Phase II Study of Ulixertinib in Children and Young Adults With Tumors Harboring Activating Mitogen-Activated Protein Kinase Pathway Alterations: APEC1621J of the National Cancer Institute-Children’s Oncology Group Pediatric MATCH Trial
Summary
The National Cancer Institute-Children’s Oncology Group (NCI-COG) Pediatric MATCH trial tested ulixertinib, a targeted therapy, in patients aged 1-21 with refractory malignancies harboring genetic alterations in the mitogen-activated protein kinase pathway. The study aimed to determine the recommended phase II dose (RP2D) and assess treatment response, safety, and progression-free survival.
Key Findings
– Ulixertinib was administered at 260 mg/m2/dose orally twice a day and was found to be the RP2D for pediatric patients.
– The study included 20 patients with various tumor types, with CNS tumors being the most common.
– No objective responses were observed, but some patients with BRAF-altered CNS tumors achieved stable disease for over 6 months.
– The six-month progression-free survival rate was 37%.
– Limited single-agent efficacy was observed in this cohort of refractory pediatric tumors.
Conclusion
Ulixertinib, a novel targeted agent with no previous pediatric data, was successfully evaluated in a national precision medicine basket trial. The study provides valuable insights into the use of ulixertinib in pediatric patients with refractory tumors.
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