This study is about an anticancer drug called ponatinib which is a tyrasine kinase inhibitor
given with chemotherapy to children, teenagers, and young adults up to 21 years of age with
Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia who have relapsed or are
resistant to other treatment.
The main aims of this study are to confirm the highest dose of ponatinib tablets and
minitablet capsules that can be given to participants with acceptable side effects, and to
evaluate if participant's leukemia achieves remission.
Participants will take ponatinib tablets with chemotherapy. For participants who cannot
swallow tablets, a minitablet form of the ponatinib will be provided. Participants will take
ponatinib for 10 weeks in combination with chemotherapy (reinduction and consolidation
blocks) and will be followed up for at least 3 years.
The drug being tested in this study is called ponatinib. Ponatinib is being tested to treat
pediatric population who have Philadelphia chromosome-positive (Ph+) acute lymphoblastic
leukemia (ALL). Ph+ mixed phenotype acute leukemia (Ph+ MPAL), or Philadelphia
chromosome-like ALL (US only) who have relapsed or are resistant or intolerant to a prior
tyrosine kinase inhibitor (TKI)-containing therapy, or who have the T315I mutation.
The study will enroll approximately 68 participants. Participants will be assigned to a
treatment group either in phase 1 or phase 2. Participants unable to swallow the ponatinib
tablets will receive the age-appropriate formulation, minitablets:
• Ponatinib + Chemotherapy
All participants will be asked to take ponatinib once daily. In phase 1 ponatinib will be
administered in combination with a chemotherapy backbone to determine the recommended phase 2
dose (RP2D) of ponatinib. In both phase 1 and phase 2, treatment consists of two 35-day
blocks of therapy (reinduction block and consolidation block). Each block will include 29
days of treatment of daily ponatinib and a chemotherapy backbone regimen followed by a rest
period from chemotherapy for a minimum of 6 days consisting of daily ponatinib only.
This is a multi-center trial and will be conducted worldwide. The overall time to participate
in this study is 6.5 years. Participants will make multiple visits to the clinic, and may be
contacted by telephone in at least 36 months of follow-up after treatment.
Pediatric Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia (Ph+ALL), Ph+ Mixed Phenotype Acute Leukemia (MPAL), Philadelphia Chromosome-Like ALL (Ph-like ALL)
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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