Recurrent Childhood Rhabdomyosarcoma

This phase I/II trial tests the safety, best dose, and effectiveness of CBX-12 in the treatment of pediatric patients (ages 12 months to 30 years) with rhabdomyosarcoma, Ewing sarcoma, or other extracranial solid tumors that have come back after a period of improvement (relapsed) or that do not respond to treatment (refractory). CBX-12 is a conjugate drug made of a peptide linked to a drug called exatecan. Upon administration, the peptide portion of CBX-12 gets inserted into the membrane of tumor cells and then exatecan is released into the tumor cells. Exatecan inhibits deoxyribonucleic acid replication in tumors cells, leading to tumor cell death. CBX-12 may be a safe and effective treatment option for pediatric patients with relapsed or refractory rhabdomyosarcoma, Ewing sarcoma, or other solid tumors.

This phase I/II trial tests the safety, side effects, best dose and how well giving daraxonrasib works for the treatment of pediatric patients with solid tumors with RAS mutations that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). Daraxonrasib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving daraxonrasib may be safe, tolerable and/or effective in treating patients with relapsed or refractory solid tumors with RAS mutations.

Functional precision medicine (FPM) is a relatively new approach to cancer therapy based on direct exposure of patient- isolated tumor cells to clinically approved drugs and integrates ex vivo drug sensitivity testing (DST) and genomic profiling to determine the optimal individualized therapy for cancer patients. In this study, we will enroll relapsed or refractory pediatric cancer patients with tissue available for DST and genomic profiling from the South Florida area, which is 69% Hispanic and 18% Black. Tumor cells collected from tissue taken during routine biopsy or surgery will be tested.