Phase 2 Study of Cabozantinib to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors Ages 1.5 to 40

What this Trial Is About

Researchers are studying cabozantinib as a maintenance treatment to prevent progression or recurrence in children and young adults with ultra-high-risk pediatric solid tumors. These tumors include neuroblastoma, Ewing sarcoma, osteosarcoma, rhabdomyosarcoma, Wilms tumor, and other rare tumors that express targets of cabozantinib or show preclinical evidence of response. The study aims to improve progression-free survival and overall survival while maintaining quality of life in patients who have achieved their best response to prior therapy. Participants will receive cabozantinib tablets at a dose of 40 mgm2 per day, up to a maximum of 420 mg per week, given in 28-day cycles for up to one year. This approach targets the critical period within 12 months after last therapy when recurrence risk is highest. The study expands current research by evaluating cabozantinib in a wider range of pediatric solid tumors and testing its use as a maintenance therapy during remission to potentially extend disease control. Throughout the study, patients will be monitored for progression-free survival at one year as the primary outcome, with additional assessments of overall survival, duration of response, and safety over several years. Participants must meet strict eligibility criteria including age, performance status, organ function, and recovery from prior treatments. The study involves regular clinical evaluations, laboratory tests, imaging, and assessment of adverse events to ensure safety and measure treatment effects over time.

A Study of Cabozantinib as a Maintenance Agent to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors