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Gemtuzumab Ozogamicin in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Acute Promyelocytic Leukemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01869803
Expanded Access Status : Approved for marketing
First Posted : June 5, 2013
Last Update Posted : March 2, 2018
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Wake Forest University Health Sciences

Brief Summary:
This clinical trial studies gemtuzumab ozogamicin in treating patients with relapsed or refractory acute myeloid leukemia or acute promyelocytic leukemia. Monoclonal antibodies, such as gemtuzumab ozogamicin, can block cancer growth in different ways. Some block the ability of cancer to grow and spread. Others find cancer cells and help kill them or carry cancer-killing substances to them.

Condition or disease Intervention/treatment
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities Adult Acute Myeloid Leukemia With Del(5q) Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) Adult Acute Myeloid Leukemia With t(15;17)(q22;q12) Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) Adult Acute Myeloid Leukemia With t(8;21)(q22;q22) Adult Acute Promyelocytic Leukemia (M3) Childhood Acute Promyelocytic Leukemia (M3) Recurrent Adult Acute Myeloid Leukemia Recurrent Childhood Acute Myeloid Leukemia Drug: gemtuzumab ozogamicin Other: laboratory biomarker analysis

Detailed Description:


I. To provide patients with acute myeloid leukemia (AML) or acute promyelocytic leukemia (APL), who have relapsed or who are refractory to standard treatments, with access to gemtuzumab ozogamicin (Mylotarg) when no other comparable or satisfactory alternative therapy is available.

II. To carefully monitor safety and to report safety information from patients receiving Mylotarg in this setting.


Patients receive gemtuzumab ozogamicin intravenously (IV) over 2 hours on days 1 and 15.

After completion of study treatment, patients are followed up every month for 1 year.

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
Official Title: Compassionate Use of Gemtuzumab Ozogamicin (Mylotarg) for Treatment of Patients With Relapsed or Refractory CD33-Positive Acute Myeloid Leukemia (AML) or Acute Promyelocytic Leukemia (APL)

Intervention Details:
  • Drug: gemtuzumab ozogamicin
    Given IV
    Other Names:
    • Calicheamicin-Conjugated Humanized Anti-CD33 Monoclonal Antibody
    • CDP-771
    • CMA-676
    • Mylotarg
  • Other: laboratory biomarker analysis
    Correlative studies

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of relapsed or refractory AML and not candidate for standard consolidation treatment after daunorubicin and cytosine arabinoside OR diagnosis of APL relapsed after tretinoin (ATRA) and arsenic trioxide therapy or APL with persisting or rising blasts, and no other comparable or satisfactory alternative therapy available (including patients not eligible for, or who have access to, investigational therapies via a clinical trial)
  • Patients must have an initial diagnosis of AML, biphenotypic acute leukemia, or APL
  • Patients must have cluster of differentiation (CD)33 positivity of > 30%
  • Eastern Cooperative Oncology Group (ECOG) performance status =< 3 / Karnofsky > 60%
  • Total bilirubin within normal institutional limits
  • Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 2 x institutional upper limit of normal
  • It is deemed ethical to provide an experimental drug (e.g., Mylotarg) that is associated with hepatotoxicity (veno-occlusive disease [VOD]) and myelosuppression
  • Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to receiving Mylotarg and for the duration of treatment; should a woman become pregnant or suspect she is pregnant while receiving treatment with Mylotarg, she should inform her treating physician immediately
  • Ability to understand and the willingness to sign a written Institutional Review Board (IRB)-approved informed consent document

Exclusion Criteria:

  • Patients may not currently be receiving any other investigational agents for leukemia
  • Patients with known untreated hepatitis C
  • Uncontrolled intercurrent illness including, but not limited to active liver disease, or psychiatric illness/social situations that would limit compliance with study requirements
  • Pregnant women are excluded from this study; breastfeeding should be discontinued if the mother is treated with Mylotarg
  • Human immunodeficiency virus (HIV)-positive patients on combination antiretroviral therapy are ineligible; appropriate studies will be undertaken in patients receiving combination antiretroviral therapy when indicated
  • Patients with a known hypersensitivity to gemtuzumab ozogamicin or its parts: recombinant humanized anti-CD33 monoclonal (hP67.6) antibody, calicheamicin derivatives or other ingredients

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01869803

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United States, North Carolina
Comprehensive Cancer Center of Wake Forest University
Winston-Salem, North Carolina, United States, 27157
Sponsors and Collaborators
Wake Forest University Health Sciences
National Cancer Institute (NCI)
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Principal Investigator: Leslie Ellis Wake Forest University Health Sciences
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Responsible Party: Wake Forest University Health Sciences Identifier: NCT01869803    
Other Study ID Numbers: CCCWFU# 99213
NCI-2013-00965 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
P30CA012197 ( U.S. NIH Grant/Contract )
First Posted: June 5, 2013    Key Record Dates
Last Update Posted: March 2, 2018
Last Verified: February 2018
Additional relevant MeSH terms:
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Leukemia, Myeloid
Leukemia, Myeloid, Acute
Leukemia, Promyelocytic, Acute
Neoplasms by Histologic Type
Antineoplastic Agents, Immunological
Antineoplastic Agents
Antibiotics, Antineoplastic