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Ponatinib-1501 (Ph+ ALL)

Clinical study of the safety and efficacy of ponatinib with chemotherapy in children with Ph+ acute lymphoblastic leukemia (ALL)
Who can enter
  • Children and young adults with Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) in whom the disease has returned (relapsed) or has become insensitive (resistant), who could not tolerate previous treatment (intolerant) or in whom a BCR-ABL1 T315I mutation has been found
  • Age: 1 up to and including 21 years old


The goal of this study is to confirm the best dose of ponatinib (the study drug) in combination with chemotherapy. It is important that this dose can be given without causing serious side effects and risks. In addition, we will determine whether the leukemia responds to treatment with ponatinib in combination with chemotherapy.

We will measure how much ponatinib is present in the blood after it has been taken in combination with chemotherapy. We will also observe the effects of ponatinib in different patients, and assess the safety and tolerability of ponatinib in combination with chemotherapy.


In Ph+ ALL, an abnormal protein called BCR-ABL is present in the leukemia cells. This protein stimulates leukemia cells to grow and survive. Ponatinib has been designed to inhibit the activity of BCR-ABL.

This is the first time ponatinib and chemotherapy have been used together in children. In adults, ponatinib is approved in several countries.

This study consists of two phases. In phase 1 of the study, we will determine the appropriate dose of ponatinib to be given with chemotherapy. In phase 2, we will use the dose determined in phase 1 and assess how well ponatinib works with chemotherapy. In both phases of the study, doctors will do blood tests to determine the amount of ponatinib in the blood.

All participants in this study will receive ponatinib and chemotherapy.

In order to participate in a study please refer to your/your child’s doctor.
For international patients: please feel welcome to contact our International Office.

Last reviewed

August 29, 2022