Neuroblastoma

This study tests a drug called ulixertinib in children and teens whose advanced cancers have a specific genetic change in the MAPK pathway. The goal is to see if the drug can shrink or control the tumor. About 20 participants with various solid tumors, lymphomas, or related disor…

Researchers are testing whether natural killer (NK) cells from a universal donor can be safely given with standard chemotherapy drugs and an immune-targeting antibody to children and young adults with neuroblastoma that has returned or stopped responding. The NK cells are modifie…

This early-stage trial tests a combination of three drugs—lenalidomide, dinutuximab, and isotretinoin—in children whose neuroblastoma has not responded to treatment or has come back. The main goal is to find the safest dose of lenalidomide when used with the other drugs. Research…

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 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. The trial involves 441 participants and focuses on safety a…

This study follows 165 children who previously took dabrafenib and/or trametinib for certain brain tumors. Researchers want to see if these drugs are safe over the long term and how they affect growth. The children continue their treatment and are monitored for side effects and d…

This study tests a personalized approach for children with non-high-risk neuroblastoma, where treatment is guided by the tumor's biology and how it responds over time. Some children may only need monitoring instead of immediate therapy, potentially avoiding harsh side effects. Th…

This early-stage trial tests an experimental drug called LY3295668 erbumine in children whose neuroblastoma has returned or not responded to treatment. The study aims to find a safe dose and see if the drug can shrink tumors, either alone or with standard chemotherapy. About 71 p…

This study is testing a type of radiation called proton beam therapy for children with neuroblastoma, a nerve cell cancer. Unlike standard radiation, proton therapy targets the tumor more precisely, which may spare nearby healthy organs and reduce side effects. The study will enr…

This early-stage trial is testing a new treatment called GPC2 CAR T cells for children with neuroblastoma or retinoblastoma that has come back or not responded to standard treatments. The therapy uses a patient's own immune cells, which are modified in a lab to better recognize a…

This pilot study is testing a new, shortened chemotherapy regimen called N9 for children with high-risk neuroblastoma, a type of cancer. The regimen combines several chemotherapy drugs given in different cycles to see if it is safe and effective. The study includes children under…

This phase 1 trial is testing a new treatment called GD2CART for children and young adults with osteosarcoma or neuroblastoma that has returned or does not respond to standard treatments. GD2CART is made by taking a patient's own T cells (a type of immune cell), adding a new gene…

This phase 2 trial tests the drug selpercatinib in children and young adults (ages 1 to 21) with advanced cancers that have a specific genetic change called a RET alteration. The goal is to see if the drug can shrink tumors or stop them from growing. Only one participant has been…

This early-phase study tests a flavored, drinkable version of the chemotherapy drug irinotecan (VAL-413) combined with another drug (temozolomide) in children and young adults up to age 30 whose solid tumors have come back. The main goal is to find the safest dose and see how the…

This phase 2 trial tests a combination of two drugs—naxitamab (an antibody) and GM-CSF (an immune booster)—in children and adults with high-risk neuroblastoma that has not responded well to previous treatments. The goal is to see if this therapy can shrink or eliminate tumors in …

This study tests a drug called tipifarnib in children and young adults (ages 1 to 21) whose cancers have a specific change in the HRAS gene. The cancers are advanced or have come back after treatment. The goal is to see if tipifarnib can shrink tumors or stop them from growing. A…

This study tested the drug ensartinib in 13 children and young adults (ages 1–21) whose cancers had come back or did not respond to treatment and had specific ALK or ROS1 gene changes. The goal was to see if the drug could shrink or stop tumor growth. While the drug targets cance…

This study tests a new type of immunotherapy for people with cancers that have returned or not responded to treatment, such as neuroblastoma, sarcoma, and certain other solid tumors. The treatment uses a patient's own T cells, which are genetically modified to recognize a protein…

This study tests whether adding an experimental antibody called Hu3F8 to a standard chemotherapy regimen (irinotecan and temozolomide) plus a immune-boosting drug (GM-CSF) is safe and effective for children with high-risk neuroblastoma. The trial enrolls patients whose neuroblast…

This early-stage trial is testing a new type of cell therapy for children with neuroblastoma that has returned or is hard to treat. The therapy uses the patient's own immune cells, which are modified in the lab to recognize and attack a protein called PHOX2B found on neuroblastom…

This phase 2 trial tested the drug ivosidenib in children and young adults (ages 1 to 21) with solid tumors, lymphomas, or histiocytic disorders that have an IDH1 mutation and have either come back after treatment or not responded. The goal was to see if the drug could shrink or …

