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NCT00070174 · CITED IN SOURCE DOCUMENTS

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

An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.

Phase
PHASE2
Status at capture
COMPLETED
Registry last update
2014-02-20

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.

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What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

No posted result sections were captured. Registered plans do not establish that a treatment works.

Who could take part
eligibility Criteria
DISEASE CHARACTERISTICS: * 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 PATIENT CHARACTERISTICS: Age * 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 Hematopoietic * Not specified Hepatic * No inadequate liver function Renal * 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 Cardiovascular * Shortening fraction at least 27% by echocardiogram OR * Ejection fraction at least 50% by MUGA Pulmonary * No proven or suspected pneumonia Other * Not pregnant or nursing * No proven or suspected sepsis or meningitis PRIOR CONCURRENT THERAPY: Biologic therapy * Not specified Chemotherapy * 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 Radiotherapy * No prior radiotherapy Surgery * Not specified Other * 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
healthy Volunteers
false
sex
ALL
std Ages
  1. CHILD
  2. ADULT
  3. OLDER_ADULT
Treatment arms and interventions
interventions
  1. name
    asparaginase
    type
    DRUG
  2. name
    busulfan
    type
    DRUG
  3. name
    cyclophosphamide
    type
    DRUG
  4. name
    cyclosporine
    type
    DRUG
  5. name
    cytarabine
    type
    DRUG
  6. name
    daunorubicin hydrochloride
    type
    DRUG
  7. name
    etoposide
    type
    DRUG
  8. name
    gemtuzumab ozogamicin
    type
    DRUG
  9. name
    methotrexate
    type
    DRUG
  10. name
    mitoxantrone hydrochloride
    type
    DRUG
  11. name
    allogeneic bone marrow transplantation
    type
    PROCEDURE
Study design
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
350
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. measure
    Safety
  2. measure
    Complete remission rate
secondary Outcomes
  1. measure
    Feasibility
  2. measure
    Effect of karyotypic abnormalities
Full study description
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.
detailed Description
OBJECTIVES: Primary * 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. Secondary * 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.
Source references
references
  1. citation
    Pollard JA, Alonzo TA, Loken M, Gerbing RB, Ho PA, Bernstein ID, Raimondi SC, Hirsch B, Franklin J, Walter RB, Gamis A, Meshinchi S. Correlation of CD33 expression level with disease characteristics and response to gemtuzumab ozogamicin containing chemotherapy in childhood AML. Blood. 2012 Apr 19;119(16):3705-11. doi: 10.1182/blood-2011-12-398370. Epub 2012 Feb 29.
    pmid
    22378848
    type
    BACKGROUND
  2. citation
    Ho PA, Kopecky KJ, Alonzo TA, Gerbing RB, Miller KL, Kuhn J, Zeng R, Ries RE, Raimondi SC, Hirsch BA, Oehler V, Hurwitz CA, Franklin JL, Gamis AS, Petersdorf SH, Anderson JE, Godwin JE, Reaman GH, Willman CL, Bernstein ID, Radich JP, Appelbaum FR, Stirewalt DL, Meshinchi S. Prognostic implications of the IDH1 synonymous SNP rs11554137 in pediatric and adult AML: a report from the Children's Oncology Group and SWOG. Blood. 2011 Oct 27;118(17):4561-6. doi: 10.1182/blood-2011-04-348888. Epub 2011 Aug 26.
    pmid
    21873548
    type
    BACKGROUND
  3. citation
