Embryonal Rhabdomyosarcoma

Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. This phase III trial aims to maintain excellent outcomes in patients with very low risk rhabdomyosarcoma (VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine and dactinomycin (VA) and examines the use of centralized molecular risk stratification in the treatment of rhabdomyosarcoma. Another aim of the study it to find out how well patients with low risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment. Finally, this study examines the effect of therapy intensification in patients who have RMS cancer with DNA mutations to see if their outcomes can be improved.

Pleuropulmonary blastoma (PPB) is a rare malignant neoplasm of the lung presenting in early childhood. Type I PPB is a purely cystic lesion, Type II is a partially cystic, partially solid tumor, Type III is a completely solid tumor. Treatment of children with PPB is at the discretion of the treating institution. This study builds off of the 2009 study and will also seek to enroll individuals with DICER1-associated conditions, some of whom may present only with the DICER1 gene mutation, which will help the Registry understand how these tumors and conditions develop, their clinical course and the most effective treatments.

Data from 40 patients are planned to be included in the study to randomize approximately 10 patients. This study involves patients with advanced/metastatic GD2-positive rhabdomyosarcoma after progression on 1st line of chemotherapy, advanced/metastatic GD2-positive Ewing sarcoma after progression on 1st line of chemotherapy and advanced/metastatic GD2-positive osteosarcoma after progression on 1st line of chemotherapy. All patients will receive therapy until disease progression (a total of 6 courses of immunotherapy). The primary objective of the study is to determine the efficacy and safety of dinutuximab beta with investigator's choice of chemotherapy.