Ganglioneuroblastoma

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 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 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 a new treatment for children and young adults with neuroblastoma that has returned or not responded to standard care. The treatment uses specially engineered immune cells from donated cord blood that are designed to recognize and attack two different markers on t…

This study is testing a bone marrow transplant from a half-matched family donor for people with high-risk solid tumors that have come back or not responded to treatment. Participants receive mild chemotherapy and radiation before the transplant, along with drugs to prevent reject…

This phase 3 trial tests a powerful mix of chemotherapy, immunotherapy, and the child's own natural killer (NK) cells for children aged 1.5 to 18 with high-risk neuroblastoma that didn't respond or came back. The goal is to shrink tumors and improve survival by helping the immune…

Researchers are building an international registry to collect health information from people with congenital central hypoventilation syndrome (CCHS), a rare condition that affects breathing control. The study asks patients with a confirmed PHOX2B gene mutation to fill out surveys…