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Combination Chemotherapy With or Without Bone Marrow Transplantation in Treating Children With Acute Myelogenous Leukemia or Myelodysplastic Syndrome

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00002798
First Posted: January 27, 2003
Last Update Posted: January 16, 2013
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.
Information provided by (Responsible Party):
National Cancer Institute (NCI)
  Purpose
Randomized phase III trial to compare the effectiveness of different chemotherapy regimens with or without bone marrow transplantation in treating children who have acute myelogenous leukemia or myelodysplastic syndrome. Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. It is not yet known which treatment regimen is more effective for acute myelogenous leukemia or myelodysplastic syndrome

Condition Intervention Phase
Childhood Acute Erythroleukemia (M6) Childhood Acute Megakaryocytic Leukemia (M7) Childhood Acute Monoblastic Leukemia (M5a) Childhood Acute Monocytic Leukemia (M5b) Childhood Acute Myeloblastic Leukemia With Maturation (M2) Childhood Acute Myeloblastic Leukemia Without Maturation (M1) Childhood Acute Myelomonocytic Leukemia (M4) Childhood Myelodysplastic Syndromes Chronic Myelomonocytic Leukemia de Novo Myelodysplastic Syndromes Refractory Anemia Refractory Anemia With Excess Blasts Refractory Anemia With Excess Blasts in Transformation Refractory Anemia With Ringed Sideroblasts Secondary Myelodysplastic Syndromes Untreated Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies Drug: asparaginase Drug: daunorubicin hydrochloride Drug: fludarabine phosphate Drug: therapeutic hydrocortisone Procedure: allogeneic bone marrow transplantation Radiation: 3-dimensional conformal radiation therapy Biological: filgrastim Drug: cytarabine Drug: idarubicin Drug: dexamethasone Drug: thioguanine Drug: etoposide Drug: methotrexate Drug: cyclophosphamide Biological: aldesleukin Drug: busulfan Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A PHASE III STUDY IN CHILDREN WITH UNTREATED ACUTE MYELOGENOUS LEUKEMIA (AML) OR MYELODYSPLASTIC SYNDROME (MDS)

Resource links provided by NLM:


Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • Proportions of patients achieving remission rate during induction therapy [ Time Frame: Up to 42 days ]
  • Proportion of patients dying or with residual disease during induction therapy [ Time Frame: Up to 42 days ]
  • Time to marrow recovery (induction phase) [ Time Frame: Up to 42 days ]
  • Frequency of toxicities, including infectious complications (induction phase) [ Time Frame: Up to 42 days ]
  • Marrow status [ Time Frame: At 14 days ]
  • Percent of blasts [ Time Frame: At the end of induction therapy ]
  • Complete remission at the end of consolidation therapy [ Time Frame: Up to 5 years ]
  • Survival following consolidation [ Time Frame: Up to 5 years ]
  • Event-free survival following consolidation [ Time Frame: Up to 5 years ]
  • Overall survival (intensification) [ Time Frame: Up to 5 years ]
  • EFS (intensification) [ Time Frame: Up to 5 years ]

Enrollment: 880
Study Start Date: August 1996
Primary Completion Date: September 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arm I (combination chemotherapy)

Patients receive treatment as in induction therapy, plus G-CSF SC beginning on day 16 and continuing until blood counts recover. If CSF is clear by day 10 of induction, patients receive cytarabine IT on days 0, 10, and 35. If CSF is not clear, patients receive triple intrathecal therapy (TIT; cytarabine, hydrocortisone, methotrexate) on days 0 and 10.

See Detailed Description

Drug: daunorubicin hydrochloride
Other Names:
  • Cerubidin
  • Cerubidine
  • daunomycin hydrochloride
  • daunorubicin
  • RP-13057
Drug: therapeutic hydrocortisone
Other Names:
  • Aeroseb-HC
  • Barseb HC
  • Cetacort
  • Cort-Dome
  • Cortef
Procedure: allogeneic bone marrow transplantation
Other Names:
  • bone marrow therapy, allogeneic
  • bone marrow therapy, allogenic
  • transplantation, allogeneic bone marrow
  • transplantation, allogenic bone marrow
Biological: filgrastim
Given SC
Other Names:
  • G-CSF
  • Neupogen
Drug: cytarabine
Given IV or IT
Other Names:
  • ARA-C
  • arabinofuranosylcytosine
  • arabinosylcytosine
  • Cytosar-U
  • cytosine arabinoside
Drug: idarubicin
Given IV
Other Names:
  • 4-demethoxydaunorubicin
  • 4-DMDR
  • DMDR
  • IDA
Drug: dexamethasone
Given PO
Other Names:
  • Aeroseb-Dex
  • Decaderm
  • Decadron
  • DM
  • DXM
Drug: thioguanine
Given PO
Other Name: 6-TG
Drug: etoposide
Given IV
Other Names:
  • EPEG
  • VP-16
  • VP-16-213
Drug: methotrexate
Given IT
Other Names:
  • amethopterin
  • Folex
  • methylaminopterin
  • Mexate
  • MTX
Experimental: Arm II (combination chemotherapy)

