ARST2531: A Randomized Phase 3 Study to Compare VAC (Higher Cyclophosphamide Dose and Intensity) versus VAC/VI (Lower Cyclophosphamide Dose and Intensity) Plus Maintenance Therapy in Patients with Newly Diagnosed Intermediate-Risk Rhabdomyosarcoma

What is the goal of the study?

Institutions in North American are currently split in terms what chemotherapy regimen is preferred for newly diagnosed children and young adults with rhabdomyosarcoma, with some using a backbone of VAC (1.2g/m2/cycle)/VI + VC maintenance and others opting for the significantly more alkylator dose intense VAC (2.2g/m2/cycle). Data from previous trials indicate that outcomes may be inferior with VAC/VI, but there are confounding factors that make retrospective comparisons challenging and there are notable implications for expected short- and long-term complications. Given that there are no novel agents available to test in this population, this randomized study will compare these two preferred backbones to confirm the preferred regimen for future standard of care. Primary aim is EFS. Secondary aims are OS, toxicity comparison, feasibility of molecular reporting on MCI, central review for DPE eligibility, and if DPE influences 4 year local failure rate. Exploratory aims include analyzing whether certain molecular factors influence outcome, analyze methylation patterns, gonadal toxicity, fertility counseling and fertility preservation, digital pathology, using patient derived biospecimens to create PDX models, and influence of household material hardship on outcomes. Definition of IR for eligibility will be similar to the previous generation of COG RMS studies. Patients up to age 50 yr old will be eligible. Local control guidelines on study reflect the current standard of care.

Who can participate in the study?

Please contact the study team listed below to learn more.

Study Team:

• PI (Study Doctor): Catherine Albert
• Study Team Member: Erin Thomas, [email protected]