Randomized Trial of Therapy of Early Phase Chronic Myelogenous Leukemia With High-Dose Imatinib Mesylate (Gleevec) Alone or in Combination With Peg-Alpha Interferon (PEG-Intron) and Sargramostim (GM-CSF)
Imatinib mesylate is a drug that blocks a protein that is responsible for the development of
CML. PEG-Intron is a natural substance made by the cells of the immune system and helps to
control CML. GM-CSF is a hormone that helps to stimulate the production of white blood
During the study you will take 400 mg of imatinib mesylate by mouth 2 times a day (800 mg a
day total). Imatinib mesylate should be taken each morning and evening with a large glass of
water. You may be given a "pill diary" to write down when (day and time) you take the drug.
You may also write in the diary any side effects you may experience. You may bring the
diary, any unused tablets, and empty bottles of imatinib mesylate with you to every visit to
the study doctor. Any unused supplies must be returned at the end of the study.
After completing 6 months of imatinib mesylate therapy, you will be randomly assigned (as in
the toss of a coin) to one of two groups. Patients in the first group will be given
PEG-Intron and GM-CSF in addition to imatinib mesylate therapy. Patients in the other group
will continue taking only imatinib mesylate.
If you are assigned to the group that will receive PEG-Intron and GM-CSF, you will continue
taking imatinib mesylate. In addition, PEG-Intron will be given as an injection under the
skin once a week. Sargramostim will be given as an injection under the skin 3 times a week.
You and/or your family members can be taught to give these injections.
Every 1-2 weeks during the first 4 weeks of the study, you will have around 2 teaspoons of
blood drawn for routine blood tests and to measure the amount of imatinib in your blood.
The blood tests will then be repeated every 6 to 8 weeks (or more often if your doctor feels
it is necessary) for as long as you are on the study. A bone marrow sample will also be
taken every 3 months for the first year and then every 4 to 6 months for as long as you are
on the study to check on the status of the disease .
You will be asked to visit the doctor for a physical exam and to have vital signs measured.
These visits will be scheduled at least every 3 months while you are on the study. The
visits may be scheduled more often depending on the status of the disease.
Update: February 2012:
Blood test are recommended 2 times per year. Your doctor will discuss with you how often
you should have blood tests. Bone marrow will be done if your doctor thinks it is needed to
check the disease. You must return to M.D. Anderson at least once every year. You may not
need a bone marrow test every visit, but you will have blood drawn to measure the amount of
disease you have.
Treatment in both groups may be continued for up to 7-10 years, or as long as the doctor
feels is necessary to control the leukemia.
If the disease gets worse or you experience any intolerable side effects, you will be taken
off the study and your doctor will discuss other treatment options with you.
This is an investigational study. All of the drugs used in this study are FDA approved and
commercially available. However, their use in this study is investigational. A total of 98
patients will take part in this study. All will be enrolled at MD Anderson.
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment
Durations of PCR negativity, cytogenetic response, hematologic control, and survival.
Primary measure of efficacy is molecular response at 12 months. Other endpoints are cytogenetic response, duration of molecular and cytogenetic response, and survival. The trial will be considered successful if it is established (with 95% probability) that molecular response rate with the combination is superior to the 19% achieved with Gleevec alone.
Jorge E Cortes, MD
M.D. Anderson Cancer Center
United States: Food and Drug Administration
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