Clinical trial · Interventional
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)
NCT00002798CI-TRIAL-00008932completedPhase 3ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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
Conditions
Conditions (16)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Childhood Acute Erythroleukemia (M6) | Acute Erythroid Leukemia | ALIAS | 0.85 |
| Childhood Acute Megakaryocytic Leukemia (M7) | Childhood Acute Megakaryoblastic Leukemia | ALIAS | 0.90 |
| Childhood Acute Monoblastic Leukemia (M5a) | Childhood Acute Monoblastic Leukemia | ONTOLOGY_EXACT | 0.85 |
| Childhood Acute Monocytic Leukemia (M5b) | Childhood Acute Monocytic Leukemia | ONTOLOGY_EXACT | 0.85 |
| Childhood Acute Myeloblastic Leukemia With Maturation (M2) | Childhood Acute Myeloid Leukemia with Maturation | ALIAS | 0.90 |
| Childhood Acute Myeloblastic Leukemia Without Maturation (M1) | Childhood Acute Myeloid Leukemia without Maturation | ALIAS |
Interventions
Interventions (16)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| 3-dimensional conformal radiation therapy | Radiation | — | UNRESOLVED |
| aldesleukin | Biological | Aldesleukin | ALIAS |
| allogeneic bone marrow transplantation | Procedure | — | UNRESOLVED |
| asparaginase | Drug | Asparaginase | ALIAS |
| busulfan | Drug | Busulfan | ALIAS |
| cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| cytarabine | Drug | Cytarabine | ALIAS |
| daunorubicin hydrochloride | Drug | Daunorubicin | ALIAS |
Design
Arms and outcomes
Arms (5)
- type
- EXPERIMENTAL
- label
- Arm I (combination chemotherapy)
- description
- 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
- interventionNames
- Drug: daunorubicin hydrochloride
- Drug: therapeutic hydrocortisone
- Procedure: allogeneic bone marrow transplantation
- Biological: filgrastim
- Drug: cytarabine
- Drug: idarubicin
- Drug: dexamethasone
- Drug: thioguanine
- Drug: etoposide
- Drug: methotrexate
- type
- EXPERIMENTAL
- label
- Arm II (combination chemotherapy)
- description
- 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
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 21 Years
Show eligibility criteria text
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 therapyReferences
Publications (6)
- DERIVEDScheidegger N, Schneider C, Alexe G, Wang YC, Alonzo TA, Basanthakumar A, Bourgeois WA, Dudkiewicz-Garbicz J, Khalid D, Merickel LA, Perry JA, Ries RE, Salhotra S, Taillon A, Gamis A, Aplenc R, Harris MH, Wunderlich M, Armstrong SA, Pollard JA, Meshinchi S, Pikman Y, Stegmaier K. Combining menin and MEK inhibition to target poor prognosis KMT2A-rearranged RAS pathway-mutant acute myeloid leukemia. Blood Adv. 2026 Jul 14;10(13):4757-4771. doi: 10.1182/bloodadvances.2025016208. PMID 42085603
- DERIVEDTarlock 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 38295280
- DERIVEDBertrums 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 36815378
- DERIVEDHo 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
- DERIVEDPollard 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.