NEUROBLASTOMA

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

This study tests whether giving two rounds of very strong chemotherapy, each followed by a stem cell transplant, can help children and young adults with high-risk neuroblastoma stay cancer-free longer. Participants must have already completed initial chemotherapy and have enough …

This early-phase study tests a personalized cell therapy for children and young adults up to age 21 with solid tumors that have returned or not responded to standard care. The therapy uses the patient's own immune T cells, which are genetically modified to recognize and attack a …

This study tests a new pill (tipifarnib) plus an approved IV drug (naxitamab) for children and young 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. About 98 participants will take part.

This study is for children with high-risk neuroblastoma who are currently 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 helps the body make more antibodies again…

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 natural killer T cells, add a special receptor to help them find and attack cancer cells, and give them back along with a drug ca…

This study tests whether adding a radioactive drug (131I-MIBG) or a targeted pill (lorlatinib) to standard treatment helps children with high-risk neuroblastoma live longer without the cancer coming back. About 750 children and young adults up to age 30 will take part. The goal i…

This study tests whether the drug hetrombopag can help children with neuroblastoma rebuild their platelet levels faster after a stem cell transplant. About 30 children will take the drug daily for up to 6 weeks. The goal is to reduce the need for platelet transfusions and improve…

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 tests a new treatment for children and young adults (ages 1 to 31) with high-risk neuroblastoma that has come back or not responded to standard therapy. The treatment combines chemotherapy drugs (temozolomide and irinotecan) with an immune-boosting drug (sargramostim),…

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

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 and young adults up to age 30 will be randomly assigned to receive either …

This early-phase trial tests a new drug (rhIL-15) combined with three standard cancer drugs for children and young adults (ages 3–35) whose neuroblastoma did not respond to treatment or returned. The main goal is to find a safe dose and check for side effects. Up to 40 participan…

This study tests the drug nivolumab in people with rare cancers that have spread and have not responded to standard treatments. It includes over 40 different rare tumor types, as long as the cancer cells have a specific marker called PD-L1. The goal is to see if nivolumab can shr…

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 this reduced treatment can safely shrink tumors and improve outcomes. About 45 children under 19 with newly diagnosed high-risk neuroblast…

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 five chemo cycles. The goal is to see if this combination leads to more complete re…

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 it can reduce side effects like heart, lung, and growth problems compared to standard radiation. About 30 participants will rec…

This early-stage trial tests a new treatment for children whose solid tumors (rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, or Wilms tumor) have come back or not responded to standard therapy. The treatment uses the patient's own immune cells, which are modified in the lab to b…

This study tests a new drug called Becotatug Vedotin in children aged 2 to 18 with certain solid tumors that have come back or spread. The drug targets a protein called EGFR found on many cancer cells. The main goal is to see if the drug is safe and to find the best dose, while a…

This study tests whether combining two drugs, DFMO and etoposide, can help children and young adults (up to age 30) whose neuroblastoma has come back or not responded to standard treatment. About 131 participants will receive the drug combination to see if it delays cancer growth…

This study tests an experimental oral drug called AMXT 1501 combined with eflornithine (DFMO) in children and young adults up to age 21 with neuroblastoma, brain tumors, or sarcomas. The goal is to find the best dose, check safety, and see if the combination helps keep the cancer…

This expanded access program provides 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 program also colle…

This early-phase study tests a four-drug combination (vorinostat plus three chemotherapy drugs) 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 a safe dose and und…

This study tests a drug called alectinib, already approved for some lung cancers, in people with rare cancers that have a specific genetic change called ALK. About 30 adults, teens, and children with ALK-positive cancers will receive alectinib to see if it shrinks tumors or stops…

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 more effectively while reducing side effects. It is for children and young…

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

This early-stage study tests a new type of stem cell transplant for children whose blood cancers or solid tumors have returned 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 tr…

This study is for children newly diagnosed with high-risk neuroblastoma, a rare and aggressive nerve cancer. It uses genetic testing to choose personalized chemotherapy, then tests whether adding the drug DFMO to standard immunotherapy helps prevent relapse. About 500 children wi…

This expanded access program provides the drug DFMO to children and young adults (up to age 30) with rare, relapsed tumors like neuroblastoma and medulloblastoma. The goal is to control the disease by targeting specific genetic markers such as MYCN or LIN28. Participants must hav…

