NCT00369317|CompletedPhase 3ResultsDMC

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

Children's Oncology Group ClinicalTrials.gov

Compare

5 other identifiers

AAML0431NCI-2009-00318CDR0000492776COG-AAML0431U10CA098543

Study Type

interventional

Target

205

Locations

4 countries

Sites

Timeline

RegisteredAug 2006

StartedMar 2007

CompletionDec 2021

Brief Summary

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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

205

participants targeted

Target at P25-P50 for phase_3

Timeline

Completed

Started Mar 2007

Longer than P75 for phase_3

Geographic Reach

4 countries

97 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

14.8 years study duration

First Submitted

Initial submission to the registry

August 24, 2006

Completed

5 days until next milestone

First Posted

Study publicly available on registry

August 29, 2006

Completed

6 months until next milestone

Study Start

First participant enrolled

March 1, 2007

Completed

6.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2013

Completed

1.1 years until next milestone

Results Posted

Study results publicly available

January 13, 2015

Completed

7 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2021

Completed

Last Updated

January 28, 2022

Status Verified

March 1, 2021

Enrollment Period

6.8 years

First QC Date

August 24, 2006

Results QC Date

January 6, 2015

Last Update Submit

January 20, 2022

Conditions

Childhood Acute Basophilic Leukemia Childhood Acute Eosinophilic Leukemia Childhood Acute Erythroleukemia (M6)Childhood Acute Megakaryocytic Leukemia (M7)Childhood Acute Minimally Differentiated Myeloid Leukemia (M0)Childhood Acute Monoblastic Leukemia (M5a)Childhood Acute Monocytic Leukemia (M5b)Childhood Acute Myeloblastic Leukemia With Maturation (M2)Childhood Acute Myeloblastic Leukemia Without Maturation (M1)Childhood Acute Myelomonocytic Leukemia (M4)Childhood Myelodysplastic Syndromes de Novo Myelodysplastic Syndromes Secondary Acute Myeloid Leukemia Secondary Myelodysplastic Syndromes Untreated Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies

Outcome Measures

Primary Outcomes (2)

Event-free Survival (EFS) at 3 Years
Time from study entry to induction failure, relapse, or death assessed at 3 years.
Overall Survival (OS) at 3 Years
Time from study entry to death, assessed at 3 years.

Secondary Outcomes (9)

Induction Remission Rate
End of induction therapy (day 112)
Percentage of Patients Experiencing Grade 3 or 4 Toxicity Assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
From the beginning of induction therapy to the end of intensification therapy
Prevalence of Leukemia Phenotype of DS Patients < 4 Years of Age at Diagnosis by Flow Cytometry
At the start of therapy
Prevalence of of GATA1 Mutations of DS Patients < 4 Years of Age at Diagnosis
At baseline and at the end of therapy (intensification) or disease relapse
Proportions of Patients in Morphologic Remission With Positive MRD by Flow Cytometry
After Induction I therapy (day 28 from start of therapy)
+4 more secondary outcomes

Study Arms (1)

Treatment (combination chemotherapy)

EXPERIMENTAL

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.

Drug: asparaginaseDrug: daunorubicin hydrochlorideDrug: cytarabineDrug: thioguanineDrug: etoposideOther: laboratory biomarker analysis