NEUROBLASTOMA

This study is testing whether adding specially prepared, donated immune cells can improve a standard treatment for neuroblastoma that has returned or not responded to prior therapy. The trial is for children and young adults (ages 1-31) and combines these 'natural killer' (NK) ce…

This study is testing a new cell therapy for children with neuroblastoma that has come back or hasn't responded to other treatments. Doctors take a patient's own immune cells, modify them in a lab to better find and attack cancer cells, and then infuse them back into the patient …

This study is testing the safety of a new two-drug combination (avutometinib and defactinib) for children and young adults (ages 3-30) whose solid tumors have come back or stopped responding to standard treatments. The main goal is to find the highest dose that is safe and causes…

This study is testing a new, intensive chemotherapy plan for children under 6 with high-risk brain tumors. The main goal is to see if adding a specific spinal fluid chemotherapy and a long period of oral 'maintenance' drugs is a workable treatment plan. Researchers will also trac…

This study is testing a type of radiation called proton beam therapy for children and young adults with neuroblastoma, a nerve cell cancer. The goal is to see if proton radiation can target the tumor more precisely than standard radiation, potentially reducing damage to nearby he…

This trial is testing if a drug called alectinib, already approved for a type of lung cancer, can help treat other rare cancers that share the same genetic change (ALK mutation). It will involve about 30 adults and children with various rare or hard-to-treat cancers. The goal is …

This trial is testing if a drug called capmatinib, already used for some lung cancers, can help adults with rare or uncommon cancers that have specific changes in a gene called MET. The goal is to see if the drug shrinks tumors or provides long-term benefit for these hard-to-trea…

This trial is testing if adding a new drug called naxitamab to the standard first round of chemotherapy can better control a serious childhood cancer called high-risk neuroblastoma. It will involve about 93 children and young adults up to age 21 who are newly diagnosed. The goal …

This is a first-in-human safety study testing a new type of donor immune cell therapy for children whose neuroblastoma or osteosarcoma has come back or hasn't responded to standard treatments. The main goal is to find the safest dose of these cells when given alongside a combinat…

This study is testing whether a combination of a vaccine, an oral sugar supplement (beta-glucan), and an immune-stimulating protein (GM-CSF) can help prevent cancer from coming back in people with high-risk neuroblastoma who are currently in remission. The treatment aims to train…

This study is testing if adding a drug called dinutuximab to the first stage of treatment helps children with newly diagnosed high-risk neuroblastoma. Dinutuximab is an immunotherapy that helps the body's own immune system find and kill cancer cells. The trial will compare the cu…

This study is testing a new, reduced chemotherapy plan called N10 for children with high-risk neuroblastoma, a serious childhood cancer. The goal is to see if this less intense treatment is still effective and safe for patients under 19 years old. Researchers want to find out if …

This trial is testing whether a targeted antibody drug called naxitamab, given with another medication called GM-CSF, can help control high-risk neuroblastoma in children and adults whose cancer hasn't responded well to previous treatments. About 122 patients with cancer in their…

This early-stage study is testing a new, flavored, oral version of a chemotherapy drug called irinotecan (named Orotecan®) in children and young adults whose solid tumors have come back. The main goals are to find a safe and effective dose and to see how the body processes the dr…

This study is testing a new, unapproved pill called silmitasertib, given alongside standard chemotherapy drugs. It aims to find a safe and effective dose for children and young adults under 30 whose solid tumors (like neuroblastoma or sarcoma) have come back or stopped responding…

This study is testing a targeted radiation treatment for children and young adults whose neuroblastoma or related tumors have not responded to standard therapies. The treatment uses a radioactive drug that seeks out and delivers radiation directly to cancer cells. Researchers aim…

This study is testing a new type of targeted radiation therapy for children whose neuroblastoma has come back or hasn't responded to standard treatments. The main goal is to find the highest dose that is safe for children to receive. Up to 18 children will receive two treatments,…

This study is testing if a drug called hetrombopag can help children with neuroblastoma recover their platelet counts faster after a stem cell transplant. Faster platelet recovery could mean fewer transfusions and a smoother recovery process. About 30 children under 18 will take …

This study is testing whether adding a drug called apatinib to standard chemotherapy can help children whose neuroblastoma has returned or hasn't responded to previous treatments. Researchers want to see if this combination can shrink tumors and help children live longer. The tri…

This early-stage study is testing a new type of personalized cell therapy for children and young adults whose neuroblastoma or osteosarcoma has come back or hasn't responded to standard treatments. Doctors take a patient's own immune cells (T cells), modify them in a lab to bette…

This study is testing a new oral drug, AMXT 1501, combined with an existing drug called DFMO, for children and young adults with aggressive cancers that have come back or didn't respond to standard treatments. The first part aims to find a safe dose, and the second part will comp…

This study is testing a two-part, high-dose chemotherapy treatment followed by two separate stem cell transplants for children and young adults with high-risk neuroblastoma. The goal is to better control the cancer and prevent it from coming back after initial treatment. Research…

This study is testing a two-drug combination (DFMO and etoposide) for children and young adults whose neuroblastoma has come back (relapsed) or hasn't responded to standard treatments (refractory). The goal is to see if this treatment can help patients live longer without their c…

