evolutionary therapy for rhabdomyosarcoma

Conditions: Rhabdomyosarcoma

Summary: this clinical trial will evaluate 4 different strategies of chemotherapy schedules in newly diagnosed participants with metastatic fusion positive (alveolar) rhabdomyosarcoma. the participant and their physician will choose from: arm a) a first strike therapy, arm b) a first strike-second strike (maintenance) therapy, arm c) an adaptively timed therapy, and arm d) conventional chemotherapy.


Eligibility criteria:
inclusion criteria: * participants must have a new histologic diagnosis of rhabdomyosarcoma * participants must have fish, pcr or other molecular confirmation of pax/foxo1 fusion per institutional standards * participants must have sufficient tissue (up to 10 unstained ffpe) for correlative testing * all participants must have distant metastatic disease; either biopsy positive or pet avid extranodal or distant nodal lesions determined by the investigator to be metastatic disease. patients with a single distant metastatic site that has been excised prior to study entry are eligible * no prior systemic chemotherapy * participants enrolled to arm b, maintenance, must be able to take oral cyclophosphamide. note: enteral administration of cyclophosphamide is allowable. * males and females of reproductive potential may not participate unless they have agreed to the use of, at minimum, two methods of contraception during and after treatment or abstinence. * women of childbearing potential should adhere to contraception for a period of 4 months after completion of systematic chemotherapy administration * men who are sexually active with women of child bearing potential should adhere to contraception for a period of 4 months after completion of systematic chemotherapy administration * all patients and/or their parents or legal guardians must have the ability to understand and the willingness to sign a written informed consent or assent document. exclusion criteria: * participants with regional lymph nodes as the only site of disease are not eligible. distant nodal sites alone are eligible * participants who are receiving any other investigational agents for rhabdomyosarcoma are ineligible * participants must not be receiving any additional medicines being given for the specific purpose of treating cancer. alternative medications including, but not limited to cannabis based products would not be a reason for exclusion * participants are ineligible if they have uncontrolled intercurrent illness including, but not limited to: * ongoing or active infection not expected to resolve with current antibiotic plan * cardiac arrhythmia * psychiatric illness/social situations that would limit compliance with study requirements * patients who are pregnant or breastfeeding are not eligible because there is no available information regarding human fetal or teratogenic toxicities. females of childbearing potential must have a negative serum or urine pregnancy test within 24 hours of starting protocol therapy. * participants who are considered unable to comply with the safety monitoring requirements of the study are not eligible

Keywords: soft tissue cancer skeletal muscle tissue cancer sarcoma