Trial Information

A Phase II Study of Decitabine and Gemtuzumab Ozogamicin in Acute Myelogenous Leukemia (AML) and High-Risk Myelodysplastic Syndrome (MDS)

The Study Drugs:

Gemtuzumab ozogamicin is designed to attach to Sialic acid-binding Ig-like lectin 3 (CD33),
a certain protein that is often found in leukemia cells, causing them to die.

Decitabine is designed to damage the DNA (the genetic material) of cells, which may cause
cancer cells to die.

Study Drug Administration:

If you are found to be eligible to take part in this study, you will receive decitabine
through a needle in your vein over 1 and 1/2 hours on Days 1-5 of each cycle. You will also
receive gemtuzumab ozogamicin by vein over about 1 hour after you receive decitabine on Day
5 of each 4-6 week cycle.

During Cycle 1 only, if a bone marrow test done 2 weeks after you receive your first study
drug treatment shows abnormal leukemia cells, you will receive another treatment with
decitabine intravenously over 1 and 1/2 hours for 5 days.

Gemtuzumab may cause allergic reactions, nausea, and vomiting. To help prevent such side
effects, you will receive Benadryl (diphenhydramine) and hydrocortisone. You may receive
these drugs by vein, or by mouth on the days you get gemtuzumab ozogamicin during the entire
study.

Study Visits:

During Cycle 1, blood (about 2 teaspoons) will be drawn at least 1 time each week for
routine tests. If the doctor thinks it is necessary, you may be asked to have additional
blood drawn.

During Cycle 1, after approximately 2 weeks of your first study drug administration , you
will have a bone marrow aspiration to decide whether you will receive additional decitabine
in Cycle 1.

During Cycles 2-3, blood (about 2 teaspoons) will be drawn for routine tests at least 2
times each month.

During Cycles 4 and beyond, blood (about 2 teaspoons) will be drawn for routine tests at
least 1 time each month.

If the doctor thinks it is necessary, you may have a bone marrow aspirate every 1 to 3
months to check the status of the disease.

Length of Study:

You may receive the combination of decitabine and gemtuzumab ozogamicin for up to 6 cycles.
After this, if your doctor thinks it is in your best interest, you may continue to take
decitabine alone for up to 24 cycles. You will be taken off study early if the disease gets
worse, you experience intolerable side effects, or your doctor thinks that it is no longer
in your best interest to receive the study drug(s).

Long-term Follow-up:

Once you are off study, you will have follow up visits every month for up to 2 years. At
these visits, blood (about 2 teaspoons) will be drawn for routine tests.

This is an investigational study. Gemtuzumab ozogamicin is FDA approved and commercially
available for the treatment of AML that has come back after treatment in patients over the
age of 65 years. Decitabine is FDA approved and commercially available for the treatment of
MDS. The use of gemtuzumab ozogamicin and decitabine in combination is investigational.

Up to 100 patients will take part in this study. All will be enrolled at M. D. Anderson.