This study tests a drug called entrectinib in children with advanced solid tumors or brain tumors that have come back or haven't responded to other treatments. The trial has two parts: first finding the best dose, then seeing how well it works in children whose tumors have specif…

This study tests a drug called erdafitinib in children and young adults (ages 1 to 21) whose cancers have come back or are not responding to treatment and have specific changes in FGFR genes. The drug works by blocking enzymes that help cancer cells grow. The main goal is to see …

This phase 2 trial is testing the drug larotrectinib in children and young adults (ages 1 to 21) with advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have a specific genetic change called an NTRK fusion. The cancers have either come back after treatment…

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 study was designed to test two immunotherapy drugs, nivolumab and ipilimumab, in children and young adults with cancers that returned or didn't respond to treatment and had many genetic mutations. The goal was to see if the drug combination was safe and could shrink tumors. …

This study tests a tumor vaccine combined with daily low-dose chemotherapy for children whose high-risk neuroblastoma has returned or not responded to standard treatment. The vaccine is designed to train the immune system to recognize and attack neuroblastoma cells, while the low…

This early-stage trial tests a new approach for children and young adults with neuroblastoma that has come back or not responded to standard treatments. Doctors take a patient's own immune cells (T-cells), genetically modify them in the lab to recognize and attack a protein calle…

This early-phase study tests a personalized cell therapy for children and young adults (up to age 26) with solid tumors that have come back or not responded to standard treatment. The therapy uses the patient's own immune cells, modified to recognize and attack a protein called B…

This early-phase trial tests a combination of three treatments—radioactive iodine (131-I-MIBG), an immunotherapy drug (nivolumab), and an antibody (dinutuximab beta)—in children whose high-risk neuroblastoma has come back or not responded to standard therapy. The main goal is to …

This early-stage trial tests a new treatment called GD2 CAR T cells in 12 children and teens (ages 1-16) with neuroblastoma that has come back or not responded to standard therapy. The treatment involves taking a patient's own immune cells, modifying them to better recognize and …

This early-phase study tests a new treatment for advanced osteosarcoma and neuroblastoma that has not responded to standard therapy. Researchers take a patient's own immune cells (T cells), add a gene to help them recognize and attack cancer cells, and combine them with a chicken…

This early-phase trial tests a new type of immune therapy for children with neuroblastoma that has come back or not responded to treatment. Researchers take a patient's own T cells, add new genes to help them recognize and kill neuroblastoma cells and survive longer, then infuse …

This early-phase trial tests whether giving children with relapsed neuroblastoma their own expanded natural killer (NK) cells along with the drug dinutuximab is safe and feasible. Some children may also receive lenalidomide. The study involves 13 participants aged up to 30 years …

This early-stage trial tests a combination of three drugs—sorafenib, cyclophosphamide, and topotecan—in children and young adults with neuroblastoma that has relapsed or not responded to treatment. The main goal is to find the highest safe dose of sorafenib when given with the tw…

This early-stage trial tests a radioactive drug called CLR 131 in 30 children and young adults with solid tumors, lymphoma, or brain tumors that have come back or not responded to standard treatments. The main goals are to find a safe dose and see if the drug can shrink tumors. P…

This early-phase trial tests a new type of cell therapy for children and young adults with solid tumors that have come back or not responded to standard treatments. The therapy uses a patient's own immune cells, which are trained in the lab to recognize and attack multiple cancer…

This study tests a daily combination of sirolimus, celecoxib, and low-dose chemotherapy in 46 children whose solid or brain tumors have returned or not responded to standard treatment. The goal is to see if this approach can shrink tumors or stop them from growing. While not a cu…

This study tests whether adding eflornithine (DFMO) to a standard chemotherapy and immunotherapy regimen can improve outcomes for children with neuroblastoma that has returned or is not responding to treatment. About 94 children will be randomly assigned to receive the standard t…

This study tests a combination of two drugs—an antibody called Hu3F8 and a growth factor called GM-CSF—in children with high-risk neuroblastoma that has come back or not responded to standard therapy. The goal is to find the safest dose and understand side effects. About 186 part…

This study tests whether the drug eflornithine, taken as a pill after immunotherapy, can help prevent high-risk neuroblastoma from coming back in children. Twenty children under 18 who have completed standard treatment will take eflornithine for maintenance. Researchers will trac…

