Clinical trial · Interventional
Combination Chemotherapy in Treating Young Patients With Down Syndrome and Acute Myeloid Leukemia or Myelodysplastic Syndromes
The Treatment of Down Syndrome Children With Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS) Under the Age of 4 Years
- 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)
This phase III trial is studying how well combination chemotherapy works in treating young patients with Down syndrome and acute myeloid leukemia or myelodysplastic syndromes. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more cancer cells.
Conditions
Conditions (15)
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 Basophilic Leukemia | Childhood Acute Basophilic Leukemia | ONTOLOGY_EXACT | 0.98 |
| Childhood Acute Eosinophilic Leukemia | — | UNRESOLVED | — |
| Childhood Acute Erythroleukemia (M6) | Acute Erythroid Leukemia | ALIAS | 0.85 |
| Childhood Acute Megakaryocytic Leukemia (M7) | Childhood Acute Megakaryoblastic Leukemia | ALIAS | 0.90 |
| Childhood Acute Minimally Differentiated Myeloid Leukemia (M0) | Childhood Acute Myeloid Leukemia with Minimal Differentiation | 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) |
Interventions
Interventions (6)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| asparaginase | Drug | Asparaginase | ALIAS |
| cytarabine | Drug | Cytarabine | ALIAS |
| daunorubicin hydrochloride | Drug | Daunorubicin | ALIAS |
| etoposide | Drug | Etoposide | ALIAS |
| laboratory biomarker analysis | Other | — | UNRESOLVED |
| thioguanine | Drug | Thioguanine | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Treatment (combination chemotherapy)
- description
- INDUCTION THERAPY COURSE I: Patients receive cytarabine IT on day 1 and cytarabine IV continuously over 96 hours, daunorubicin hydrochloride IV continuously, and oral thioguanine BID on days 1-4. COURSE II: Patients receive high-dose cytarabine IV over 3 hours BID on days 1, 2, 8, and 9 and asparaginase (IM) on days 2 and 9. COURSE III: Patients receive treatment as in course I. COURSE IV: Patients receive cytarabine IV, daunorubicin hydrochloride IV, and oral thioguanine as in course I INTENSIFICATION THERAPY: Patients receive cytarabine IV continuously over 168 hours on days 1-7 and etoposide IV over 1 hour on days 1-3. Treatment repeats every 28 days for 2 courses in the absence of disease progression or unacceptable toxicity.
- interventionNames
- Drug: asparaginase
- Drug: daunorubicin hydrochloride
- Drug: cytarabine
- Drug: thioguanine
- Drug: etoposide
- Other: laboratory biomarker analysis
Primary outcomes (2)
- measure
- Event-free Survival (EFS) at 3 Years
- timeFrame
- Time from study entry to induction failure, relapse, or death assessed at 3 years.
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 4 Years
Show eligibility criteria text
Inclusion Criteria: * Diagnosis DS or DS mosaicism by karyotype or chromosomal analysis * Diagnosis of myelodysplastic syndromes (MDS) with \< 30% blasts or acute myeloid leukemia (AML) * Newly diagnosed disease * Patients with a history of transient myeloproliferative disorder (TMD) are eligible provided the patient is diagnosed with AML or MDS at \> 90 days of age AND meets either of the following criteria: * At least 30% blasts in the bone marrow regardless of time since resolution of TMD * More than 8 weeks since resolution of TMD with ≥ 5% blasts in the bone marrow * Immunophenotype required for study entry * No promyelocytic leukemia * Shortening fraction ≥ 27% by echocardiogram OR ejection fraction ≥ 50% by radionuclide angiogram * Bilirubin ≤ 1.5 times upper limit of normal (ULN) * AST or ALT \< 2.5 times ULN * Creatinine adjusted according to age as follows: * No greater than 0.4 mg/dL (≤ 5 months) * No greater than 0.5 mg/dL (6 months -11 months) * No greater than 0.6 mg/dL (1 year-23 months) * No greater than 0.8 mg/dL (2 years-5 years) * No greater than 1.0 mg/dL (6 years-9 years) * No greater than 1.2 mg/dL (10 years-12 years) * No greater than 1.4 mg/dL (13 years and over \[female\]) * No greater than 1.5 mg/dL (13 years to 15 years \[male\]) * No greater than 1.7 mg/dL (16 years and over \[male\]) * Creatinine clearance or radioisotope glomerular filtration rate at least 70 mL/min * No evidence of dyspnea at rest * No exercise intolerance * Pulse oximetry \> 94% * No prior chemotherapy, radiotherapy, or any antileukemic therapy * Intrathecal cytarabine therapy given at diagnosis allowed * Prior therapy for TMD allowed
References
Publications (1)
- DERIVEDTaub JW, Berman JN, Hitzler JK, Sorrell AD, Lacayo NJ, Mast K, Head D, Raimondi S, Hirsch B, Ge Y, Gerbing RB, Wang YC, Alonzo TA, Campana D, Coustan-Smith E, Mathew P, Gamis AS. Improved outcomes for myeloid leukemia of Down syndrome: a report from the Children's Oncology Group AAML0431 trial. Blood. 2017 Jun 22;129(25):3304-3313. doi: 10.1182/blood-2017-01-764324. Epub 2017 Apr 7. PMID 28389462