Venetoclax in Pediatric and Young Adult Patients With Relapsed/Refractory Solid Tumors: Results of a Phase 1 Study

Key Points

• To evaluate the safety and efficacy of venetoclax in children and young adults with relapsed or refractory solid tumors.
• Conducted a Phase 1 study to determine the recommended dose of venetoclax.
• Used a dosing regimen of 800 mg/day over 21-day cycles.
• Combined venetoclax with Cy-Topo chemotherapy.
• Recommended dose of venetoclax determined to be 800 mg/day.
• The combination treatment showed a predictable safety profile.
• Clinical activity was modest in the studied cohorts.