ADVL1622 Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors

This open label two-stage phase 2 trial includes the following solid tumor strata: ewing sarcoma, rhabdomyosarcoma, non-rhabdomyosarcoma soft tissue sarcomas (nRSTS), Wilms tumor (non-statistical cohort) and other rare solid tumors (non-statistical cohort). XL184 will be administered orally once daily on a continuous dosing schedule of 28 day cycles at a dose of 40 mg/m2/day (cumulative weekly dose of 280 mg/m2 using a dosing nomogram). The primary endpoint for the selected disease strata will be the objective response rate. We will also evaluate progression free survival (PFS), time to progression, overall survival, and if feasible, compare PFS to historical controls. Pharmacokinetics and pharmacodynamics of XL184 in pediatric and adolescent patients will also be further studied.