This study tests a drug called tegavivint in children, teens, and young adults whose solid tumors have come back or stopped responding to treatment. The goal is to find the safest dose and see if it can shrink tumors by blocking a key growth signal inside cancer cells. About 147 …

This study tests a radioactive drug called I-MIBG in people with neuroblastoma (a nerve cancer) or pheochromocytoma/paraganglioma (adrenal tumors) that have not responded to standard treatments. The drug seeks out and delivers radiation directly to cancer cells. The goal is to se…

This study tests an experimental drug called zilovertamab vedotin in children and young adults (ages 1 to <18) whose blood cancers (like B-cell acute lymphoblastic leukemia, diffuse large B-cell lymphoma, or Burkitt lymphoma) or solid tumors (neuroblastoma or Ewing sarcoma) have …

This early-stage study tests a combination of two drugs, avutometinib and defactinib, in children and young adults (ages 3 to 30) with advanced or recurrent solid tumors that have specific genetic changes. The main goal is to find the safest dose with the fewest side effects. The…

This early-phase study tests whether adding chemotherapy directly into the spinal fluid (intrathecal topotecan) plus maintenance pills is feasible for children under 6 with high-risk embryonal brain tumors. About 15 children will receive this treatment after standard high-dose ch…

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, which are modified in a lab to better recognize and attack cancer cells. The main goals …

This study tests whether a special vaccine, combined with a sugar called beta-glucan and a protein (GM-CSF), can help prevent high-risk neuroblastoma from returning in children who are currently in complete remission. The treatment aims to train the immune system to recognize and…

This study tests a drug called apatinib combined with two chemotherapy drugs (irinotecan and temozolomide) in children aged 5 to 18 with neuroblastoma that has returned or not improved after standard treatment. The goal is to see if the combination can shrink tumors and help chil…

This early-phase study tests a new treatment called B7-H3 CAR T cells for children and young adults whose solid tumors (like neuroblastoma or sarcoma) have come back or not responded to standard therapies. The treatment involves taking a patient's own immune cells, modifying them…

This study tests a special PET scan using 18F-DOPA to improve how doctors see and measure certain tumors and brain conditions. It includes 800 children and adults with congenital hyperinsulinism, neuroblastoma, neuroendocrine tumors, Parkinson's disease, Lewy body dementia, or br…

This study tests a new imaging technique (68Ga-DOTATATE PET/CT) for children with neuroblastoma, a common childhood tumor. It compares this method to the standard 123I-MIBG scan to see which better detects tumors. The goal is to improve staging and treatment decisions. About 20 c…

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

This study tests whether special PET scans using targeted tracers can more accurately detect and stage neuroblastoma and pheochromocytoma/paraganglioma tumors in people aged 1 to 70. Participants receive an injection of a radioactive tracer, then undergo imaging to find primary a…

This study looks at whether adding a special support program (called RISE) to usual care can reduce financial and material hardships for low-income families of children with high-risk neuroblastoma. About 84 children and their parents will take part, with half getting the extra s…

This study collects 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 between b…

This study looks at how to safely preserve fertility in children with neuroblastoma or Ewing sarcoma. When ovarian or testicular tissue is frozen before cancer treatment, there is a risk that cancer cells could be put back into the body later. Researchers will compare two lab met…

This study aims to collect and store extra blood, tissue, urine, and tumor samples from children and adults diagnosed with childhood cancers like leukemia, sarcoma, and neuroblastoma. The samples are taken only when already needed for medical care, so no extra procedures are requ…

This research study aims to discover new genes that may cause cancer to run in families. It will enroll up to 1,500 people from families where multiple members have had childhood cancers or related conditions. Participants provide blood samples and health information, which are s…

This study watches how children and their caregivers recover after major cancer surgery. About 40 kids and their guardians will wear smartwatches, answer surveys, and give extra blood samples for up to a year. The goal is to find patterns in activity, sleep, and blood markers tha…

This study aims to understand the genetic and biochemical makeup of neuroblastic tumors in children. By analyzing tumor samples and blood, researchers hope to learn how these tumors grow and spread. The information gathered will help doctors choose the most effective treatments f…

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

This study collects samples of tumor tissue, blood, and bone marrow from 1,000 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 tum…

This study looks at whether a blood test can help doctors track neuroblastoma, a type of childhood cancer. Researchers will collect blood samples from 200 patients over time to look for tiny bits of tumor DNA. The goal is to see if changes in this DNA match how the disease is res…