Current Research Studies

ARST2032: A Prospective Phase 3 Study of Patients with Newly Diagnosed Very Low-risk and Low-risk Fusion Negative Rhabdomyosarcoma


What is the goal of the study?

Patients with low-risk rhabdomyosarcoma (LR-RMS) have excellent outcomes with 4-year event free survival (EFS) rates of approximately 90% when treated on recent North American trials (D9602 and ARST0331). Molecular prognostic features have recently been described for fusion negative RMS but have yet to be incorporated into a prospective clinical trial. By reclassifying patients with LR-RMS who have adverse molecular features (TP53 or MYOD1 mutations), the LR-RMS group may be enriched with patients with a more favorable prognosis. This study will redefine patients with LR-RMS into two subgroups: 1) very low-risk (VLR): Patients with fusion negative (FN), Stage 1/CG I disease with MYOD1/TP53 wildtype (WT) tumors, and 2) low-risk (LR): Patients with Stage 1/CG II, or Stage 2/CG I/II or CG III (orbit only) disease with MYOD1/TP53 WT tumors. Patients with VLR-RMS will be treated with 24 weeks of VA therapy, representing both a reduction in the duration of therapy compared to that used in D9602 as well as an elimination of any alkylating agent as used in ARST0331. Patients with LR-RMS will be treated with 12 weeks of VAC followed by 12 weeks of VA with the goal of achieving superior outcomes in this now enriched population (MYOD1/TP53 WT). For patients with clinically defined VLR or LR disease whose tumors harbor pathogenic MYOD1 or TP53 mutations, therapy will be escalated to 42 weeks of VAC therapy.

Who can participate in the study?

Please contact the study team listed below to learn more.

Study Team: