A Phase II Study Evaluating a Range of Doses of Ponatinib for Resistant Chronic Myelogenous Leukemia

Full Title

A Randomized, Open-label, Phase 2 Trial of Ponatinib in Patients with Resistant Chronic Phase Chronic Myeloid Leukemia to Characterize the Efficacy and Safety of a Range of Doses


Ponatinib is a drug used to treat chronic myelogenous leukemia (CML) that is still growing despite treatment with similar drugs such as imatinib, nilotinib, dasatinib, or bosutinib (drugs called tyrosine kinase inhibitors, or TKIs). However, the standard ponatinib dose of 45 mg can cause side effects such as narrowed or blocked blood vessels (“vascular occlusive events” and related symptoms or problems) in some patients.

In this study, researchers are comparing the daily dosages of 45 mg, 30 mg, and 15 mg of ponatinib in patients with resistant CML to see which dose achieves the best response without causing significant side effects. Patients will be randomly assigned to a particular dosage group. Ponatinib is taken orally (by mouth).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have CML in chronic phase that has continued to grow despite treatment with at least two different TKIs.
  • Patients may not have previously received ponatinib.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients age 18 and older.

For more information and to inquire about eligibility for this study, please contact Dr. Michael Mauro at 212-639-3107.