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Gemtuzumab Ozogamicin in Treating Young Patients With Newly Diagnosed Acute Myeloid Leukemia Undergoing Remission Induction and Intensification Therapy

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: NCT00070174
Recruitment Status : Completed
First Posted : October 7, 2003
Last Update Posted : February 20, 2014
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Children's Oncology Group

Brief Summary:

RATIONALE: Giving chemotherapy before a donor bone marrow transplant helps stop the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. Also, monoclonal antibodies, such as gemtuzumab ozogamicin, can find cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets.

PURPOSE: This phase II trial is studying how well gemtuzumab ozogamicin works in treating young patients who are undergoing remission induction, intensification therapy, and allogeneic bone marrow transplant for newly diagnosed acute myeloid leukemia.

Condition or disease Intervention/treatment Phase
Leukemia Drug: asparaginase Drug: busulfan Drug: cyclophosphamide Drug: cyclosporine Drug: cytarabine Drug: daunorubicin hydrochloride Drug: etoposide Drug: gemtuzumab ozogamicin Drug: methotrexate Drug: mitoxantrone hydrochloride Procedure: allogeneic bone marrow transplantation Phase 2

Detailed Description:



  • Determine the safety of gemtuzumab ozogamicin in children with newly diagnosed acute myeloid leukemia undergoing intensive remission induction and intensification therapy.
  • Determine the complete remission rate of patients treated with this regimen.


  • Determine the feasibility of performing biological studies (e.g., FLT3-ITD and MRD) for risk group stratification in these patients.
  • Determine the effect of karyotypic abnormalities on survival in patients treated with this regimen.

OUTLINE: This is a multicenter study.

  • Induction I: Patients receive high-dose cytarabine (ARA-C) IV twice daily on days 1-10; daunorubicin IV over 6 hours on days 1, 3, and 5; etoposide IV over 4 hours on days 1-5; and gemtuzumab ozogamicin IV over 2 hours on day 6. Patients with CNS-negative disease receive ARA-C intrathecally (IT) on day 1. Patients with CNS-positive disease receive ARA-C IT twice weekly for 2-3 weeks. Between days 28-35, patients are evaluated. Patients achieving remission or who have no more than 20% blasts proceed to induction II.
  • Induction II: Patients receive ARA-C IV twice daily on days 1-8; ARA-C IT on day 1; and daunorubicin IV and etoposide IV as in induction I. Between days 28-35 patients are evaluated. Patients achieving complete remission proceed to intensification course I.
  • Intensification course I: Patients receive ARA-C IV over 1 hour twice daily on days 1-5; ARA-C IT as in induction II; and etoposide IV over 1 hour on days 1-5. Patients are evaluated at day 28. Patients with a 5/6 or 6/6 matched family donor proceed to allogeneic bone marrow transplantation. All other patients in complete remission proceed to intensification course II.
  • Intensification course II: Patients receive ARA-C IV over 2 hours twice daily on days 1-4; ARA-C IT as in induction II; mitoxantrone IV over 1 hour on days 3-6; and gemtuzumab ozogamicin IV over 2 hours on day 7. Patients are evaluated on day 28 and then proceed to intensification course III.
  • Intensification course III: Patients receive ARA-C IV over 3 hours twice daily on days 1, 2, 8, and 9 and asparaginase intramuscularly on days 2 and 9.
  • Allogeneic bone marrow transplantation: Patients receive a preparative regimen comprising busulfan IV over 2 hours 4 times daily on days -9 to -6 and cyclophosphamide IV over 1 hour once daily on days -5 to -2. Allogeneic stem cells are infused on day 0.
  • Graft-versus-host disease prophylaxis: Patients receive oral or IV cyclosporine twice daily on days -1 to 50 and methotrexate IV once daily on days 1, 3, 6, and 11.

In all courses, treatment continues in the absence of disease progression or unacceptable toxicity.

Patients are followed monthly for 6 months, every 2 months for 6 months, every 4 months for 1 year, every 6 months for 1 year, and then annually thereafter.

