Ponatinib Hydrochloride as a Second Treatment Option for Chronic Myeloid Leukemia Patients Resistant or Intolerant to Other Medications
Sponsor: M.D. Anderson Cancer Center
ClinicalTrials ID:NCT01746836
This study is testing how well the drug ponatinib hydrochloride works for patients with chronic myeloid leukemia (CML) who have not responded to or cannot tolerate other treatments like imatinib, dasatinib, or nilotinib. Ponatinib may help stop cancer cell growth by blocking a protein they need.
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Program Overview
This phase II study is exploring the effectiveness of ponatinib hydrochloride as a second treatment option for patients with chronic myeloid leukemia (CML) in the chronic phase. These patients have either not responded to or cannot tolerate their initial treatments with drugs like imatinib, dasatinib, or nilotinib. Ponatinib works by blocking a protein that cancer cells need to grow, potentially helping to control or stop the cancer.
Description
The main goals of this study are to see how many patients with chronic phase CML, who are resistant to other treatments, achieve a significant reduction in cancer cells after 6 months of taking ponatinib. The study will also look at how long it takes for any side effects to appear.
Secondary goals include measuring how many patients achieve different levels of response to ponatinib over time, such as major or complete responses, and how long these responses last. The study will also track how long patients live without the cancer getting worse and overall survival rates. Researchers will evaluate the side effects of ponatinib and the likelihood of developing certain mutations. They will also study differences in treatment responses based on patient characteristics and pre-treatment mutations, and investigate why some patients may become resistant to ponatinib. Additionally, the study will assess the impact of symptoms on patients taking ponatinib.
Exploratory goals include investigating specific genetic markers that might predict treatment outcomes.
Eligibility Criteria
Inclusion Criteria
- Diagnosed with chronic myeloid leukemia (CML) in the chronic phase, confirmed by specific genetic tests.
- Have not responded to or cannot tolerate one of the approved second-generation treatments (bosutinib, dasatinib, or nilotinib) as defined by specific medical guidelines.
- Be over 18 years old.
- Be in good general health with a performance status score of 0 to 2, meaning fully active or restricted in physically strenuous activity but ambulatory.
- Have adequate liver and kidney function based on specific blood tests.
- Agree to sign an informed consent form acknowledging the study's investigational nature.
- Women who can become pregnant must use effective birth control during the study and for at least 3 months after the last dose.
- Men must also use effective birth control to prevent pregnancy in partners.
- Women must have a negative pregnancy test before starting the study.
- Must stop taking previous CML treatments (except hydroxyurea) at least 48 hours before starting the study and recover from any side effects to a mild level.
Exclusion Criteria
- Have taken other targeted treatments for CML besides bosutinib, dasatinib, or nilotinib.
- Have severe heart disease or uncontrolled cardiovascular conditions.
- Have uncontrolled psychiatric disorders.
- Have uncontrolled high blood pressure or diabetes.
- Be pregnant or breastfeeding.
- Have a history of pancreatitis within the past year.
- Be in an advanced phase of CML, such as accelerated or blast phase, as defined by specific medical criteria.
- Have received more than one approved treatment for CML or any unapproved investigational treatments.
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