Low-Dose Cytarabine in Treating Infants With Down Syndrome and Transient Myeloproliferative Disorder
|ClinicalTrials.gov Identifier: NCT00411281|
Recruitment Status : Withdrawn (Per Group Chair: This study will not move forward.)
First Posted : December 13, 2006
Last Update Posted : September 30, 2015
RATIONALE: Drugs used in chemotherapy, such as cytarabine, work in different ways to stop the growth of abnormal cells, either by killing the cells or by stopping them from dividing. Giving low-doses of cytarabine may be an effective treatment for Down syndrome and transient myeloproliferative disorder. Sometimes the disease may not need treatment until it progresses. In this case, observation may be sufficient.
PURPOSE: This phase III trial is studying low-dose cytarabine to see how well it works in treating infants with Down syndrome and transient myeloproliferative disorder.
|Condition or disease||Intervention/treatment||Phase|
|Leukemia||Drug: cytarabine Procedure: observation||Phase 3|
- Determine whether very low-dose cytarabine can improve event-free survival (EFS) rates in infants with high-risk transient myeloproliferative disorder (TMD), using high-risk TMD patients from clinical trial COG-A2971 for historic comparison, and in infants with intermediate-risk TMD, using intermediate-risk TMD patients from clinical trial COG-A2971 for historic comparison.
- Maintain the current high overall EFS rate in low-risk TMD patients.
- Assess the toxicity of this regimen in these patients.
OUTLINE: This is a nonrandomized, multicenter, crossover study. Patients are stratified according to disease risk (high or intermediate vs low).
- Group I (patients with high- or intermediate-risk transient myeloproliferative disorder [TMD]): Patients receive very low-dose cytarabine subcutaneously twice daily on days 1-7. Treatment repeats every 14 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving stable disease or complete or hepatic clinical remission undergo observation.
- Group II (patients with low-risk TMD): Patients are observed. If symptoms of intermediate- or high-risk disease develop, patients may crossover to group I.
After completion of study treatment, patients are followed periodically for 10 years.
PROJECTED ACCRUAL: A total of 180 patients will be accrued for this study.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Treatment of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)|
|Study Start Date :||March 2006|
|Primary Completion Date :||November 2007|
|Study Completion Date :||November 2007|
Experimental: Group I
Patients receive very low-dose cytarabine subcutaneously twice daily on days 1-7. Treatment repeats every 14 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving stable disease or complete or hepatic clinical remission undergo observation.
Patients are observed. If symptoms of intermediate- or high-risk disease develop, patients may crossover to group I.
- Event-free survival
- Overall survival
- Disease-related mortality
- Percentage of patients experiencing grade 3-4 toxicity
- Incidence of subsequent leukemia in patients for whom transient myeloproliferative disorder is resolved
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00411281
|Study Chair:||April D. Sorrell, MD||Rutgers Cancer Institute of New Jersey|
|Study Chair:||Jeffrey Taub, MD||Children's Hospital of Michigan|