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Clinical Trials in Canada / NCT02521493
Active, not recruiting Phase 3

Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome

NCT02521493 · tracked via the Priya Life Science Canada tracker
Phase
Phase 3
Started
2015-12-23
Last updated
2026-08-19

Condition(s) studied

Acute Myeloid LeukemiaDown SyndromeMyelodysplastic SyndromeMyeloid Leukemia Associated With Down SyndromeMyeloproliferative Neoplasm

Investigational drug(s) / intervention(s)

Asparaginase →Asparaginase Erwinia chrysanthemi →Cytarabine →Daunorubicin Hydrochloride →Etoposide →Laboratory Biomarker AnalysisMitoxantrone Hydrochloride →Thioguanine →

Asparaginase: Given IM or IV

Asparaginase Erwinia chrysanthemi: Given IM or IV

Cytarabine: Given IT and IV

Daunorubicin Hydrochloride: Given IV

Etoposide: Given IV

Laboratory Biomarker Analysis: Correlative studies

Mitoxantrone Hydrochloride: Given IV

Thioguanine: Given PO

Study summary

This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Response-based chemotherapy separates patients into different risk groups and treats them according to how they respond to the first course of treatment (Induction I). Response-based treatment may be effective in treating acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome while reducing the side effects.

Eligibility

Sex
ALL
Min age
91 Days
Max age
3 Years
Healthy volunteers
No
Inclusion Criteria: * Patients must have constitutional trisomy 21 (Down syndrome) or trisomy 21 mosaicism (by karyotype or fluorescence in situ hybridization \[FISH\]) * Patient has one of the following: * Patient has previously untreated de novo AML and meets the criteria for AML with \>= 20% bone marrow blasts as set out in the World Health Organization (WHO) Myeloid Neoplasm classification * Attempts to obtain bone marrow either by aspirate or biopsy must be made unless clinically prohibitive; in cases where it is clinically prohibitive, peripheral blood with an excess of 20% blasts and in which adequate flow cytometric and cytogenetics/FISH testing is feasible can be substituted for the marrow exam at diagnosis * Patient has cytopenias and/or bone marrow blasts but does not meet the criteria for the diagnosis of AML (WHO Myeloid Neoplasm classification) because of \< 20% marrow blasts and meets the criteria for a diagnosis of myelodysplastic syndrome (MDS) * For patients who do not meet criteria for AML or MDS as outlined above; patient has a history of transient myeloproliferative disorder (which may or may not have required chemotherapy intervention and: * Is \> 8 weeks since resolution of transient myeloproliferative disease (TMD) with \>= 5% blasts, OR * Has an increasing blast count (\>= 5%) in serial bone marrow aspirates performed at least 4 weeks apart * Children who have previously received chemotherapy, radiation therapy or any anti-leukemic therapy are not eligible for this protocol, with the exception of cytarabine for the treatment of TMD * There are no minimal organ function requirements for enrollment on this study * Note: Previous cardiac repair with sufficient cardiac function is not an exclusion criteria * Each patient's parents or legal guardians must sign a written informed consent * All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human subjects research must be met Exclusion Criteria: * Patients with promyelocytic leukemia (French-American-British \[FAB\] M3) * Prior therapy * Patients =\< 30 days from the last dose of cytarabine used for treatment of TMD

Primary outcome measure(s)

Trial sites (191)

FacilityCityRegionStatus
Children's Hospital of Alabama Birmingham Alabama
Providence Alaska Medical Center Anchorage Alaska
Banner Children's at Desert Mesa Arizona
Phoenix Childrens Hospital Phoenix Arizona
Arkansas Children's Hospital Little Rock Arkansas
Kaiser Permanente Downey Medical Center Downey California
Loma Linda University Medical Center Loma Linda California
Miller Children's and Women's Hospital Long Beach Long Beach California
Children's Hospital Los Angeles Los Angeles California
Cedars-Sinai Medical Center Los Angeles California
Valley Children's Hospital Madera California
UCSF Benioff Children's Hospital Oakland Oakland California
Kaiser Permanente-Oakland Oakland California
Children's Hospital of Orange County Orange California
Lucile Packard Children's Hospital Stanford University Palo Alto California
Sutter Medical Center Sacramento Sacramento California
University of California Davis Comprehensive Cancer Center Sacramento California
Rady Children's Hospital - San Diego San Diego California
Naval Medical Center -San Diego San Diego California
UCSF Medical Center-Mission Bay San Francisco California
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center Torrance California
Children's Hospital Colorado Aurora Colorado
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center Denver Colorado
Connecticut Children's Medical Center Hartford Connecticut
Yale University New Haven Connecticut
Alfred I duPont Hospital for Children Wilmington Delaware
MedStar Georgetown University Hospital Washington D.C. District of Columbia
Children's National Medical Center Washington D.C. District of Columbia
Broward Health Medical Center Fort Lauderdale Florida
Golisano Children's Hospital of Southwest Florida Fort Myers Florida
UF Health Cancer Institute - Gainesville Gainesville Florida
Memorial Regional Hospital/Joe DiMaggio Children's Hospital Hollywood Florida
Nemours Children's Clinic-Jacksonville Jacksonville Florida
Palms West Radiation Therapy Loxahatchee Groves Florida
University of Miami Miller School of Medicine-Sylvester Cancer Center Miami Florida
Nicklaus Children's Hospital Miami Florida
AdventHealth Orlando Orlando Florida
Arnold Palmer Hospital for Children Orlando Florida
Nemours Children's Hospital Orlando Florida
Nemours Children's Clinic - Pensacola Pensacola Florida

+ 151 more sites — see the full list on the official registry below.

More Children's Oncology Group trials in Canada

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT02521493 on ClinicalTrials.gov ↗ ← All trials in Canada