An Open-Label, Single-Arm, Phase 1/2 Study Evaluating the Safety and Efficacy of Ponatinib for the Treatment of Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors in Pediatric Participants

What this Trial Is About

Researchers are evaluating ponatinib, a drug being studied for children aged 1 to under 18 years who have advanced leukemias, lymphomas, or solid tumors. The study aims to assess the safety, tolerability, how the drug moves through the body, and its effectiveness in treating these conditions. This is an open-label, single-arm phase 1/2 trial focusing on pediatric participants with recurrent or refractory cancers. In the phase 1 part of the study, ponatinib is given in doses based on age groups to find the highest dose that can be tolerated and to recommend a dose for phase 2. In phase 2, ponatinib is given at the determined dose to further assess its effects. The drug is administered as tablets or formulations appropriate for children. Participants include those with chronic myeloid leukemia, other leukemias, lymphomas, and solid tumors, with specific criteria for prior treatments and genetic markers. Participants will be monitored through various assessments including response to treatment measured by blood and bone marrow tests, imaging scans, and side effects over several months. The primary outcomes include identifying dose-limiting toxicities and measuring responses like major cytogenetic or hematologic responses depending on the condition. Safety and drug behavior in the body are also tracked. The study duration and follow-up depend on the phase and participant response, with continuous evaluation of treatment effects and safety.

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