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

This study has suspended participant recruitment.
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Wake Forest University Health Sciences Identifier:
First received: May 31, 2013
Last updated: April 13, 2017
Last verified: April 2017
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 Intervention
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

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
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)

Resource links provided by NLM:

Further study details as provided by Wake Forest University Health Sciences:

Primary Outcome Measures:
  • Access to gemtuzumab ozogamicin [ Time Frame: Up to 1 year ]
  • Incidence of adverse events as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 [ Time Frame: Up to 1 year ]

Estimated Enrollment: 30
Study Start Date: August 2013
Estimated Study Completion Date: August 2020
Estimated Primary Completion Date: August 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment (gemtuzumab ozogamicin)
Patients receive gemtuzumab ozogamicin IV over 2 hours on days 1 and 15.
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

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.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01869803

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)
Principal Investigator: Leslie Ellis Wake Forest University Health Sciences
  More Information

Responsible Party: Wake Forest University Health Sciences Identifier: NCT01869803     History of Changes
Other Study ID Numbers: CCCWFU# 99213
NCI-2013-00965 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
P30CA012197 ( US NIH Grant/Contract Award Number )
Study First Received: May 31, 2013
Last Updated: April 13, 2017

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Leukemia, Promyelocytic, Acute
Neoplasms by Histologic Type
Antineoplastic Agents processed this record on April 26, 2017