This study is testing a two-part treatment to prevent high-risk neuroblastoma from returning in patients who are currently in remission. It combines a vaccine designed to train the immune system to attack cancer cells with an oral supplement (β-glucan) that may boost the immune r…

This study is testing a new drug called tegavivint in children, teens, and young adults whose solid tumors or lymphomas have come back or haven't responded to other treatments. The main goals are to find the safest dose, understand the side effects, and see if the drug can help s…

This study is testing a new combination of two drugs for children and young adults whose high-risk neuroblastoma has returned or not responded to standard treatments. It combines an experimental pill called tipifarnib with an approved IV drug, naxitamab. The main goals are to see…

This study is testing the safety of a new type of stem cell transplant for children whose cancers have come back or are very hard to treat. Doctors use a half-matched family donor and a special process to remove certain immune cells from the transplant, aiming to reduce complicat…

This is a first-in-human study testing a new type of personalized cell therapy for children and young adults (up to age 21) whose solid tumors have come back or haven't responded to standard treatments. Doctors will collect a patient's own immune cells (T cells), genetically modi…

This study is testing a new drug, rhIL-15, combined with three standard cancer drugs, to see if it is safe and can help control neuroblastoma. It is for children and young adults (ages 3-35) whose cancer has come back or hasn't responded to other treatments. The main goal is to f…

This large, late-stage study is testing if adding one of two new treatments to the current intensive therapy helps children with newly diagnosed, high-risk neuroblastoma. One approach uses a radioactive drug that targets the tumor. The other uses a daily pill (lorlatinib) for chi…

This trial provides expanded access to an experimental drug called DFMO for children and young adults (ages 0-30) with rare, relapsed cancers like neuroblastoma and medulloblastoma. The goal is to see if DFMO can help control these aggressive cancers after standard treatments hav…

This study is testing a new drug called zilovertamab vedotin in children and young adults whose cancers have come back or haven't responded to standard treatments. It will enroll about 90 participants aged 1 year and older with specific blood cancers or solid tumors. The main goa…

This is a first-in-human safety study for a new, two-part cell therapy for children and young adults whose cancers have come back or haven't responded to standard treatments. Doctors will collect a patient's own immune cells, genetically modify them to better target the cancer, a…

This study is testing a personalized treatment approach for children and young adults with high-risk neuroblastoma, an aggressive childhood cancer. It first uses genetic testing to select targeted drugs alongside standard chemotherapy, then tests whether adding an experimental dr…

This study is testing a new type of stem cell transplant for children and young adults whose solid tumors have returned or not responded to standard treatments. Doctors are using a donor's stem cells that have been specially processed to remove certain immune cells, then combinin…

This study is testing new types of imaging scans to better find and map rare nerve and adrenal tumors (neuroblastoma and pheochromocytoma/paraganglioma). Researchers will give participants special tracer injections and use PET or SPECT scans to see if these new methods can spot t…

This study is testing an improved version of a special PET scan called 18F-DOPA. The goal is to see if using a newer scanner and a common medication (furosemide) can make the images clearer, especially in the lower abdomen. This could help doctors better detect and plan treatment…

This study is testing whether a pill called DFMO can prevent neuroblastoma, a serious childhood cancer, from coming back in patients who are currently in remission. It will involve 441 children and young adults considered high-risk, who will take the pill daily for two years. The…

This study is testing whether adding a special support program to usual care helps low-income families whose children have high-risk neuroblastoma. About 84 families will be randomly assigned to receive either the usual care or the usual care plus the new support program for 6 mo…

This study aims to collect and store blood, bone marrow, and tumor samples from children with high-risk neuroblastoma. Researchers will use these samples to learn more about the cancer's characteristics and to grow cells in the lab for future testing. The goal is to discover bett…

This study aims to discover new genes that cause cancer to run in families, especially those affecting children. Researchers will collect health information, blood, and sometimes leftover tumor samples from families where multiple members have had cancer. The goal is to build a l…

This study aims to understand how children and their families recover from the stress of major cancer surgery. Researchers will have children and a caregiver wear smartwatches, answer questionnaires, and provide extra blood samples during routine draws to look for patterns linked…

This study aims to understand the genetic and biological characteristics of neuroblastoma tumors and how they relate to a patient's outcome. Researchers will analyze tumor, blood, and bone marrow samples from over 2,000 participants to identify genetic markers. The goal is to lea…

This study aims to make fertility preservation safer for children with neuroblastoma or Ewing's sarcoma. Before cancer treatment, children can have ovary or testicle tissue frozen to protect future fertility, but there's a risk the frozen tissue contains hidden cancer cells. Rese…

This study aims to create 3D models called 'immune organoids' from children's own cells to better understand how pediatric cancers interact with the immune system. Researchers will use tissue samples from up to 108 patients aged 25 or younger with brain tumors, kidney tumors, neu…

This study aims to collect and store blood, tumor, and tissue samples from children and adults with cancer to create a large research repository. The goal is to help scientists better understand cancer biology by studying these samples in the lab. Participants provide samples onl…

This study aims to collect and analyze tissue samples from people undergoing surgery for endocrine tumors (like thyroid, adrenal, or pancreatic tumors). Researchers will study the genetic and molecular makeup of these tumors to learn how to better tell benign from cancerous growt…