Rhabdomyosarcoma

This phase 2 trial tests the drug cabozantinib in children and young adults with rare cancers like sarcomas, Wilms tumor, and others that have come back or not responded to treatment. Cabozantinib works by blocking enzymes that help tumors grow and form blood vessels. The study a…

This phase 3 trial tests whether adding the drug temsirolimus to standard chemotherapy helps children and young adults with intermediate-risk rhabdomyosarcoma, a type of muscle cancer. About 325 participants receive either standard chemo or standard chemo plus temsirolimus. The s…

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 study tests whether adding a drug called vinorelbine to standard chemotherapy can better control high-risk rhabdomyosarcoma, a rare and aggressive muscle cancer in children and young adults. About 118 participants will receive either the standard treatment or the new combina…

This early-phase trial tests a combination of two drugs—mocetinostat and vinorelbine—in children, adolescents, and young adults with rhabdomyosarcoma that has spread, cannot be surgically removed, or has returned after treatment. Mocetinostat works by blocking enzymes that help c…

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 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 personalized treatment plan for children with a rare muscle cancer called rhabdomyosarcoma. Depending on their risk level, patients receive standard or stronger chemotherapy, plus focused proton beam radiation and/or surgery to remove the tumor. The goal is to …

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 tested a combination of two chemotherapy drugs, irinotecan and carboplatin, as the first treatment for children and adults newly diagnosed with intermediate- or high-risk rhabdomyosarcoma, a type of muscle cancer. The goal was to see if this drug pair could shrink tumo…

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 study is testing a new way to choose chemotherapy for children with high-risk or relapsed soft tissue sarcomas. Doctors grow a miniature version of the patient's tumor in the lab (called an organoid) and test different drugs on it to see which works best. The goal is to see …

This early-stage trial tests a treatment made from a patient's own immune cells, called tumor-infiltrating lymphocytes (TIL). Doctors remove a piece of the tumor, grow the immune cells in a lab, and then infuse them back into the patient to attack the cancer. The study includes 4…

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 new drug called PEN-866, which stays in tumor cells longer than normal cells, combined with two standard chemotherapy drugs (vincristine and temozolomide). It is for people aged 12 to 39 with sarcomas (Ewing sarcoma or rhabdomyosarcoma) that have returned or no…

This early-phase trial tests a new experimental treatment called CATCH T cells in up to 12 adults with certain solid tumors (like liver cancer or Wilms tumor) that have returned or not responded to standard care. The cells are the patient's own T cells, genetically modified in th…

This study tests a precise radiation technique called image-guided radiotherapy (IGRT) for children and young adults with bone or soft tissue tumors. By using CT, MRI, and PET scans to pinpoint the tumor, doctors aim to deliver radiation more accurately and reduce damage to healt…

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 early-phase trial tests a new immunotherapy called AGAR T cells in children with solid tumors such as liver cancer, Wilms tumor, and rhabdomyosarcoma that have returned or not responded to standard care. The cells are made from the patient's own T cells, which are geneticall…

This study tests four different chemotherapy schedules in children and adults with a rare, aggressive muscle cancer that has spread. Participants choose from a first-strike approach, a first-strike followed by maintenance, an adaptive schedule, or standard chemo. The goal is to s…

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 tests a combination of two drugs, cobolimab and dostarlimab, in children and young adults (up to age 21) with advanced solid tumors that have not responded to other treatments. The goal is to find the safest dose and see if the combination can shrink tumors. The study …

This study investigates how well different imaging methods—ultrasound, CT, and MRI—can identify the cause of neck swellings in children. Researchers will compare imaging results with the final diagnosis from biopsy, lab tests, or follow-up. The goal is to see if imaging can relia…

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 whether a supervised strength and balance exercise program is feasible for children and teens undergoing chemotherapy for bone or soft tissue sarcoma in the lower leg. The goal is to see if this 'prehabilitation' can reduce muscle and bone weakening, and improve …

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 tests whether special MRI scans can measure oxygen levels inside children's sarcoma tumors. Low oxygen (hypoxia) can make tumors harder to treat. The scans are done before and after standard chemotherapy or radiation to see if they can predict how well the tumor respon…