REFRACTORY NEUROBLASTOMA

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 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 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 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 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 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…

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 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 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 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 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…