This early-stage trial tests a new type of immunotherapy for children and young adults with solid tumors that have not responded to standard treatments. The therapy uses the patient's own immune cells (T cells) that are genetically modified to recognize and attack cancer cells ca…

This study tested whether a fecal transplant (transferring healthy gut bacteria from a donor) could help treat neuroblastoma, a rare childhood cancer. The approach aimed to fix gut bacteria imbalances before and during standard cancer treatment. Only one patient was enrolled, and…

This study tests whether adding two chemotherapy drugs (irinotecan and temozolomide) to the standard immunotherapy regimen (dinutuximab, GM-CSF, isotretinoin) can safely prevent high-risk neuroblastoma from returning after intensive initial therapy. About 41 children and young ad…

This study tests whether a new type of PET scan (18F-MFBG) can detect neuroblastoma tumors as accurately as the current standard scan (123I-MIBG). About 84 children and adults with newly diagnosed, high-risk neuroblastoma will receive both scans to compare results. If the new sca…

This study tests whether a special PET scan using a radioactive form of methionine (a natural amino acid) can help doctors see tumors in children and young adults with various cancers, including brain tumors and sarcomas. About 503 participants will be scanned to see if the metho…

This study tests a new radioactive tracer called 18F-DA for PET scans to see if it can find neuroblastoma and pheochromocytoma tumors more clearly than the current tracer. About 20 children and adults with known or suspected tumors will receive the tracer and be monitored for sid…

This study compares a new ultra-high resolution PET/CT scanner, the NeuroEXPLORER, with standard clinical PET/CT scanners for imaging the head and neck. Researchers aim to see if the new device provides more detailed images to improve diagnosis of conditions like dementia, Parkin…

This study tests whether a new type of PET/CT scan, using a radioactive tracer called 68Ga-DOTATOC, can find neuroendocrine tumors (NETs) and similar tumors more safely and accurately than current imaging methods. About 800 people with known or suspected NETs will receive the sca…

This study involves 20 children and young adults with neuroendocrine tumors (like neuroblastoma) that have spread. Researchers are testing whether a special PET/CT scan using a radioactive tracer called 68Ga-DOTATATE can find tumors better than the standard MIBG scan. The goal is…

This study is testing whether whole body MRI scans can help find cancers early in children and adults with Li-Fraumeni syndrome, a genetic condition that greatly raises cancer risk. About 150 participants will receive annual whole body MRI scans for four years to see if they keep…

This study tests whether sodium thiosulfate can prevent hearing loss in children and young adults (ages 1 month to 39 years) who are receiving cisplatin chemotherapy for relapsed or hard-to-treat liver tumors and other embryonal cancers. Some participants also get vorinostat. The…

This study tests if giving the drug dinutuximab over 4 hours or less is safe and practical for children with high-risk neuroblastoma. Currently, standard infusions take 10-20 hours and require a hospital stay. The goal is to see if a shorter infusion can be used in an outpatient …

This study looks at how parents make treatment decisions when their child has relapsed neuroblastoma, a type of cancer. Researchers want to understand if parents' choices match their true preferences, and whether they feel regret or burden over time. By following 96 parents, the …

This study screens children and teens with advanced solid tumors, lymphomas, or histiocytic disorders to find genetic changes in their cancer cells. Participants undergo biopsies and scans to identify mutations that can be targeted by specific drugs. The goal is to see if matchin…

This study looks at whether it's possible to screen for long-term health problems in people who survived high-risk neuroblastoma. Researchers will evaluate survivors who finished treatment at least 2 years ago, using tests for heart, nerve, and other health issues. The goal is to…

This study looks at tumor samples from children and young adults (ages 1 to 30) with high-risk neuroblastoma that has come back or not responded to treatment. The goal is to find specific genetic changes and immune system markers that could guide future treatments. No new therapy…

This research study follows 376 survivors of high-risk neuroblastoma to see what long-term side effects they experience from their past treatments. By collecting blood, urine, and quality-of-life data, researchers hope to learn which therapies cause fewer late health problems lik…

This pilot study is testing a radioactive antibody called 124I-Humanized 3F8 to see how it travels through the body and attaches to tumors in people with melanoma, neuroblastoma, or sarcoma. Seven participants will receive the antibody and then have PET/CT scans at several time p…

This study searches for new gene changes (mutations) that appear in cancer patients but not in their healthy parents. By comparing DNA from 1,275 people with certain cancers and their families, researchers hope to find genetic clues that increase cancer risk. The goal is to bette…