The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ponatinib in children aged 1 to \< 18 years with advanced leukemias, lymphomas, and solid tumors.
Karnofsky performance status ≥ 40% for participants ≥ 16 years old or Lansky Play Scale ≥ 40% for pediatric participants < 16 years old.
Participants must have recovered to < Grade 2 per the NCI CTCAE v5.0 or to baseline from any non-hematologic toxicities (except alopecia) due to previous therapy.
Willingness to avoid pregnancy or fathering children.
Immunosuppressive therapy (including post stem cell transplant regimens) within 14 days before the first dose of ponatinib.
Any targeted cancer therapy (including TKIs) within 7 days before the first dose of ponatinib.
Any other investigational anticancer agents within 30 days or 5 half-lives, whichever is longer, before randomization.
Any monoclonal antibody-directed anticancer therapy within 5 half-lives of the first dose of ponatinib.
Single group
Ponatinib
DrugPonatinib administered as a tablet or age-appropriate formulation for pediatric participants according to age-based cohort assignment.
Phase 1: Number of dose-limiting toxicities
Defined as the occurrence of any protocol-defined toxicities occurring after dosing and up to and including Day 28, except those toxicities with a clear alternative explanation.
Phase 2: Efficacy of ponatinib assessed by major cytogenetic response (MCyR) in participants with chronic-phase chronic myeloid leukemia (CP-CML)
Defined as complete cytogenetic response (CCyR) or partial cytogenetic response (PCyR) as assessed by conventional cytogenetics or fluorescence in situ hybridization (FISH).
Phase 2: Efficacy of ponatinib assessed by major hematologic response (MaHR) or major molecular response (MMR) in participants with BCR-ABL-positive leukemias
Assessed by polymerase chain reaction (PCR).
Phase 2: Efficacy of ponatinib assessed by complete response (CR) in participants with leukemias other than BCR-ABL-positive leukemias to determine the efficacy of ponatinib
Phase 1: Number of treatment-emergent adverse events
Phase 1: Tmax of ponatinib
Time to maximum concentration.
Phase 1: AUCss,0-24 of ponatinib
Area under the steady-state plasma or serum concentration-time curve from Hour 0 to 24.
Phase 1: t½ of ponatinib
Apparent terminal-phase disposition half-life.
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