Ponatinib as Second Line Therapy for Patients With Chronic Myeloid Leukemia in Chronic Phase Resistant or Intolerant to Imatinib
Study Drug Administration:
You will take ponatinib by mouth 1 time every day while you are on study with about a cup (8
ounces) of water. You may take ponatinib with or without food. If you vomit a dose, you
should not take ponatinib again to make up for that dose. You should wait until your next
scheduled dose. You will complete a study diary in which you will record the date and time
that you take the study drug each time. If you miss any doses, you will also note this in
the study diary. You will bring this diary to every study visit.
The study staff will help you schedule your study visits. The following tests and procedures
will be performed:
- Blood (about 1/2 tablespoon each time) will be drawn for routine tests every 1-2 weeks
for the first 4 weeks, every 4-6 weeks for the first year, every 3-4 months for second
year, then every 4-6 months after that. These tests can be done by your home doctor
and sent to your study doctor.
- You will have an EKG every 3 months for the first year.
- You will have a physical exam and you will be asked about any drugs you may be taking
and any side effects you may be having every 3 months for the first year, then every
6-12 months after that.
- Blood (about 2 teaspoons) will be drawn to check the status of the disease every 3-4
months for the first year, then every 6-12 after that.
- You will have a bone marrow aspirate for genetic testing and to check the status of the
disease every 3-4 months for the first year, then every 6-12 months for the next 2
years, then every 2-3 years after that.
Length of Study:
You may take the study drug for up to 5 years. You will be taken off study early if
intolerable side effects occur, if the disease gets worse, or if you are unable to follow
Your participation on the study will be over when you have completed the follow-up
visit/call (described below).
If the disease gets worse or the disease never responds to treatment with ponatinib, blood
(about 1 tablespoon) will be drawn about 30 days after your last dose of ponatinib to check
for changes in the BCR-ABL protein which may explain why there was no response to the study
Within 30 days after you leave the study, you will be called or you will come to the clinic
to learn about any side effects or symptoms you may be having. If you are called, this call
will last about 2-3 minutes.
This is an investigational study. Ponatinib is FDA approved to treat patients with certain
types of leukemia. Its use in this study is investigational.
Up to 50 participants will be enrolled in this study. All will be enrolled at MD Anderson.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Major cytogenetic response (MCyR) With Second Line Ponatinib Therapy
Binary indicator of major cytogenetic remission at 6 months from the start of therapy, denoted by MCyR6. Kaplan and Meier used to estimate the unadjusted distributions of time to toxicity, duration of MCyR, and the times to transformation to accelerated phase or blastic phase CML. Appropriate time-to-event regression models fit to the event time data to assess the effects of patient covariates on these event time variables, with the particular models determined by preliminary goodness-of -fit analyses. The distributions of MCyR6 and MMR tabulated and effects of baseline patient covariates on these variables will be assessed by logistic regression.
Jorge Cortes, MD
UT MD Anderson Cancer Center
United States: Food and Drug Administration
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