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Combination Chemotherapy Followed by Melphalan and Peripheral Stem Cell Transplantation in Treating Children With Newly Diagnosed Acute Myeloid Leukemia

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ClinicalTrials.gov Identifier: NCT00004056
Recruitment Status : Completed
First Posted : May 20, 2004
Last Update Posted : July 28, 2014
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Children's Oncology Group

Brief Summary:

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Peripheral stem cell transplantation may allow doctors to give higher doses of chemotherapy drugs and kill more cancer cells.

PURPOSE: Phase I trial to study the effectiveness of combination chemotherapy followed by melphalan and peripheral stem cell transplantation in treating children who have newly diagnosed acute myeloid leukemia that has not been treated previously.

Condition or disease Intervention/treatment Phase
Leukemia Biological: filgrastim Drug: asparaginase Drug: cytarabine Drug: daunorubicin hydrochloride Drug: melphalan Drug: thioguanine Procedure: peripheral blood stem cell transplantation Phase 1

Detailed Description:

OBJECTIVES: I. Determine the feasibility and toxicity of timed sequential remission induction and consolidation in children with newly diagnosed acute myeloid leukemia. II. Determine the feasibility and toxicity of a single high dose of melphalan with peripheral blood stem cell rescue following an intense timed sequential induction and consolidation in these children.

OUTLINE: This is a multicenter study. Remission induction: Patients receive daunorubicin IV over 15 minutes on days 1-3, cytarabine IV continuously on days 1-7, oral thioguanine daily on days 1-7, and cytarabine intrathecally (IT) on day 1. Cytarabine IV over 3 hours is administered every 12 hours on days 10-12. Filgrastim (G-CSF) is administered IV or subcutaneously (SQ) beginning on day 13 and continuing until blood counts recover. On approximately day 28, patients undergo a bone marrow aspirate and biopsy to assess response. Patients who have attained an M1 or M2a status proceed to consolidation or, if a 5/5 or 6/6 HLA matched sibling donor is available, proceed to allogeneic bone marrow transplantation. Patients with greater than 25% blasts go off study. Consolidation 1: Patients receive daunorubicin IV over 15 minutes on days 1 and 2, cytarabine IV over 3 hours every 12 hours on days 1, 2, 8, and 9, and asparaginase on days 2 and 9. G-CSF IV or SQ begins on day 10 and continues until blood counts recover. Consolidation 2: Patients receive cytarabine IV over 3 hours every 12 hours on days 1, 3, and 5. G-CSF IV or SQ begins on day 6 and continues until blood counts recover. Peripheral blood stem cells (PBSC) are collected after the second course of consolidation. Consolidation 3: Treatment is repeated as in consolidation 1. Patients who remain in morphologic remission after consolidation 3 proceed with therapy. Patients receive melphalan IV over 30 minutes on day -2, then PBSC are reinfused on day 0. G-CSF IV or SQ begins on day 1 and continues until blood counts recover. Patients are followed every 6 months for 4 years and then annually thereafter.

PROJECTED ACCRUAL: A total of 20-30 patients will be accrued for this study within 8 months.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 35 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Treatment of Newly Diagnosed Childhood AML Using a Timed-Sequential Remission Induction and Consolidation Followed by Single Dose Melphalan With Peripheral Stem Cell Rescue: A POG Pilot Study
Study Start Date : October 1999
Primary Completion Date : October 2002
Study Completion Date : March 2007

Arm Intervention/treatment
Experimental: Chemo + STEM cell
See detailed description.
Biological: filgrastim
Other Names:
  • Granulocyte Colony-Stimulating Factor
  • r-metHuG-CSF
  • GCSF
  • Neupogen®
  • NSC #614629
Drug: asparaginase
Other Names:
  • E. coli
  • Elspar
  • NSC #109229
Drug: cytarabine
Other Names:
  • cytosine arabinoside
  • AraC
  • Cytosar
  • NSC #063878
Drug: daunorubicin hydrochloride
Other Names:
  • daunomycin
  • DNR
  • Cerubidine
  • NSC #82151
Drug: melphalan
Other Names:
  • L-phenylalanine mustard
  • L-PAM
  • L-sarcolysin
  • Alkeran
  • NSC #008806
Drug: thioguanine
Other Names:
  • 6-thioguanine
  • 6-TG
  • NSC #000752
Procedure: peripheral blood stem cell transplantation

Primary Outcome Measures :
  1. Feasibility and toxicity of an intensive regimen that uses timed-sequential therapy [ Time Frame: Length of study ]
    To determine the feasibility and toxicity of an intensive regimen that uses timed-sequential therapy as a strategy for both remission induction and consolidation of newly diagnosed children with AML.

  2. Feasibility and toxicity of a single high dose of melphalan with peripheral stem cell rescue [ Time Frame: Length of study ]
    To test the feasibility and toxicity of a single high dose of melphalan with peripheral stem cell rescue following an intense timed-sequential induction and consolidation.

Secondary Outcome Measures :
  1. Make observations regarding PCR evidence of Minimal Residual Disease [ Time Frame: Length of study ]
    To make observations regarding PCR evidence of Minimal Residual Disease in patients with relevant specific translocations who obtain a clinical remission.

Information from the National Library of Medicine

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

DISEASE CHARACTERISTICS: Histologically proven, previously untreated primary acute myeloid leukemia (AML) Isolated granulocytic sarcoma (myeloblastoma) allowed Patients with cytopenias and bone marrow blasts greater than 5% but less than 30% eligible only if there is karyotypic abnormality characteristic of de novo AML (t(8;21), inv16, t(9;11), etc.) OR unequivocal presence of megakaryoblasts No acute promyelocytic leukemia (M3) No Down syndrome

PATIENT CHARACTERISTICS: Age: 21 and under Performance status: Not specified Life expectancy: Not specified Hematopoietic: Not specified Hepatic: Bilirubin no greater than 3 times upper limit of normal Renal: Creatinine no greater than 1.5 mg/dL Uric acid no greater than 8.0 mg/dL Cardiovascular: Cardiac function normal by echocardiogram Pulmonary: No uncontrolled, life threatening pneumonia Other: No uncontrolled, life threatening sepsis or meningitis Not pregnant Fertile patients must use effective contraception


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): NCT00004056

  Hide Study Locations
United States, Alabama
University of Alabama Comprehensive Cancer Center
Birmingham, Alabama, United States, 35294
United States, Arizona
Arizona Cancer Center
Tucson, Arizona, United States, 85724
United States, Arkansas
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72205
United States, California
Lucile Packard Children's Hospital at Stanford
Palo Alto, California, United States, 94304
Children's Hospital and Health Center
San Diego, California, United States, 92123-4282
United States, Florida
Nemours Children's Clinic
Jacksonville, Florida, United States, 32207
United States, Georgia
Emory University Hospital - Atlanta
Atlanta, Georgia, United States, 30322
United States, Illinois
Children's Memorial Hospital, Chicago
Chicago, Illinois, United States, 60614
United States, Maine
Maine Children's Cancer Program
Scarborough, Maine, United States, 04074
United States, Maryland
Johns Hopkins Oncology Center
Baltimore, Maryland, United States, 21231
United States, Massachusetts
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, United States, 02114
United States, Michigan
Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
United States, Missouri
Cardinal Glennon Children's Hospital
Saint Louis, Missouri, United States, 63104
United States, New Jersey
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
Tomorrows Children's Institute
Hackensack, New Jersey, United States, 07601
United States, New York
Mount Sinai School of Medicine
New York, New York, United States, 10029
United States, Texas
Simmons Cancer Center - Dallas
Dallas, Texas, United States, 75235-9154
Cook Children's Medical Center - Fort Worth
Fort Worth, Texas, United States, 76104
United States, Wisconsin
Midwest Children's Cancer Center
Milwaukee, Wisconsin, United States, 53226
Canada, Quebec
Montreal Children's Hospital
Montreal, Quebec, Canada, H3H 1P3
Sponsors and Collaborators
Children's Oncology Group
National Cancer Institute (NCI)
Study Chair: Craig A. Hurwitz, MD Maine Children's Cancer Program at Barbara Bush Children's Hospital

Hurwitz CA, Chang M, Graham M, et al.: Timed-sequential remission induction and intensification followed by stem cell rescue for childhood AML -a POG pilot study. [Abstract] Proceedings of the American Society of Clinical Oncology 21: A-1553, 2002.

Responsible Party: Children's Oncology Group
ClinicalTrials.gov Identifier: NCT00004056     History of Changes
Other Study ID Numbers: 9822
First Posted: May 20, 2004    Key Record Dates
Last Update Posted: July 28, 2014
Last Verified: July 2014

Keywords provided by Children's Oncology Group:
untreated childhood acute myeloid leukemia and other myeloid malignancies
childhood acute monoblastic leukemia and acute monocytic leukemia (M5)
childhood acute myeloblastic leukemia without maturation (M1)
childhood acute myeloblastic leukemia with maturation (M2)
childhood acute myelomonocytic leukemia (M4)
childhood acute erythroleukemia (M6)
childhood acute megakaryocytic leukemia (M7)
childhood acute minimally differentiated myeloid leukemia (M0)

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Alkylating Agents
Myeloablative Agonists
Antibiotics, Antineoplastic
Topoisomerase II Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Adjuvants, Immunologic