    Ho PA, Kuhn J, Gerbing RB, Pollard JA, Zeng R, Miller KL, Heerema NA, Raimondi SC, Hirsch BA, Franklin JL, Lange B, Gamis AS, Alonzo TA, Meshinchi S. WT1 synonymous single nucleotide polymorphism rs16754 correlates with higher mRNA expression and predicts significantly improved outcome in favorable-risk pediatric acute myeloid leukemia: a report from the children's oncology group. J Clin Oncol. 2011 Feb 20;29(6):704-11. doi: 10.1200/JCO.2010.31.9327. Epub 2010 Dec 28.
    pmid
    21189390
    type
    BACKGROUND
  4. citation
    Ho PA, Alonzo TA, Kopecky KJ, Miller KL, Kuhn J, Zeng R, Gerbing RB, Raimondi SC, Hirsch BA, Oehler V, Hurwitz CA, Franklin JL, Gamis AS, Petersdorf SH, Anderson JE, Reaman GH, Baker LH, Willman CL, Bernstein ID, Radich JP, Appelbaum FR, Stirewalt DL, Meshinchi S. Molecular alterations of the IDH1 gene in AML: a Children's Oncology Group and Southwest Oncology Group study. Leukemia. 2010 May;24(5):909-13. doi: 10.1038/leu.2010.56. Epub 2010 Apr 8.
    pmid
    20376086
    type
    BACKGROUND
  5. citation
    Ho PA, Zeng R, Alonzo TA, Gerbing RB, Miller KL, Pollard JA, Stirewalt DL, Heerema NA, Raimondi SC, Hirsch B, Franklin JL, Lange B, Meshinchi S. Prevalence and prognostic implications of WT1 mutations in pediatric acute myeloid leukemia (AML): a report from the Children's Oncology Group. Blood. 2010 Aug 5;116(5):702-10. doi: 10.1182/blood-2010-02-268953. Epub 2010 Apr 22.
    pmid
    20413658
    type
    BACKGROUND
  6. citation
    Phillips CL, Gerbing R, Alonzo T, Perentesis JP, Harley IT, Meshinchi S, Bhatla D, Radloff G, Davies SM. MDM2 polymorphism increases susceptibility to childhood acute myeloid leukemia: a report from the Children's Oncology Group. Pediatr Blood Cancer. 2010 Aug;55(2):248-53. doi: 10.1002/pbc.22519.
    pmid
    20582981
    type
    BACKGROUND
  7. citation
    Pollard JA, Alonzo TA, Gerbing RB, Ho PA, Zeng R, Ravindranath Y, Dahl G, Lacayo NJ, Becton D, Chang M, Weinstein HJ, Hirsch B, Raimondi SC, Heerema NA, Woods WG, Lange BJ, Hurwitz C, Arceci RJ, Radich JP, Bernstein ID, Heinrich MC, Meshinchi S. Prevalence and prognostic significance of KIT mutations in pediatric patients with core binding factor AML enrolled on serial pediatric cooperative trials for de novo AML. Blood. 2010 Mar 25;115(12):2372-9. doi: 10.1182/blood-2009-09-241075. Epub 2010 Jan 7.
    pmid
    20056794
    type
    BACKGROUND
  8. citation
    Berman JN, Gerbing RB, Alonzo TA, Ho PA, Miller K, Hurwitz C, Heerema NA, Hirsch B, Raimondi SC, Lange B, Franklin JL, Gamis A, Meshinchi S. Prevalence and clinical implications of NRAS mutations in childhood AML: a report from the Children's Oncology Group. Leukemia. 2011 Jun;25(6):1039-42. doi: 10.1038/leu.2011.31. Epub 2011 Mar 1. No abstract available.
    pmid
    21358716
    type
    BACKGROUND
  9. citation
    Ho PA, Alonzo TA, Gerbing RB, Pollard J, Stirewalt DL, Hurwitz C, Heerema NA, Hirsch B, Raimondi SC, Lange B, Franklin JL, Radich JP, Meshinchi S. Prevalence and prognostic implications of CEBPA mutations in pediatric acute myeloid leukemia (AML): a report from the Children's Oncology Group. Blood. 2009 Jun 25;113(26):6558-66. doi: 10.1182/blood-2008-10-184747. Epub 2009 Mar 20.
    pmid
    19304957
    type
    BACKGROUND
  10. citation
    Sung L, Alonzo TA, Gerbing RB, Aplenc R, Lange BJ, Woods WG, Feusner J, Franklin J, Patterson MJ, Gamis AS; Children's Oncology Group. Respiratory syncytial virus infections in children with acute myeloid leukemia: a report from the Children's Oncology Group. Pediatr Blood Cancer. 2008 Dec;51(6):784-6. doi: 10.1002/pbc.21710.
    pmid
    18680152
    type
    BACKGROUND
  11. citation
    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.
    type
    BACKGROUND
  12. citation
