Phase I-II Study of Low-Dose Azacitidine (Vidaza) in Patients With Chronic Myeloid Leukemia Who Have Minimal Residual Disease While Receiving Therapy With Tyrosine Kinase Inhibitors (VZ-CML-PI-0236)
Study Drug Administration:
If you are found to be eligible to take part in this study, you will continue receiving the
same TKI at the dose you had been receiving for the last 6 months.
You will receive azacitidine either as an injection under your skin or through a vein every
day for 3 to 7 days of each 28-day cycle. The dose and how often you take the drug will
depend on when you enter the study. The study staff will tell you how often you will
receive the drug.
In the first part of the study, you will be assigned to a dose level of azacitidine based on
when you join this study. Up to 2 dose levels of azacitidine will be tested. At least 3
participants will be enrolled at each dose level. The first group of participants will
receive the lowest dose level. The next group will receive a higher dose than the group
before it, if no intolerable side effects were seen. This will continue until the highest
tolerable dose of azacitidine is found. This is called the Dose Escalation Group.
Once the highest tolerated dose has been found, an extra 36 participants will receive
azacitidine at this dose level. This is called the Expansion Group.
If you have severe side effects from the study drug, the study doctor may decide to reduce
and/or stop drug dosing until your side effects improve. If the doctor thinks it is in you
best interest, your dose may be increased.
At every visit, you will be asked about any side effects you have had and to list any drugs
you may be taking.
- Every 1-2 weeks for 8 weeks, then at the start of every cycle, blood (about 1 teaspoon)
will be drawn for routine tests.
- Every 2-4 weeks for 8 weeks, then before each cycle, blood (about 1 teaspoon) will also
be drawn to test your kidney and liver function.
- Before each cycle for 3 cycles, then every 3-6 cycles until 1 year, then every 6-12
cycles, blood (about 1 tablespoon) drawn for molecular testing.
- Every 3-6 months in Year 1, then as often the doctor thinks it is needed, you will have
a bone marrow aspirate to check the status of the disease.
- If you are in the Expansion Group, every 3 months (+/- 1 month) for the first 6 months,
then every 6-12 months, you will have a complete physical exam.
- If you are in the Dose Escalation Group, every 2 weeks for the first month, Months 1,
2, 3, and 6 (+/- 1 month), and then every 6-12 months, you will have a complete
Length of Study:
You may continue taking the study drug for as long as the doctor thinks it is in your best
interest. You will no longer be able to take the study drug if the disease gets worse, if
intolerable side effects occur, or if you are unable to follow study directions.
Your participation on the study will be over once you have completed the end-of-treatment
and follow-up visits.
This is an investigational study. TKIs are approved for the treatment of Chronic Myeloid
Leukemia (CML). Azacitidine is FDA approved and commercially available for the treatment of
patients with MDS. The combination of these drugs to treat CML is investigational.
Up to 48 patients will take part in this study. All will be enrolled at MD Anderson.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Maximum tolerated dose (MTD) of Azacitidine
MTD is defined as the highest dose level in which 6 patients have been treated and one or fewer patient experiences Dose-Limiting Toxicity (DLT) within the first course of treatment.
First 28 day cycle
Jorge Cortes, MD
UT MD Anderson Cancer Center
United States: Food and Drug Administration
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