed tumors like neuroblastoma and medulloblastoma. The drug targets tumors with specific genetic markers (LIN28, MYCN, or ODC). The goal is to provide treatment when no ot…

This study tests proton beam radiation for children with neuroblastoma, a nerve cell cancer. Proton radiation targets the tumor more precisely than standard X-ray radiation, which may reduce damage to nearby healthy organs. Researchers will track side effects like heart, lung, an…

This study tests alectinib, a drug already approved for some lung cancers, in patients with rare cancers that have an ALK gene change. About 30 adults and children with ALK-positive cancers will receive alectinib to see if it shrinks tumors or stops them from growing. The goal is…

This early-phase study tests a four-drug combination (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 goals are to find a sa…

This study tests a two-step treatment for children and young adults with high-risk neuroblastoma. After initial chemotherapy, participants 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 see if…

This study tests whether adding a radioactive drug (131I-MIBG) or a targeted pill (lorlatinib) to standard intensive 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 diagn…

This study tests whether adding the drug dinutuximab to standard chemotherapy and other treatments helps children with high-risk neuroblastoma live longer without the cancer coming back. About 478 children and young adults up to age 30 with newly diagnosed high-risk neuroblastoma…

This early-phase study tests a new treatment for children whose neuroblastoma or osteosarcoma has come back or not responded to standard therapy. The treatment uses the patient's own T cells (a type of immune cell) that have been genetically modified to recognize and attack GD2, …

This study tests a combination of two drugs (apatinib and 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 this combo can shrink tumors and help children live longer. About 125 children will t…

This expanded access program offers a radioactive drug called 131 I-MIBG to children aged 1 year and older with neuroblastoma that has not responded to standard treatments or has returned. The drug delivers radiation directly to cancer cells to kill them. The goal is to provide t…

This early-phase study tests whether adding a chemotherapy drug (topotecan) given directly into the spinal fluid, followed by maintenance chemo, is safe and feasible for children under 6 with high-risk embryonal brain tumors. About 15 children will participate. The goal is to see…

This study tests whether a bivalent vaccine, combined with beta-glucan and GM-CSF, can help prevent high-risk neuroblastoma from coming back in children who are currently in complete remission. The treatment aims to strengthen the immune system to recognize and attack any remaini…

This study tests a chemotherapy plan called N10 for children with high-risk neuroblastoma, a serious nerve cell cancer. The goal is to see if it works well and is safe enough to use as a first treatment. About 45 children under 19 with newly diagnosed high-risk neuroblastoma will…

This study tests a drug called naxitamab, given with GM-CSF, for people with high-risk neuroblastoma that is still present in the bones or bone marrow after standard treatments. The goal is to see how many patients respond and how long the response lasts. Participants receive tre…

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 and delivers radiation directly to them. Researchers want to see if it can shrink tumors and how safe it …

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

This early-stage study tests a new type of stem cell transplant for children whose blood cancers or solid tumors have come back or are hard to treat. Doctors use a special method to remove certain immune cells from the donor's stem cells and give a drug called zoledronate after t…

This early-phase study tests 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 with manageable side effects. The drugs targe…

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

This study is for children with neuroblastoma or osteosarcoma that has come back or not responded to treatment. It tests a new approach: giving donor immune cells (gamma delta T cells) along with standard chemotherapy and an antibody drug. The main goal is to find the safest dose…

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 returning. About 441 participants will take the drug twice daily and be …

This study is for children newly diagnosed with high-risk neuroblastoma, a serious nerve cell cancer. Researchers are adding a drug called naxitamab to the usual chemotherapy to see if it helps more tumors shrink or disappear. About 93 children will take part, and the goal is to …

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 aims to see if special PET scans using different radioactive tracers can accurately find and stage neuroblastoma and pheochromocytoma/paraganglioma tumors. About 30 people aged 1 to 70 with suspected or confirmed disease will receive an injection of a tracer and then h…

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 Pediatric RISE) to usual care can reduce financial and material hardships for low-income families of children with high-risk neuroblastoma. About 84 families will be randomly assigned to get either usual care al…

This study aims to collect tumor and healthy tissue samples from up to 2,415 people scheduled for surgery or biopsy for endocrine tumors (such as thyroid, adrenal, or pancreatic tumors). Researchers will analyze the samples to find genetic and other molecular differences that cou…

This study aims to create immune organoids—tiny, lab-grown models of the immune system—from the cells of children and young adults with brain tumors, kidney tumors, neuroblastoma, or sarcoma. Researchers will use these organoids to study how tumors interact with immune cells. The…

This study watches how children and their caregivers recover after major cancer surgery. About 40 kids and their parents will wear smartwatches, answer surveys, and give extra blood samples for up to a year. The goal is to find patterns that show how well someone is bouncing back…

This study collects extra blood, urine, and tumor samples from children and adults with childhood cancers (like leukemia or sarcoma) to create a large tissue repository. Researchers will use these samples to study the genetic and molecular makeup of these cancers. The goal is to …

This research study aims to discover new genes that may cause cancer to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had childhood cancers. Participants will not receive direct treatment o…

This study aims to understand the genetic and biochemical makeup of neuroblastic tumors, which can affect how aggressive the cancer is and what treatments work best. Researchers will analyze tumor samples, blood, bone marrow, and stem cells from over 2000 participants. The goal i…

This study collects samples of tumor tissue, blood, and bone marrow from 1,000 children with high-risk neuroblastoma. Researchers aim to learn more about the cancer cells and find better ways to detect and track the disease. The goal is to improve future treatments and prevent th…

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 …