Ponatinib Treatment Study for Children Ages 1 to 17 With Recurrent or Resistant Leukemias, Lymphomas, or Solid Tumors

What this Trial Is About

Researchers are evaluating ponatinib for children aged 1 to under 18 years who have advanced leukemias, lymphomas, or solid tumors that are recurrent or resistant to previous treatments. This study aims to assess the safety, tolerability, how the drug moves through the body, and the effectiveness of ponatinib in treating these serious cancers in a pediatric population. The study has two phases. In Phase 1, ponatinib is given in tablet or age-appropriate formulations according to age-based dosing groups to find the maximum tolerated dose and recommend a dose for Phase 2. In Phase 2, participants receive ponatinib at the recommended dose to evaluate its effectiveness. The treatment is given orally with dosing adjusted based on age and disease type. Participants will be closely monitored through various assessments including bone marrow tests to detect genetic markers, imaging scans to measure tumor size, and evaluations of blood and molecular responses. Researchers will track side effects and drug levels in the body over months. The study measures outcomes such as major response rates and survival indicators, with follow-up continuing for up to 12 months to assess the treatment impact and safety in children.

Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors