Combination Chemotherapy With or Without Bone Marrow Transplantation in Treating Children With Acute Myelogenous Leukemia or Myelodysplastic Syndrome
A PHASE III STUDY IN CHILDREN WITH UNTREATED ACUTE MYELOGENOUS LEUKEMIA (AML) OR MYELODYSPLASTIC SYNDROME (MDS)
4 other identifiers
interventional
880
1 country
1
Brief Summary
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_3
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
August 1, 1996
CompletedFirst Submitted
Initial submission to the registry
November 24, 2000
CompletedFirst Posted
Study publicly available on registry
January 27, 2003
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2006
CompletedJanuary 16, 2013
January 1, 2013
10.1 years
November 24, 2000
January 15, 2013
Conditions
Outcome Measures
Primary Outcomes (11)
Proportions of patients achieving remission rate during induction therapy
Up to 42 days
Proportion of patients dying or with residual disease during induction therapy
Up to 42 days
Time to marrow recovery (induction phase)
Up to 42 days
Frequency of toxicities, including infectious complications (induction phase)
Up to 42 days
Marrow status
At 14 days
Percent of blasts
At the end of induction therapy
Complete remission at the end of consolidation therapy
Up to 5 years
Survival following consolidation
Up to 5 years
Event-free survival following consolidation
Up to 5 years
Overall survival (intensification)
Up to 5 years
EFS (intensification)
Up to 5 years
Study Arms (5)
Arm I (combination chemotherapy)
EXPERIMENTALPatients 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
Arm II (combination chemotherapy)
EXPERIMENTALPatients 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
Arm III (combination chemotherapy, aldesleukin)
EXPERIMENTALPatients receive interleukin-2 IV continuously on days 1-4 and 9-18.
Arm IV (combination chemotherapy)
ACTIVE COMPARATORNo further treatment
Arm V (combination chemotherapy, radiotherapy)
EXPERIMENTALPatients undergo radiotherapy to the chloroma 5 days a week for 2 weeks.
Interventions
Given SC
Given IV or IT
Given IV
Given PO
Given PO
Given IV
Given IT
Given IV
Eligibility Criteria
You may qualify if:
- 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
- +21 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Children's Oncology Group
Arcadia, California, 91006-3776, United States
Related Publications (5)
Tarlock K, Gerbing RB, Ries RE, Smith JL, Leonti A, Huang BJ, Kirkey D, Robinson L, Peplinski JH, Lange B, Cooper TM, Gamis AS, Kolb EA, Aplenc R, Pollard JA, Alonzo TA, Meshinchi S. Prognostic impact of cooccurring mutations in FLT3-ITD pediatric acute myeloid leukemia. Blood Adv. 2024 May 14;8(9):2094-2103. doi: 10.1182/bloodadvances.2023011980.
PMID: 38295280DERIVEDBertrums EJM, Smith JL, Harmon L, Ries RE, Wang YJ, Alonzo TA, Menssen AJ, Chisholm KM, Leonti AR, Tarlock K, Ostronoff F, Pogosova-Agadjanyan EL, Kaspers GJL, Hasle H, Dworzak M, Walter C, Muhlegger N, Morerio C, Pardo L, Hirsch B, Raimondi S, Cooper TM, Aplenc R, Gamis AS, Kolb EA, Farrar JE, Stirewalt D, Ma X, Shaw TI, Furlan SN, Brodersen LE, Loken MR, Van den Heuvel-Eibrink MM, Zwaan CM, Triche TJ, Goemans BF, Meshinchi S. Comprehensive molecular and clinical characterization of NUP98 fusions in pediatric acute myeloid leukemia. Haematologica. 2023 Aug 1;108(8):2044-2058. doi: 10.3324/haematol.2022.281653.
PMID: 36815378DERIVEDHo 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: 20413658DERIVEDPollard 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: 20056794DERIVEDHo 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: 19304957DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Beverly Lange
Children's Oncology Group
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- NIH
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 24, 2000
First Posted
January 27, 2003
Study Start
August 1, 1996
Primary Completion
September 1, 2006
Last Updated
January 16, 2013
Record last verified: 2013-01