    Cooper TM, Franklin J, Gerbing RB, Alonzo TA, Hurwitz C, Raimondi SC, Hirsch B, Smith FO, Mathew P, Arceci RJ, Feusner J, Iannone R, Lavey RS, Meshinchi S, Gamis A. AAML03P1, a pilot study of the safety of gemtuzumab ozogamicin in combination with chemotherapy for newly diagnosed childhood acute myeloid leukemia: a report from the Children's Oncology Group. Cancer. 2012 Feb 1;118(3):761-9. doi: 10.1002/cncr.26190. Epub 2011 Jul 15.
    pmid
    21766293
    type
    RESULT
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        t, or radiographic evidence of an intracranial, intradural chloroma).</li></ul></div><p id="_1943" tabindex="-1">COG trials (including <a href="/clinicaltrials/NCT00070174">AAML03P1 [NCT00070174]</a>, <a href="/clinicaltrials/NCT00372593">AAML0531 [NCT00372593]</a>, and <a href="/clinicaltrials/NCT01371981">AAML1031 [NCT01371981]
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        ence of an intracranial, intradural chloroma).</li></ul></div><p id="_1943" tabindex="-1">COG trials (including <a href="/clinicaltrials/NCT00070174">AAML03P1 [NCT00070174]</a>, <a href="/clinicaltrials/NCT00372593">AAML0531 [NCT00372593]</a>, and <a href="/clinicaltrials/NCT01371981">AAML1031 [NCT01371981]</a>) used a modified v
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        duction):</p> <div class="pdq-content-list"><ol id="_913"><li>The Children's Oncology Group (COG) completed two trials&#8212;<a href="/clinicaltrials/NCT00070174">AAML03P1 (NCT00070174)</a>, a pilot study, and <a href="/clinicaltrials/NCT00372593">AAML0531 (NCT00372593)</a>, a randomized trial&#8212;that examined the in
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        <div class="pdq-content-list"><ol id="_913"><li>The Children's Oncology Group (COG) completed two trials&#8212;<a href="/clinicaltrials/NCT00070174">AAML03P1 (NCT00070174)</a>, a pilot study, and <a href="/clinicaltrials/NCT00372593">AAML0531 (NCT00372593)</a>, a randomized trial&#8212;that examined the incorporation of gemtuzum
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        . </p> <p id="_929" tabindex="-1">Evidence (CNS prophylaxis):</p> <div class="pdq-content-list"><ol id="_930"><li>The COG <a href="/clinicaltrials/NCT00070174">AAML03P1 (NCT00070174)</a> and <a href="/clinicaltrials/NCT00372593">AAML0531 (NCT00372593)</a> trials used single-agent cytarabine for prophylaxis.[<a href="
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        29" tabindex="-1">Evidence (CNS prophylaxis):</p> <div class="pdq-content-list"><ol id="_930"><li>The COG <a href="/clinicaltrials/NCT00070174">AAML03P1 (NCT00070174)</a> and <a href="/clinicaltrials/NCT00372593">AAML0531 (NCT00372593)</a> trials used single-agent cytarabine for prophylaxis.[<a href="#cit/section_6.56">56</
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        ction cycles and three consolidation cycles). In the previous COG <a href="/clinicaltrials/NCT00372593">AAML0531 (NCT00372593)</a> and <a href="/clinicaltrials/NCT00070174">AAML03P1 (NCT00070174)</a> trials, patients who did not undergo HSCT received five cycles of chemotherapy.[<a href="#cit/section_6.63">63</a>] <div class="pdq
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        consolidation cycles). In the previous COG <a href="/clinicaltrials/NCT00372593">AAML0531 (NCT00372593)</a> and <a href="/clinicaltrials/NCT00070174">AAML03P1 (NCT00070174)</a> trials, patients who did not undergo HSCT received five cycles of chemotherapy.[<a href="#cit/section_6.63">63</a>] <div class="pdq-content-list"><ul id="
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        Updated: April 15, 2025
        datetime
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        display
        April 15, 2025

    Preserved source evidence · Not medical advice