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

Phase 2
16 Years
Not Enrolling
Acute Myelogenous Leukemia, Myelodysplastic Syndrome

Thank you

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 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.

Inclusion Criteria:

1. Understand and voluntarily sign an informed consent form.

2. Age >/= to 16 years at the time of signing the informed consent form.

3. Diagnosis of Acute myeloid leukemia (AML) [other than acute promyelocytic leukemia
(APL)] with refractory/relapsed disease. Patients with newly diagnosed AML will be
eligible if not a candidate for intensive chemotherapy. Patients with high-risk
Intermediate-2 or high by International Prognostic Scoring System (IPSS) or >/= to
10% blasts) MDS will also be eligible. All non-hematological toxicity of previous
cancer therapy should have resolved to toxicities not involving major organs).

4. Eastern Cooperative Oncology Group (ECOG) performance status of entry.

5. Laboratory test results within these ranges (unless due to leukemia): Serum
creatinine (SGOT) and/or alanine aminotransferase (ALT) (SGPT) Upper limit of normal (ULN) if related to disease

6. Women of childbearing potential (WCBP) must have a negative urine pregnancy test
within 7 days and must either commit to continued abstinence from heterosexual
intercourse or adopting at least one highly effective method of contraception. These
methods include intra-uterine device, tubal ligation, partner's vasectomy, hormonal
birth control pills. Men must agree not to father a child and agree to use a condom
if his partner is of child bearing potential.

Exclusion Criteria:

1. Pregnant or breastfeeding females.

2. Any condition, including the presence of laboratory abnormalities, which places the
patient at unacceptable risk.

3. Use of any other experimental drug or therapy for leukemia within 7 days unless there
is clear evidence of rapid disease progression.

4. Use of hydrea to control proliferative disease will be allowed prior to starting
therapy on study and for up to 7 days each during cycle 1-3 (Maximum daily dose of

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of Participants With Complete Response (CR)

Outcome Description:

Complete Response (CR) was defined as normalization of peripheral blood and bone marrow with /= 1 * 10^9 /l, and a platelet count of >/= 100 & 10^9 /l. Evaluation after each treatment course (5-6 weeks) up to 6 cycles.

Outcome Time Frame:

Up to 36 weeks

Safety Issue:


Principal Investigator

Gautam Borthakur, MBBS

Investigator Role:

Study Chair

Investigator Affiliation:

UT MD Anderson Cancer Center


United States: Institutional Review Board

Study ID:




Start Date:

February 2008

Completion Date:

August 2012

Related Keywords:

  • Acute Myelogenous Leukemia
  • Myelodysplastic Syndrome
  • Leukemia
  • AML
  • MDS
  • High-Risk Myelodysplastic Syndrome
  • Decitabine
  • 5-aza-2 deoxycytidine
  • Dacogen
  • Gemtuzumab Ozogamicin
  • Mylotarg
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Myelodysplastic Syndromes
  • Preleukemia



UT MD Anderson Cancer Center Houston, Texas  77030