PROJECTED ACCRUAL: A total of 330 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 350 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Treatment of Newly Diagnosed Childhood Acute Myeloid Leukemia (AML) Using Intensive MRC-Based Therapy and Gemtuzumab Ozogamicin (GMTZ): A COG Pilot Study
Study Start Date : December 2003
Actual Primary Completion Date : September 2006
Actual Study Completion Date : December 2013

Primary Outcome Measures :
  1. Safety
  2. Complete remission rate

Secondary Outcome Measures :
  1. Feasibility
  2. Effect of karyotypic abnormalities

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:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Newly diagnosed primary acute myeloid leukemia (AML)

    • At least 20% bone marrow blasts
    • Meets the customary FAB criteria for AML

      • Patients with cytopenias and bone marrow blasts who do not meet the FAB criteria are eligible provided they have a karyotypic abnormality characteristic of de novo AML (e.g., t[8;21], inv16, or t[16;16]) OR they have the unequivocal presence of megakaryoblasts
    • Isolated granulocytic sarcoma (myeloblastoma) allowed regardless of the results outlined above
  • Previously untreated disease
  • No promyelocytic leukemia (FAB M3)
  • No documented myelodysplastic syndromes (preleukemia) (e.g., chronic myelomonocytic leukemia, refractory anemia [RA], RA with excess blasts, or RA with ringed sideroblasts)
  • No juvenile myelomonocytic leukemia
  • No Fanconi's anemia, Kostmann syndrome, Shwachman syndrome, or any other known bone marrow failure syndrome
  • No Down syndrome



  • 1 month to 21 years* NOTE: *Children under 1 month of age who have progressive disease are allowed

Performance status

  • Karnofsky 50-100% (over 16 years of age) OR
  • Lansky 50-100% (ages 1 to 16)* NOTE: Children under 1 year of age do not require a performance status

Life expectancy

  • Not specified


  • Not specified


  • No inadequate liver function


  • No inadequate renal function
  • No hyperuricemia (greater than 8.0 mg/dL)
  • Creatinine clearance or radioisotope glomerular filtration rate (GFR) at least 70 mL/min OR an equivalent normal GFR OR
  • Creatinine no greater than 1.5 times normal


  • Shortening fraction at least 27% by echocardiogram OR
  • Ejection fraction at least 50% by MUGA


  • No proven or suspected pneumonia


  • Not pregnant or nursing
  • No proven or suspected sepsis or meningitis


Biologic therapy

  • Not specified


  • No prior chemotherapy except intrathecal cytarabine administered that was administered at diagnosis

Endocrine therapy

  • Prior topical and inhalation steroids allowed
  • No concurrent steroids as antiemetics


  • No prior radiotherapy


  • Not specified


  • No prior antileukemic therapy
  • No concurrent pressor agent or ventilatory support unless approved by the study chair
  • No concurrent participation in another COG therapeutic study

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): NCT00070174

Show Show 148 study locations
Sponsors and Collaborators
Children's Oncology Group
National Cancer Institute (NCI)
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Study Chair: Janet Franklin, MD, MPH Children's Hospital Los Angeles
Publications of Results:
Walter RB, Alonzo TA, Gerbing RB, et al.: High expression of the very late antigen (VLA)-4 (CD49d) integrin predicts for reduced risk of relapse and better outcome in pediatric acute myeloid leukemia (AML): A report from the Children's Oncology Group. [Abstract] Blood 114 (22): A-1592, 2009.
Franklin J, Alonzo T, Hurwitz CA, et al.: COG AAML03P1: efficacy and safety in a pilot study of intensive chemotherapy including gemtuzumab in children newly diagnosed with acute myeloid leukemia (AML). [Abstract] Blood 112 (11): A- 136, 2008.

Other Publications:
Berman JN, Gerbing RB, Sung L, et al.: Prevalence and clinical implications of N-RAS mutations in childhood AML - A report from the Children's Oncology Group. [Abstract] Blood 114 (22): A-3115, 2009.
Pollard J, Alonzo T, Gerbing R, et al.: Prevalence and prognostic significance of c-KIT mutations in pediatric CBF AML patients enrolled on serial CCG/COG protocols. [Abstract] Blood 110 (11): A-1442, 2007.

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Children's Oncology Group Identifier: NCT00070174    
Other Study ID Numbers: AAML03P1
CDR0000330133 ( Other Identifier: Clinical )
COG-AAML03P1 ( Other Identifier: Children's Oncology Group )
First Posted: October 7, 2003    Key Record Dates
Last Update Posted: February 20, 2014
Last Verified: February 2014
Keywords provided by Children's Oncology Group:
untreated childhood acute myeloid leukemia and other myeloid malignancies
childhood acute monocytic leukemia (M5b)
childhood acute megakaryocytic leukemia (M7)
childhood acute minimally differentiated myeloid leukemia (M0)
childhood acute myeloblastic leukemia with maturation (M2)
childhood acute myeloblastic leukemia without maturation (M1)
childhood acute myelomonocytic leukemia (M4)
childhood acute monoblastic leukemia (M5a)
childhood acute erythroleukemia (M6)
Additional relevant MeSH terms:
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Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Abortifacient Agents, Nonsteroidal
Abortifacient Agents
Reproductive Control Agents
Antimetabolites, Antineoplastic