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

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 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 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 early-stage trial tests a personalized cell therapy for children with recurrent neuroblastoma or newly diagnosed DIPG, two aggressive cancers. The treatment uses the child's own immune cells (dendritic cells and T cells) and stem cells, trained to attack their specific tumor…

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 clinical trial tests a new type of immunotherapy called 4SCAR-T, which uses a patient's own immune cells engineered to recognize and attack three different markers (GD2, PSMA, and CD276) on neuroblastoma cancer cells. The study enrolls children and adults (ages 1 to 65) with…

This phase 1 trial tests an experimental drug, DXC006, in people with advanced solid tumors (like small cell lung cancer and neuroblastoma) or blood cancers (like multiple myeloma) who have not responded to standard treatments. DXC006 is an antibody-drug conjugate designed to del…

This phase 2 trial tests a new approach for children and young adults with relapsed or hard-to-treat neuroblastoma. Patients receive donor immune cells (NK cells) along with standard chemotherapy and an antibody therapy. The goal is to see if this combination can shrink tumors or…

This phase 2 trial tests whether specially trained donor immune cells, called CIML-NK cells, can be safely given to 20 children and young adults (ages 1-39) with relapsed or refractory neuroblastoma. The cells are made from a half-matched donor and infused alongside standard dinu…

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 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 Phase II trial tests a bivalent vaccine (with an immune booster) plus two different schedules of oral β-glucan in 94 people with high-risk neuroblastoma who are in complete remission. The goal is to see which schedule triggers stronger antibodies against the cancer, potentia…

This study tests a radioactive drug called I-MIBG in people with resistant neuroblastoma or related tumors (pheochromocytoma and paraganglioma). The drug is designed to deliver radiation directly to cancer cells. Researchers want to see if it can shrink tumors and how safe it is.…

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 early-stage trial tests a new type of cell therapy for children whose embryonal tumors (rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, or Wilms tumor) have returned or not responded to standard treatments. The therapy uses the patient's own immune cells, engineered to bette…

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 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 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 early-phase trial is testing a new type of immunotherapy called B7-H3 CAR T cells in children and young adults whose solid tumors (such as neuroblastoma, sarcoma, or osteosarcoma) have come back or not responded to standard treatments. The therapy involves taking a patient's…

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 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 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 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 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 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 expanded access program provides 131 I-MIBG therapy for children and young adults with refractory or relapsed neuroblastoma, a cancer that hasn't responded to standard treatments. The therapy uses a radioactive compound that is taken up by cancer cells, delivering radiation …

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-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 early-stage study tests whether giving children with high-risk solid tumors a special type of immune cell (called TAA-T) is safe. The cells are designed to recognize and attack proteins found on tumor cells. Up to 36 children whose cancer has not responded to standard treatm…

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 expanded access program provides the drug DFMO to children and young adults up to age 30 with rare relapsed tumors such as neuroblastoma or medulloblastoma. The goal is to control the disease by targeting specific genetic markers. Participants take DFMO by mouth twice daily,…

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 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 trial is testing whether giving children with advanced neuroblastoma or osteosarcoma a special type of immune cell (gamma delta T cells) from a healthy donor, along with several standard cancer drugs, is safe and tolerable. The study will enroll 24 children aged …

This trial tests whether hetrombopag, a drug that stimulates platelet production, can help children with neuroblastoma recover their platelets faster after a stem cell transplant. Thirty children will take the drug daily for up to 6 weeks. The main goal is to see if it increases …

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 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 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 type of stem cell transplant for children with blood cancers or solid tumors that have relapsed or not responded to standard treatment. Doctors use a half-matched donor's stem cells, specially filtered to remove certain immune cells, and give th…

This early-stage trial tests a new approach for children with neuroblastoma that has come back or not responded to standard treatment. Researchers take the child's own natural killer T cells, add genes to help them recognize and attack neuroblastoma cells, and give them back alon…

This study tests a new type of PET/CT scan using a radioactive tracer called 68Ga-DOTATATE to see if it can detect neuroblastoma tumors better than the current standard scan (123I-MIBG). The study will enroll 20 children and young adults with suspected or confirmed neuroblastoma.…

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 tests a special dye called anti-GD2-800CW that makes neuroblastoma cells glow during surgery. About 22 children with neuroblastoma will receive a single dose of the dye before their operation. The goal is to find the safest and most effective dose to help surgeons see …

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 study looks at two lab tests (RT-qPCR and ddPCR) to find leftover cancer cells in frozen ovarian or testicular tissue from children treated for neuroblastoma or Ewing sarcoma. The goal is to make it safer for these children to later use their stored tissue to have children o…

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 extra blood, urine, and tumor samples from children and adults with certain cancers to build a large tissue repository. Researchers will analyze these samples to learn more about the genetic and molecular makeup of these tumors. The goal is to support future s…

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 understand how cancer treatments like radiation, chemotherapy, and surgery affect health years later. By comparin…

This study enrolls people with endocrine tumors (such as thyroid, parathyroid, adrenal, or pancreatic tumors) who are scheduled for surgery or biopsy. Researchers collect samples of the tumor, nearby healthy tissue, blood, and urine to analyze genetic and molecular changes. The g…

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 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 how to detect it better. The goal is to improve future treatments and monitoring of the…

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 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 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…