Ponatinib - Frontline for Chronic Myeloid Leukemia (CML) in Chronic Phase (CP)
The goal of this clinical research study is to learn if ponatinib can help to control CML in accelerated phase. The safety of this drug will also be studied.
Ponatinib is designed to block the function of BCR-ABL, which is the abnormal protein responsible for causing leukemia in certain cells.
Ponatinib may cause a blood clot to form in an artery or in a vein. Depending on the location of the clot, this could cause a heart attack, a stroke, severe damage to other tissue, or death. A blood clot may occur within 2 weeks after you start taking the drug. About 25% (1 in 4) of patients taking the drug form an abnormal clot. Blood clots can occur in patients that do not have other known risk factors for forming clots. If you develop a blood clot, you will need to stop taking ponatinib. In some cases, emergency surgery could be needed to remove the clot and restore blood flow.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Ponatinib as Initial Therapy for Patients With Chronic Myeloid Leukemia in Chronic Phase|
- Complete Cytogenetic Response (CCyR) [ Time Frame: 6 months ] [ Designated as safety issue: No ]The binary indicator of complete cytogenetic remission measured from start of therapy to 6 months, denoted by CCR6. Method of Kaplan and Meier21 used to estimate the unadjusted distributions of time to toxicity, duration of CCR, and the times to transformation to accelerated phase or blastic phase chronic myeloid leukemia (CML).
- Time to Toxicity [ Time Frame: 3 months ] [ Designated as safety issue: Yes ]Time to toxicity (T) defined as any grade 3 or 4 drug-related non-hematologic adverse event that has not resolved to grade 2 or less after 6 weeks of optimal therapeutic management, or drug-related toxicity of any grade that in the opinion of the investigator prevents further therapy with ponatinib. Secondary outcomes include duration of complete cytogenic response (CCR), time to transformation to accelerated phase or blastic phase chronic myeloid leukemia (CML), and major molecular response (MMR). Time to toxicity monitored using the Bayesian method of Thall, et al.
|Study Start Date:||April 2012|
|Estimated Primary Completion Date:||April 2016 (Final data collection date for primary outcome measure)|
Patients receive ponatinib at a dose of 30 mg orally, once daily. If a dose is missed or vomited, the next dose should not be increased to account for missing a dose. Total duration of therapy 3 to 5 years.
Starting dose: 30 mg by mouth once daily.
Other Name: AP24534
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 should not eat within 2 hours before or after taking the drug. 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. Bring this diary to every study visit, as described below.
The tests and procedures for this study have a wide range of time in which they can be done. In general, your schedule of study visits will be as follows:
- Weekly in Month 1
- Monthly in Year 1
- Three (3) to 4 times in Year 2
- Two (2) to 3 times in every year after that
The study staff will help you schedule your study visits. The following tests and procedures will be performed:
- Every 1-2 weeks for the first 4 weeks, then every 4-6 weeks for the first year, then every 3-4 months for the next year, then every 4-6 months after that, blood (about 1/2 tablespoon) will be drawn for routine tests.
- Every 3 months for the first year, you will have an ECG.
- Every 3 months for the first year, then every 6-12 months after that, you will have a physical exam.
- Every 3-4 months for the first year, then every 6-12 months after that, blood (about 2 teaspoons) will be drawn to measure levels of leukemia cells in your body.
- 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, you will have a bone marrow aspirate for genetic testing and to check the status of the disease.
Length of Participation:
You may continue taking 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 study directions.
Your participation on the study will be over when you have completed the follow-up visit/call.
If you leave the study, you will be called or you will come to the clinic within 30 days 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 80 patients will take part in this study. All will be enrolled at MD Anderson.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01570868
|Contact: Jorge Cortes, MD||713-794-5783|
|United States, Texas|
|University of Texas MD Anderson Cancer Center||Recruiting|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Jorge Cortes, MD||UT MD Anderson Cancer Center|