Patients receive fludarabine IV over 24 hours on days 0 and 1, cytarabine IV over 72 hours on days 2-4, and idarubicin IV over 15 minutes on days 0-2. G-CSF begins on day 6 and continues until blood counts recover. Patients also receive TIT on days -1 and 7, if CSF is not clear on day 10 of induction. Patients on both arms are reassessed on day 35. Those patients with M1 marrow proceed to intensification; all others are removed from the study.

Intensification: See Detailed Description

Drug: asparaginase
Other Names:
  • ASNase
  • Colaspase
  • Crasnitin
  • Elspar
  • L-ASP
Drug: fludarabine phosphate
Other Names:
  • 2-F-ara-AMP
  • Beneflur
  • Fludara
Drug: therapeutic hydrocortisone
Other Names:
  • Aeroseb-HC
  • Barseb HC
  • Cetacort
  • Cort-Dome
  • Cortef
Procedure: allogeneic bone marrow transplantation
Other Names:
  • bone marrow therapy, allogeneic
  • bone marrow therapy, allogenic
  • transplantation, allogeneic bone marrow
  • transplantation, allogenic bone marrow
Biological: filgrastim
Given SC
Other Names:
  • G-CSF
  • Neupogen
Drug: cytarabine
Given IV or IT
Other Names:
  • ARA-C
  • arabinofuranosylcytosine
  • arabinosylcytosine
  • Cytosar-U
  • cytosine arabinoside
Drug: idarubicin
Given IV
Other Names:
  • 4-demethoxydaunorubicin
  • 4-DMDR
  • DMDR
  • IDA
Drug: thioguanine
Given PO
Other Name: 6-TG
Drug: methotrexate
Given IT
Other Names:
  • amethopterin
  • Folex
  • methylaminopterin
  • Mexate
  • MTX
Drug: cyclophosphamide
Given IV
Other Names:
  • CPM
  • CTX
  • Cytoxan
  • Endoxan
  • Endoxana
Drug: busulfan
Other Names:
  • BSF
  • BU
  • Misulfan
  • Mitosan
  • Myeloleukon
Experimental: Arm III (combination chemotherapy, aldesleukin)
Patients receive interleukin-2 IV continuously on days 1-4 and 9-18.
Drug: daunorubicin hydrochloride
Other Names:
  • Cerubidin
  • Cerubidine
  • daunomycin hydrochloride
  • daunorubicin
  • RP-13057
Biological: filgrastim
Given SC
Other Names:
  • G-CSF
  • Neupogen
Drug: cytarabine
Given IV or IT
Other Names:
  • ARA-C
  • arabinofuranosylcytosine
  • arabinosylcytosine
  • Cytosar-U
  • cytosine arabinoside
Drug: idarubicin
Given IV
Other Names:
  • 4-demethoxydaunorubicin
  • 4-DMDR
  • DMDR
  • IDA
Drug: dexamethasone
Given PO
Other Names:
  • Aeroseb-Dex
  • Decaderm
  • Decadron
  • DM
  • DXM
Drug: thioguanine
Given PO
Other Name: 6-TG
Drug: etoposide
Given IV
Other Names:
  • EPEG
  • VP-16
  • VP-16-213
Biological: aldesleukin
Other Names:
  • IL-2
  • Proleukin
  • recombinant human interleukin-2
  • recombinant interleukin-2
Active Comparator: Arm IV (combination chemotherapy)
No further treatment
Drug: daunorubicin hydrochloride
Other Names:
  • Cerubidin
  • Cerubidine
  • daunomycin hydrochloride
  • daunorubicin
  • RP-13057
Biological: filgrastim
Given SC
Other Names:
  • G-CSF
  • Neupogen
Drug: cytarabine
Given IV or IT
Other Names:
  • ARA-C
  • arabinofuranosylcytosine
  • arabinosylcytosine
  • Cytosar-U
  • cytosine arabinoside
Drug: idarubicin
Given IV
Other Names:
  • 4-demethoxydaunorubicin
  • 4-DMDR
  • DMDR
  • IDA
Drug: dexamethasone
Given PO
Other Names:
  • Aeroseb-Dex
  • Decaderm
  • Decadron
  • DM
  • DXM
Drug: thioguanine
Given PO
Other Name: 6-TG
Drug: etoposide
Given IV
Other Names:
  • EPEG
  • VP-16
  • VP-16-213
Experimental: Arm V (combination chemotherapy, radiotherapy)
Patients undergo radiotherapy to the chloroma 5 days a week for 2 weeks.
Drug: daunorubicin hydrochloride
Other Names:
  • Cerubidin
  • Cerubidine
  • daunomycin hydrochloride
  • daunorubicin
  • RP-13057
Radiation: 3-dimensional conformal radiation therapy
Other Names:
  • 3D conformal radiation therapy
  • 3D-CRT
Biological: filgrastim
Given SC
Other Names:
  • G-CSF
  • Neupogen
Drug: cytarabine
Given IV or IT
Other Names:
  • ARA-C
  • arabinofuranosylcytosine
  • arabinosylcytosine
  • Cytosar-U
  • cytosine arabinoside
Drug: idarubicin
Given IV
Other Names:
  • 4-demethoxydaunorubicin
  • 4-DMDR
  • DMDR
  • IDA
Drug: dexamethasone
Given PO
Other Names:
  • Aeroseb-Dex
  • Decaderm
  • Decadron
  • DM
  • DXM
Drug: thioguanine
Given PO
Other Name: 6-TG
Drug: etoposide
Given IV
Other Names:
  • EPEG
  • VP-16
  • VP-16-213

