Study of Arsenic Trioxide with Chemotherapy for Treating Children Up to 18 Years Old with p53-Mutated Cancers

What this Trial Is About

Researchers are evaluating the safety and effectiveness of arsenic trioxide combined with chemotherapy for treating pediatric cancer patients who have p53 mutations. These mutations are linked to Li-Fraumeni syndrome, a hereditary condition that greatly increases the risk of developing multiple cancers at a young age. Current treatments for this condition are limited and often involve DNA-damaging therapies that can lead to additional tumors. This study aims to explore a treatment that targets mutant p53 without causing DNA damage, which could offer new options for affected children. Participants will first receive their standard first-line chemotherapy for their specific cancer type. If the cancer does not respond or remains stable, arsenic trioxide will be added to their existing chemotherapy regimen starting on the third day of each treatment cycle. The arsenic trioxide is given intravenously at a dose based on body weight, over six hours daily for ten days during each cycle. This approach will be tested in a single-arm, multi-center trial that also includes laboratory experiments to confirm arsenic trioxides ability to restore p53 function in the patients specific mutation. During the study, participants will be closely monitored with regular assessments of their cancers response to treatment, including measurements of tumor size four weeks after starting arsenic trioxide combined therapy. Researchers will also track progression-free survival, overall survival, and any adverse events for up to three years. Guardians will provide informed consent, and patients must have measurable tumors and confirmed p53 mutations that respond to arsenic trioxide in lab tests. This careful monitoring aims to ensure safety and gather important data on the treatments impact over time.

Arsenic Trioxide Combined With Chemotherapy for the Treatment of p53-mutated Pediatric Cancer