NEUROBLASTOMA

This study tests a special stem cell transplant for children, teens, and young adults (ages 6 months to 25 years) with solid tumors that have come back or not responded to treatment. The transplant uses cells from a donor, with certain immune cells removed to reduce side effects,…

This phase II trial is testing whether adding the drug naxitamab (Danyelza) to standard chemotherapy improves outcomes for children newly diagnosed with high-risk neuroblastoma. About 93 participants will receive naxitamab during five cycles of induction chemo. The goal is to see…

This early-phase trial 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 the safest dose of vo…

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

This early-phase trial tests a new treatment made from a patient's own immune cells, engineered to better recognize and attack certain childhood solid tumors (rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, and Wilms tumor). The study enrolls up to 18 children and young adults wh…

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. The transplant uses a half-matched donor and removes certain immune cells to lower the risk of graft-versus-host disease. A drug ca…

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

This study tests a drug called zilovertamab vedotin in children and young adults whose blood cancers (like certain leukemias and lymphomas) or solid tumors (neuroblastoma, Ewing sarcoma) have come back or not responded to treatment. The goal is to see if the drug is safe and can …

This early-stage trial tests a new immunotherapy for children and young adults up to age 21 with solid tumors that have come back or not responded to standard treatment. The therapy uses the patient's own immune cells, which are modified in a lab to recognize and attack a protein…

This study tests a combination of drugs (liposomal mitoxantrone, capecitabine, and anlotinib) in children, adolescents, and young adults whose solid tumors have returned or not responded to previous treatments. The goal is to see if this regimen can shrink tumors or stop them fro…

This study tests two oral drugs, eflornithine (DFMO) and AMXT 1501, in children and young adults up to age 26 with certain cancers that have come back or not responded to treatment. The cancers include neuroblastoma, brain tumors, and sarcomas. The goal is to find a safe dose and…

This early-stage trial tests a new treatment called B7-H3CART, which uses a patient's own immune cells modified to target a protein (B7-H3) found on certain solid tumors. It includes up to 41 children and young adults with relapsed neuroblastoma, sarcoma, or osteosarcoma who have…

This phase 2 trial is testing whether a reduced chemotherapy regimen called N10 is safe and effective for children with high-risk neuroblastoma. The study will enroll 45 children under 19 years old who have not had more than one prior chemo cycle. The goal is to see if this light…

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 study tests a new pill called tipifarnib combined with an FDA-approved drug, naxitamab, for people with neuroblastoma that has come back or not responded to treatment. The goal is to see if the combination is safe and helps shrink tumors or slow the disease. The trial is for…

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

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

This study tests whether a bivalent vaccine, taken 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 remission. The treatment works by training the immune system to recognize and attack…

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 the tumors when other treatments have stopped working. Up to 28 adults will receive treatment for up to 12 months, an…

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 program offers the drug DFMO to children and young adults (up to age 30) with rare, relapsed tumors like neuroblastoma or medulloblastoma that have specific genetic markers. The goal is to control the disease and provide treatment access when standard options are limited. Pa…

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 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 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 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 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 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 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 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 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 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 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 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 study looks at two lab tests (RT-qPCR and ddPCR) to find leftover cancer cells in ovarian or testicular tissue that was frozen to preserve fertility in children with neuroblastoma or Ewing sarcoma. The goal is to see which test is better at detecting tiny amounts of cancer, …

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

This study is testing whether special PET scans using targeted tracers can accurately find and stage neuroblastoma and pheochromocytoma/paraganglioma tumors. About 30 people aged 1 to 70 will receive an injection of a radioactive tracer and then have a PET scan. The scan results …

This phase 3 study is testing an improved PET/CT imaging method using a radioactive tracer called 18F-DOPA. Researchers want to see if it can better detect small lesions and reduce image artifacts in patients with conditions like congenital hyperinsulinism, neuroblastoma, neuroen…

This study compares a new imaging technique (68Ga-DOTATATE PET/CT) with the standard scan (123I-MIBG) in 20 children with neuroblastoma, a common childhood cancer. The goal is to see if the new method is safe and provides clearer pictures to guide treatment decisions. Participant…

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 tissue, blood, and bone marrow samples from 1,000 children with high-risk neuroblastoma. Researchers will analyze these samples to learn more about how the cancer grows and returns, and to find better ways to detect and measure the disease. The goal is to impr…

This study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…

This study follows about 50,000 people who survived childhood cancer (diagnosed before age 21 between 1970 and 1999) and 10,000 of their siblings. Researchers want to learn how cancer treatments like radiation, chemotherapy, and surgery affect health years later. The goal is to b…

This study is testing whether smartwatches and blood tests can measure how well children recover after surgery to remove a solid tumor. About 40 children and their caregivers will wear smartwatches and answer questionnaires before surgery and for up to a year after. Researchers w…

This study looks at whether checking for pieces of tumor DNA in the blood (called ctDNA) can help doctors see how well treatment is working and find cancer returning early in children with neuroblastoma. About 200 children with newly diagnosed or returning neuroblastoma will give…

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 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 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…