  Show Detailed Description

  Eligibility

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.


Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Histologically confirmed previously untreated acute myeloid leukemia (AML) in patients 1 month to 21 years of age

    • Infants under 1 month with progressive disease eligible

      • Supportive care may be given to confirm that the leukemia is not regressing prior to entry
    • No acute promyelocytic leukemia (FAB M3)
    • No acute undifferentiated leukemia (FAB M0)
  • Histochemical verification of AML required by the following stains:

    • Wright or Giemsa
    • Peroxidase
    • PAS
    • Chloroacetate esterase
    • Sudan black
    • Nonspecific esterase (NSE) with and without fluoride (NaF) inhibition
    • Combined NSE/NaF and butyrate inhibition or diagnosis of megakaryoblasticleukemia (FAB M7) should be supported by one of the following:

      • CD41 reactivity
      • Glycoprotein 1b reactivity
      • Factor VIII-related antigen reactivity
      • Platelet peroxidase on electron microscopy
  • The following are also eligible:

    • Myelodysplastic syndromes, including:

      • Refractory anemia (RA) *
      • RA with ringed sideroblasts (RARS) *
      • RA with excess blasts (RAEB)
      • RAEB in transformation (RAEBt)
      • Chronic myelomonocytic leukemia (CMML)
    • AML with monosomy 7
    • Granulocytic sarcoma (chloroma) with or without marrow involvement
    • Mixed lineage leukemia with 2 morphologically defined populations provided the predominant population is myeloid
  • No Downs syndrome
  • No juvenile chronic myelogenous leukemia
  • No Fanconi's anemia
  • No secondary AML
  • Performance status - Not specified
  • No prior anticancer chemotherapy
  • Prior topical or inhaled steroids for nonmalignant conditions allowed
  • No prior anticancer radiotherapy
  • No prior antileukemic therapy
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT00002798


Locations
United States, California
Children's Oncology Group
Arcadia, California, United States, 91006-3776
Sponsors and Collaborators
National Cancer Institute (NCI)
Investigators
Principal Investigator: Beverly Lange Children's Oncology Group
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00002798     History of Changes
Other Study ID Numbers: NCI-2012-01834
2961
U10CA098543 ( U.S. NIH Grant/Contract )
CDR0000064883 ( Registry Identifier: PDQ (Physician Data Query) )
First Submitted: November 24, 2000
First Posted: January 27, 2003
Last Update Posted: January 16, 2013
Last Verified: January 2013

Additional relevant MeSH terms:
Syndrome
Leukemia
Leukemia, Myeloid, Acute
Anemia
Myelodysplastic Syndromes
Preleukemia
Leukemia, Myeloid
Leukemia, Myelomonocytic, Acute
Leukemia, Myelomonocytic, Chronic
Anemia, Refractory
Anemia, Refractory, with Excess of Blasts
Leukemia, Monocytic, Acute
Leukemia, Erythroblastic, Acute
Anemia, Aplastic
Leukemia, Megakaryoblastic, Acute
Disease
Pathologic Processes
Neoplasms by Histologic Type
Neoplasms
Hematologic Diseases
Bone Marrow Diseases
Precancerous Conditions
Myelodysplastic-Myeloproliferative Diseases
Myeloproliferative Disorders
Hydrocortisone 17-butyrate 21-propionate
Cortisol succinate
Hydrocortisone acetate
Dexamethasone
Hydrocortisone
Fludarabine